Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
- The importance and advantages of microbial systems in biopharmaceuticals
- Platform for non-mAb biopharmaceutical development
- High Throughput process development
- CASPON technology a solution for the development of therapeutic peptides
- Future prospects, such as sustainability and competitiveness
Cell Line Development plays a crucial role in establishing Master Cell Banks for clinical and commercial biomanufacturing. This involves creating subclones and undergoing multiple stages of rigorous assessment, leading to the selection of a final clone used for the project's entire duration. Decision-making in this process hinges on extensive datasets obtained from advanced analytical methods. The introduction of high-throughput platforms like the Berkeley Light Beacon and automated micro-bioreactor systems has resulted in generating vast datasets, which often consist of thousands of data points in each experiment. Moreover, the need to integrate process and performance data from various scales, including deep-well plates, shake flasks, and bioreactor processes, is essential for a thorough analysis. Collectively, these factors pose significant challenges in data processing and analysis, which are critical for informed decisionmaking in Cell Line Development. Here, we propose a holistic method for digitizing the entire cell line development and selection process. Our approach begins with implementing laboratory and data automation tools to streamline the generation and handling of raw data. We then establish automated data pipelines using the Databricks platform, enabling the integration of various data types and data of different scales into a specially designed database. This database comprehensively encompasses data on cell line creation, assessment, and selection. Additionally, we develop visualization dashboards linked in real-time to the database, significantly reducing time spent on data processing. Finally, we leverage this streamlined data to build predictive models using open-source Python machine-learning algorithms, enhancing the cell line selection process. Our proposed digital framework ensures a data-driven approach, optimizing the selection of highquality cell lines for clinical and commercial manufacturing purposes.
- Approaches to reaching high yield downstream purification
- Removing aggregates and contaminates from your titer
- Removal of partially-full capsids from your full-capsid yield
- Tailored design and engineering of polymeric nanoparticles for optimal cell interaction, targeting, and therapeutic delivery.
- Diverse delivery strategies and tissue targeting approaches for specific disease applications
- Effective encapsulation techniques to preserve cell viability, functionality, and differentiation potential during delivery
- Case study: how are these being explored/used
- Tailored design and engineering of polymeric nanoparticles for optimal cell interaction, targeting, and therapeutic delivery.
- Diverse delivery strategies and tissue targeting approaches for specific disease applications
- Effective encapsulation techniques to preserve cell viability, functionality, and differentiation potential during delivery
- Case study: how are these being explored/used
In the biopharma industry, various techniques are utilized to enhance yield and quality of the target protein produced by stable cell pools and accelerate overall CLD timeline. In this presentation, we will show a case study of a method for minipool productivity enrichment via co-expression of the target protein with a fluorescent biosensor protein using an IRES, combined with state-of-the-art automation tools to allow productivity enhancement and reduce timeline for overall cell line development efforts.
This presentation delves into the evolving landscape of supply chain management for CGTs, offering insights into the latest developments that are reshaping the industry. The session begins with an overview of traditional supply chain practices, highlighting their limitations and the pressing need for adaptation in an ever-changing global market. From there, it transitions into an exploration of cutting-edge innovations such as artificial intelligence (AI), machine learning, blockchain technology, and Internet of Things (IoT). These technologies are revolutionizing supply chain processes, enhancing efficiency, visibility, and responsiveness across the entire network. We will also consider predictive analytics to digital twins and autonomous systems, which can empower organizations to anticipate disruptions, optimize inventory management, and streamline operations for maximum effectiveness. In addition, we will consider sustainability initiatives, circular economy practices, agile supply chains, and the integration of advanced robotics and automation. By offering a comprehensive overview of these innovations, tools, and trends, attendees will be equipped with the knowledge and insights necessary to navigate the complexities of modern supply chain management.
This presentation delves into the evolving landscape of supply chain management for CGTs, offering insights into the latest developments that are reshaping the industry. The session begins with an overview of traditional supply chain practices, highlighting their limitations and the pressing need for adaptation in an ever-changing global market. From there, it transitions into an exploration of cutting-edge innovations such as artificial intelligence (AI), machine learning, blockchain technology, and Internet of Things (IoT). These technologies are revolutionizing supply chain processes, enhancing efficiency, visibility, and responsiveness across the entire network. We will also consider predictive analytics to digital twins and autonomous systems, which can empower organizations to anticipate disruptions, optimize inventory management, and streamline operations for maximum effectiveness. In addition, we will consider sustainability initiatives, circular economy practices, agile supply chains, and the integration of advanced robotics and automation. By offering a comprehensive overview of these innovations, tools, and trends, attendees will be equipped with the knowledge and insights necessary to navigate the complexities of modern supply chain management.
Dark Horse Consulting Representative
Dark Horse Consulting Representative
This presentation describes a process characterization (PC) study that evaluated the effect of three vial thaw parameters using a fractional factorial design. This study aimed to identify an optimal thawing process that maximizes cell viability and density. Results from this study have contributed to the standardization and refinement of the vial thaw process, particularly through a DOE approach focused on two molecules, thereby establishing best practices for the vial thaw procedure platform
Cell-based manufacturing of gene therapy molecules presents multiple challenges in maintaining high product yield and quality. Traditionally, isolating the desired DNA molecules is challenging. Additionally, cell-based approaches require long manufacturing timelines. To address these speed and quality challenges, we developed an enzymatic method capable of producing DNA drug substance at g/L scale and with >99% purity using chromatographic methods. This cell-free process has enabled highly accelerated production timelines compared to cell-based methods and the enzymatic platform facilitates diverse production application with constructs up to at least 7000bp in size.
Cell-based manufacturing of gene therapy molecules presents multiple challenges in maintaining high product yield and quality. Traditionally, isolating the desired DNA molecules is challenging. Additionally, cell-based approaches require long manufacturing timelines. To address these speed and quality challenges, we developed an enzymatic method capable of producing DNA drug substance at g/L scale and with >99% purity using chromatographic methods. This cell-free process has enabled highly accelerated production timelines compared to cell-based methods and the enzymatic platform facilitates diverse production application with constructs up to at least 7000bp in size.
- Reducing wait times and logistical magnitude to reduce overhead costs
- Increasing accessibility, distributing to multiple locations closer to patient populations and remove transportation cost barriers
- How QC release testing and material kitting and management will be handled for POC manufacturing in this ecosystem.
- Establishing harmonized regulatory standards across different regions
- Implementing robust oversight mechanisms to ensure compliance with regulatory requirements and maintain product in a distributed manufacturing model.
- EMA vs FDA approach
- Reducing wait times and logistical magnitude to reduce overhead costs
- Increasing accessibility, distributing to multiple locations closer to patient populations and remove transportation cost barriers
- How QC release testing and material kitting and management will be handled for POC manufacturing in this ecosystem.
- Establishing harmonized regulatory standards across different regions
- Implementing robust oversight mechanisms to ensure compliance with regulatory requirements and maintain product in a distributed manufacturing model.
- EMA vs FDA approach
Abstract TBC
Abstract TBC
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
Continuous improvement methodology has been employed for many years to improve manufacturing where effectiveness depends on team experience. Advanced modeling technology for biomanufacturing promises to make continuous improvement methodology more widespread and allow teams to achieve expert results with far less experience. This presentation will illustrate model based continuous improvement across a drug substance biomanufacturing process using an industrial case study.
We developed CRISPR-Cas9 mediated engineered universal stem cells, via elimination of MHC-I/II and targeted insertions of NK and macrophage-resistant factors. We explored the use of a kill switch to remove cells if required. Our precise gene-editing approach generated well characterized GMP compatible clonal hypoimmunogenic lines, minimizing off-target effects. These cells differentiated into human ventricular progenitor cells maturing into cardiomyocytes. This work paves the way for off-the-shelf, allogeneic cell therapy applications.
- Process design and implementation for bi- and multi-specfic ADCs
- Elucidating the drug to antibody ratio (DAR) of your product
- Analytical considerations for the characterisation of complex biologics
- Process design and implementation for bi- and multi-specfic ADCs
- Elucidating the drug to antibody ratio (DAR) of your product
- Analytical considerations for the characterisation of complex biologics
- How can early-stage/research professionals best prepare for GMP
- Critical aspects to keep in mind transitioning from early-stage development into more commercial manufacturing.
- Examples from experiences and lessons learnt
- Cross-team collaboration and pre-emptively preventing down the road bottlenecks
- How can early-stage/research professionals best prepare for GMP
- Critical aspects to keep in mind transitioning from early-stage development into more commercial manufacturing.
- Examples from experiences and lessons learnt
- Cross-team collaboration and pre-emptively preventing down the road bottlenecks
- How can early-stage/research professionals best prepare for GMP
- Critical aspects to keep in mind transitioning from early-stage development into more commercial manufacturing.
- Examples from experiences and lessons learnt
- Cross-team collaboration and pre-emptively preventing down the road bottlenecks
- How can early-stage/research professionals best prepare for GMP
- Critical aspects to keep in mind transitioning from early-stage development into more commercial manufacturing.
- Examples from experiences and lessons learnt
- Cross-team collaboration and pre-emptively preventing down the road bottlenecks
- How can early-stage/research professionals best prepare for GMP
- Critical aspects to keep in mind transitioning from early-stage development into more commercial manufacturing.
- Examples from experiences and lessons learnt
- Cross-team collaboration and pre-emptively preventing down the road bottlenecks
Antibody drug conjugate therapeutics are an increasingly common modality due to their targeted ability to deliver various cytotoxic or immune-modulating payloads to specific cell types or tissues. This presentation covers a case study navigating the added complexity of selecting an appropriate mAb binder, payload, conjugation method, and drug-antibody ratio with emphasis on opportunities and challenges to accelerate pre-clinical development and IND filing.
Considerations when designing a scale up strategy; Tools for scale up calculation and facility fit assessment; Case studies.
The focus on accelerated development of new modalities for advanced therapies and challenges with complex drug candidates has driven adoption of novel concepts and technologies in biopharmaceutical development and manufacturing. The active drug substances have become more structurally complex and R&D timelines continue to be compressed, the technologies necessary to provide safe, robust and economical access to these molecules needs to keep pace. Emerging technologies such as continuous processing, automation, high throughput experimentation, predictive modeling in an integrated fashion can expedite process development and commercial manufacturing while ensuring efficiency and delivering quality products. All these aspects help to minimize the time and cost associated with development and manufacture of drugs and bring medicines to patients more effectively. This presentation will share AbbVie’s strategy and experiences in integrating such end-to-end technologies into process development and manufacturing facilities and discuss the challenges and opportunities associated with our approaches.
This presentation will cover the strategy for clone and process platform selection for a therapeutic fusion protein. The selection process involves generating clonal cell lines, assessing the cell culture process performance, and evaluating product quality attributes in automated microbioreactors. Perspectives from product development, analytical product quality comparability, cost of good reduction, and strategies for further process development will be discussed.
We present a digital twin model-based process control (MPC) strategy for the successful glucose feeding in a bioreactor using only daily offline measurements. Our MPC strategy is composed of two kinds of formulars for predicting 1) glucose feeding amounts at daily sample time-points and 2) glucose feeding amounts at unsampled time-points.
- Navigating the complex grey area of manufacturing for Phase 1 and 2 trials
- Keeping efficiency while complying with limited guidance
- Need conversation between regulators and developers
- Lessons learnt and case studies from companies moving between clinical phases
- Therapeutic Developer/CDMO dynamics in the phase 1 and beyond
- How to best leverage CDMO expertise and capital efficiency
- Navigating the complex grey area of manufacturing for Phase 1 and 2 trials
- Keeping efficiency while complying with limited guidance
- Need conversation between regulators and developers
- Lessons learnt and case studies from companies moving between clinical phases
- Therapeutic Developer/CDMO dynamics in the phase 1 and beyond
- How to best leverage CDMO expertise and capital efficiency
- Ensuring the quality and consistency of the end product
- Conjugation challenges associated with drug design
- Effective collaboration with CMOs in Preparation for commercialization of ADCs
- Ensuring the quality and consistency of the end product
- Conjugation challenges associated with drug design
- Effective collaboration with CMOs in Preparation for commercialization of ADCs
Ultragenyx is developing an investigational gene therapy, UX701, an adeno-associated viral vector-based gene therapy product, for the treatment of Wilson disease. UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene. Ultragenyx has initiated Cyprus2+, a seamless Phase 1/2/3 study of a single intravenous infusion of UX701 in Wilson disease (NCT04884815). UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™. This presentation reviews our first late-stage development activities for our upstream Pinnacle PCL™ platform process including parameter risk assessment, scale-down model qualification, and process characterization, to establish process controls for the upstream unit operations in our 2000L manufacturing process. The process characterization studies help us understand our parameter space and mitigate operational and quality risks which are critical for successful implementation of a robust commercial manufacturing process to provide consistent product quality throughout the product’s lifecycle.
Ultragenyx is developing an investigational gene therapy, UX701, an adeno-associated viral vector-based gene therapy product, for the treatment of Wilson disease. UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene. Ultragenyx has initiated Cyprus2+, a seamless Phase 1/2/3 study of a single intravenous infusion of UX701 in Wilson disease (NCT04884815). UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™. This presentation reviews our first late-stage development activities for our upstream Pinnacle PCL™ platform process including parameter risk assessment, scale-down model qualification, and process characterization, to establish process controls for the upstream unit operations in our 2000L manufacturing process. The process characterization studies help us understand our parameter space and mitigate operational and quality risks which are critical for successful implementation of a robust commercial manufacturing process to provide consistent product quality throughout the product’s lifecycle.
Roundtable Discussions
Cell Culture Optimization
- Cell Line Development: Selecting and developing an optimal cell line is crucial for achieving high product yields and quality. Identifying a cell line with desirable characteristics, such as growth rate, productivity, and stability, can be a time-consuming and complex process.
- Cultivation Conditions: Maintaining optimal conditions for cell growth and productivity is challenging. Factors like temperature, pH, nutrient availability, and gas exchange need to be carefully controlled to ensure optimal performance. Achieving consistent and reproducible results across large-scale bioreactors adds another layer of complexity
Bioreactor Scale-Up:
- Transition from Lab Scale to Production Scale: Scaling up bioprocesses from small laboratory bioreactors to large-scale production facilities can lead to challenges in maintaining consistent conditions. Factors like mixing, mass transfer, and heat dissipation become more complex as the scale increases. Achieving uniform distribution of nutrients and gases becomes crucial for maintaining cell health and product quality. Take into account Perfusion versus Fed-Batch.
- Bioreactor Design: The design of bioreactors for large-scale production must consider factors such as hydrodynamics, shear stress, and heat transfer to ensure optimal cell growth and product formation. Choosing the right type of bioreactor and ensuring scalability without compromising performance is a significant challenge
Process Monitoring and Control:
- Real-time Monitoring: Continuous monitoring of various parameters, such as cell viability, metabolite concentrations, and product titer, is essential for process control and optimization. Implementing reliable real-time monitoring techniques can be challenging, particularly for complex bioprocesses.
- Process Control: Maintaining tight control over the bioprocess is crucial to achieve consistent product quality. Controlling variables such as pH, dissolved oxygen, and nutrient concentrations requires advanced control strategies. Deviations from optimal conditions can negatively impact cell growth, viability, and product quality
An acceleration in the speed at which therapies are breaking through from basic scientific discovery to development is placing a premium on innovative business development models. Biotech innovators, Pharma, and VCs alike are competing for successful win-win deal-making models in the face of novel personalized applications of gene therapies, gene editing, regenerative medicines, vaccines, therapies, and companion biomarkers. This session will cover best practices and strategies in finding the right partnerships to engage.
- Emphasize the advantages of not dealing with cell growth, maintenance, and contamination
- Explore innovations and strategies aimed at making cell-free bioprocessing economically feasible
- Reduce COGs for large-scale cell-free systems
- Exploring the unique challenges of T-cell engager manufacturing compared to traditional biologics
- Pre-emptive design and optimization of robust production processes
- Case study example
- Exploring the unique challenges of T-cell engager manufacturing compared to traditional biologics
- Pre-emptive design and optimization of robust production processes
- Case study example
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
Continuous manufacturing has become more prevalent across the biopharmaceutical industry in recent years. The ambr250ht perfusion system stands as a promising option for bench-scale culture work, but first must be proven as a replacement for the more common 3L scale. Through statistical analysis, the differences and similarities between these scales can be investigated to qualify their equivalence
- Unpacking the current barriers to RNA gene therapy use
- Advancements in the processing of RNA to improve outcomes (remove unwanted cells, viral concentration, purification)
- Scalable processes
- Unpacking the current barriers to RNA gene therapy use
- Advancements in the processing of RNA to improve outcomes (remove unwanted cells, viral concentration, purification)
- Scalable processes
- What to expect?
- Common pitfalls
Chinese hamster ovary (CHO) cells are the most widely used mammalian host for industrial-scale production of mAbs and other protein biologics. Selection of high-producing cell lines is a major endeavor in the process of manufacturing a novel biologic and requires an extensive and lengthy screening campaign of several hundreds of clonally-derived cell lines. We have previously reported the development of an efficient cumate-inducible expression system. Here, we present a new GMP-banked parental cell line, amenable to both constitutive or cumate-inducible expression. We first present our process for selecting CHO pools in suspension culture and then cell lines using a semi-automated approach, where imaging analysis provides >99% probability that selected cell lines are single-cell derived. Recent plasmid engineering efforts allowed to increase cell line productivity by 75% where~70% of selected clones show stable expression after at least 60 generations in culture. We also present recent development of a selection approach allowing to screen for more productive CHO minipools prior to single cell cloning. Optimizing several parameters such as cDNA sequence, signal peptide sequence, and suspension minipool selection process, we were able to increase titer for IgG1s from 2.5-3.0 g/L to 5.5 g/L using commercial medium and feed. Finally, we present data supporting the use of stable cumate-inducible CHO pools for clinical development of trimeric SARS-CoV-2 spike subunit vaccine antigens.
Next generation manufacturing through implementation of advanced technologies is one of the current focus areas to enable agile, reliable, cost-effective, and timely production of biopharmaceuticals at highest quality standards. New modalities beyond standard mAB characteristics and complex supply chain logistics in a VUCA environment are factors to consider when designing the plant of the future and will impact the competitive advantage through application of advanced technology concepts. The talk will provide examples of deployment in context with business needs and added value.
Control and management of materials is a key pillar of GMP manufacturing. An initiative is in progress which aims to harmonize the approaches of material management between clinical and commercial Drug Substance production sites to increase efficiency and supply chain sustainability within the organization. We will present the background and drivers, strategies, and current status of this initiative.
- Specific improvements in cell culture media formulations
- The impact of enhanced media on cell growth and productivity
- Challenges and potential solutions in adopting better cell culture media
- Balancing pH and osmolality for an optimal culture environment
Cell lines derived from the caterpillar, Spodoptera frugiperda (Sf), are the most commonly used hosts in the baculovirus-insect cell system (BICS), but they can harbor Sf-rhabdovirus (Sf-RV) contaminants. In this presentation, I will review our efforts to isolate the first Sf-RV negative (Sf-RVN™) Sf cell line, as well as the characteristics of this cell line indicating it is an improved, seamless alternative for Sf-RV-contaminated lines.
- Can scale down models help with implementation?
Generic Raman spectroscopy models, built using data from multiple processes, offer a method for streamlining Raman model development and integration in upstream bioprocessing. Effective implementation of generic Raman models requires consideration of several factors, such as the presence of any biases in the calibration dataset. The large volume of data used to develop generic Raman models also makes them ideal for creating hierarchical Raman models, which use the outputs of one or more base models as the inputs for a new model—e.g., cell culture pH.
Transposase-mediated semi-targeted transgene integration systems deliver highly productive and genetically stable clones; however, it is sometimes challenging to characterize target gene integration sites for production cell lines with relatively higher gene copy numbers. This presentation will discuss the use of NGS and in silico predictive tools to support the generation of cell line stability data package to meet regulatory expectations.
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
- Scalable methods to optimize EV Isolation & Purification
- Efficient techniques for manipulating EV properties (size, surface ligands, cargo loading etc.) for enhanced targeting and in vivo delivery
- Challenges and potential strategies for adhering to strict GMP regulations for clinical-grade EV production.
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
The volumes of data generated by upstream bioprocesses during development, technology transfer and GMP production are only as good as the tools to make the data available for consumption and advanced data analytics. This talk will provide current highlights from our 12-year journey digitizing bench and pilot scale bioreactor processes to enable paperless data aggregation and generation of persona dashboards for daily analysis by data consumers and executives alike.
- What are the key criteria for choosing a cell or gene therapy manufacturing partner? (Expertise, capacity, regulatory knowledge, technology fit)
- Approaching start-up build from the ground up to develop new therapeutics
- Ensuring preparedness for CDMOs
- How can different stakeholders identify complementary strengths to build mutually beneficial partnerships?
- Best practices to ensure quality control and compliance throughout manufacturing.
- Building trust and transparency in long-term CGT manufacturing partnerships
- What are the key criteria for choosing a cell or gene therapy manufacturing partner? (Expertise, capacity, regulatory knowledge, technology fit)
- Approaching start-up build from the ground up to develop new therapeutics
- Ensuring preparedness for CDMOs
- How can different stakeholders identify complementary strengths to build mutually beneficial partnerships?
- Best practices to ensure quality control and compliance throughout manufacturing.
- Building trust and transparency in long-term CGT manufacturing partnerships
- What are the key criteria for choosing a cell or gene therapy manufacturing partner? (Expertise, capacity, regulatory knowledge, technology fit)
- Approaching start-up build from the ground up to develop new therapeutics
- Ensuring preparedness for CDMOs
- How can different stakeholders identify complementary strengths to build mutually beneficial partnerships?
- Best practices to ensure quality control and compliance throughout manufacturing.
- Building trust and transparency in long-term CGT manufacturing partnerships
- What are the key criteria for choosing a cell or gene therapy manufacturing partner? (Expertise, capacity, regulatory knowledge, technology fit)
- Approaching start-up build from the ground up to develop new therapeutics
- Ensuring preparedness for CDMOs
- How can different stakeholders identify complementary strengths to build mutually beneficial partnerships?
- Best practices to ensure quality control and compliance throughout manufacturing.
- Building trust and transparency in long-term CGT manufacturing partnerships
- Ensuring effective CMC, analytical development, and quality monitoring for the production of safe and consistent therapeutics in line with regulatory advice
- Anticipating potential bottlenecks and addressing these to ensure smooth scalability.
- Working with partners to assist in the manufacturing of next generation therapeutics.
- Ensuring effective CMC, analytical development, and quality monitoring for the production of safe and consistent therapeutics in line with regulatory advice
- Anticipating potential bottlenecks and addressing these to ensure smooth scalability.
- Working with partners to assist in the manufacturing of next generation therapeutics.
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
In an effort to access cutting edge research and stay ahead of the curve during this unprecedented time, investors and pharma companies are taking less risks. What long term trends have fizzled out and what is on the rise to increase innovation? Are new strategies sustainable as increasing investment rounds requiring more and more funds to obtain new treatments, therapies and technologies?
Leading biopharma experts and VCs will share their insights on how to engage investors, which trends to watch for, how to position technologies and discoveries to attract investors, and navigate this evolving landscape.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
*This event requires a separate ticket which can be purchased at a discount during your conference registration*
The BWB Awards @ Biotech Week Boston brings together the faces and names that make the Boston community the beating heart of the biotech world.
This evening celebratory gala features a night of networking, food, drink, entertainment and recognition on September 23rd, 2024 in Boston. We're excited to roll out the red carpet and honor the top individuals, teams, and organizations that make the life sciences ecosystem vibrant and innovative.
Full details on the BWB Awards 2024 can be found at https://informaconnect.com/bwb-awards/
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
This presentation will cover the points to consider for enabling multi-use systems in a Next Generation Biotechnology (NGB) facility. Multi-use systems improve operability while reducing cost and facility environmental impact. A framework for enabling multi use systems will be proposed including a review of batch definition, genealogies, hold times, product quality assessments, microbial control, compliance, validation, and regulatory considerations.
- How are regulatory agencies approaching the rapid adoption of advanced technologies?
- What will the regulatory framework for the implementation of AI look like?
- How can regulatory agencies and industry work together to make safe and smart manufacturing a reality?
- Applications of generative AI within bioprocessing and next-gen therapeutics manufacturing
- Optimization of process parameters and product design for efficient production
- Ethical considerations and responsible development of AI in biomanufacturing – what to keep in mind.
- Applications of generative AI within bioprocessing and next-gen therapeutics manufacturing
- Optimization of process parameters and product design for efficient production
- Ethical considerations and responsible development of AI in biomanufacturing – what to keep in mind.
Given the complexity of biological products, effective control strategy and life cycle management are critical to delivering quality products with intended efficacy. This presentation will focus on an overview and guidelines to develop and implements analytical control strategy approach to streamline and harmonize quality control testing and method life cycle.
- How do we define AI/ML?
- Which industries have already implemented AI/ML? How can the Biopharma industry learn from this?
- Is Biopharma prepared for this transition?
- Early adopter’s vs late adopters: Will there be any impact?
- How can small, mid-size, and large companies strategize for ML/AI implementation?
- Can it be beneficial for all areas of biopharmaceutical development (discovery, development, clinical and manufacturing)
- Cost reduction and accelerated timelines for project
In the slowly evolving landscape of bioprocess development and manufacturing, digital bioprocess-twins have emerged as potential accelerators. While advanced algorithms are at the heart of this endeavor, they are just one piece of the puzzle. The talk delves into key discussion points that are integral to this paradigm shift. The foundation of accelerated process development and automated process control starts with a clever experimental design, in-time data accessibility combined with powerful modeling algorithms. The talk will highlight the advantages of using hybrid modeling, while emphasizing the other critical aspects of his journey. Several industrial relevant upstream showcases for microbial and mammalian cell lines will be highlighted. Thereby, concepts to save experimental effort by up to 70% will be elaborated, and the modeling structure created in the late-stage development will be reused for real-time monitoring and control in the later stages. Additionally, a downstream optimization showcase for UF/DF/SPTFF will be highlighted.
The Cell Line Development (CLD) group within the Genomic Medicine Unit CMC department at Sanofi is dedicated to the establishment of best-in-class Producer Cell Lines (PCLs) for manufacturing of life-changing gene therapy products. The development of a robust PCL requires optimization of a multitude of process and operating factors; however, additional functionality can also be unlocked through the implementation of an automation-enabled and data-driven workflow. We have implemented several enhancements in our PCL development process, including liquid handling automation and state-of-the-art automated cell imaging and seeding systems. This newly developed automation-enabled next-generation process will lead future high-throughput capabilities for PCL development.
Production of biologics require adequate cellular capacity to maintain desired productivity and quality. Topics covered include how tuned energy supply improve secreted productivity in HEK293. How tuning of secretory helper proteins in CHO increase level of active sulfatase product 150-fold. We will also cover cell and protein engineering strategies for improved AAV production.
This presentation will focus on the technical and manufacturing capabilities for cell therapy commercialization with discussion of CMC requirements, manufacturing launch readiness, and supply considerations. Specific considerations for a cell therapy and partnership with contract manufacturing organizations will also be discussed including lessons learned.
This presentation will focus on the technical and manufacturing capabilities for cell therapy commercialization with discussion of CMC requirements, manufacturing launch readiness, and supply considerations. Specific considerations for a cell therapy and partnership with contract manufacturing organizations will also be discussed including lessons learned.
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
- Specific and strategic considerations for process and analytical development of advanced therapies
- Optimising and scaling-up of manufacturing operations post approval
- Preparing for regulatory filing
*This event requires a separate ticket which can be purchased at a discount during your conference registration*
The BWB Awards @ Biotech Week Boston brings together the faces and names that make the Boston community the beating heart of the biotech world.
This evening celebratory gala features a night of networking, food, drink, entertainment and recognition on September 23rd, 2024 in Boston. We're excited to roll out the red carpet and honor the top individuals, teams, and organizations that make the life sciences ecosystem vibrant and innovative.
Full details on the BWB Awards 2024 can be found at https://informaconnect.com/bwb-awards/
Shifting from batch to continuous manufacturing is a promising way of lowering manufacturing costs of by allowing operations to run simultaneously with reduced downtime, higher productivity, and at several-fold smaller scale. This is particularly promising for gene therapy products using recombinant adeno-associated viral vectors (AAV), as treatments currently cost up to USD 3.5 million per patient and have high cost-of-goods ranging up to USD 1 million per dose. In this talk, we present approaches for intensification of downstream AAV manufacturing processes using principles of continuous processing.
Disposable technology is being used more each year in the biotechnology industry. Disposable bioreactors allow the user to avoid expenses associated with cleaning, assembly and operations, as well as equipment validation. Disposable bioreactors have played a key role to meet the increasing run rate of the Cell Culture Pilot Plant while maintaining a high success rate, reducing labor costs, increasing efficiency, and lowering the risk of contamination. Recent effort to evaluate the next generation 10:1 turndown single use bioreactor with different cell retention devices to challenges oxygen mass transfer, carbon-dioxide stripping while assessing foaming, and vent filter sizing. This presentation will focus primarily on the advantage of the next generation high turndown single use bioreactor that comes with different sparger options (enhanced DHS and mircrosparger), and tubing for different cell retention device connection. Additionally, the high turndown provided a wide range of working volume allowing us to truly test the true scale down of the cell retention device for its maximum flux and filter throughput. The evaluation provided important system performance, operation experience, and cell culture performance data when comparing the next generation high turndown disposable system with respect to the legacy 2:1 and 5:1 turndown single use bioreactors, and conventional stainless-steel bioreactor system.
The biopharmaceutical industry is increasingly confronted with the dual challenges of complex product demands and a volatile market landscape, necessitating unprecedented levels of supply chain agility and resilience. This presentation addresses the critical role of digital innovation in transforming biopharma supply chains, enabling them to adapt swiftly to market fluctuations and unexpected disruptions. We explore the integration of advanced technologies such as artificial intelligence (AI), and machine learning (ML) to enhance visibility, optimize operations, and improve decision-making processes. Through case studies and industry insights, we demonstrate how digital tools can facilitate precise inventory management, robust cold chain logistics, and regulatory compliance, thereby reducing waste and ensuring timely delivery of vital products.
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
- An update on NIIMBL’s activities working towards key fundamentals
- Security of supply chains
- Flexibility of facilities to match varied and changing demand across portfolios of products
- Faster development with supply chains that can match this
- Sustainability for raw materials, components, and energy
- Addressing unique needs for vaccine modalities
- Optimization of PAT and tech transfer
- Scalability considerations
- Managing internal and external manufacturing operations
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
- The development of first in class novel complexed miRNA-based cellular reprogramming therapeutics
- Exploring the use of miRNA for the treatment of Alzheimer’s Disease, Diabetes and Heart Disease
- The development of first in class novel complexed miRNA-based cellular reprogramming therapeutics
- Exploring the use of miRNA for the treatment of Alzheimer’s Disease, Diabetes and Heart Disease
- Navigating diverse region-specific frameworks to expedite approvals.
- Build strategic partnerships with local distributors to facilitate market entry.
- Tailor marketing and patient education strategies
- Collaborating with local health services/distributors in preparation for product launch
- Ensuring preparedness for your supply/logistics chain across goal market
- E.g. Cryopreservation facility/availability when transporting
- Navigating diverse region-specific frameworks to expedite approvals.
- Build strategic partnerships with local distributors to facilitate market entry.
- Tailor marketing and patient education strategies
- Collaborating with local health services/distributors in preparation for product launch
- Ensuring preparedness for your supply/logistics chain across goal market
- E.g. Cryopreservation facility/availability when transporting
Mammalian cell bioprocesses have been monitored traditionally by off-line staining of cell samples. Newer techniques can offer rapid and continuous monitoring without manual sampling. Novel optical and dielectric protocols will be explained and discussed as alternatives for monitoring cells in bioprocesses. These methods can be used to provide growth profiles as well as the metabolic status of cell populations. They offer distinct advantages as in-line or at-line monitoring tools.
Aurion Biotech is taking their off-the-shelf, allogeneic cell therapy (AURN001) from the lab to clinical trials with the goal of becoming the first mass-scale cell therapy to help restore vision to 17M+ patients around the world with corneal endothelial disease. Building a mass scale cell therapy means:
- Fulfilling a significant unmet patient need caused by the global shortage of donor corneas for transplant, in ways that are accessible and equitable
- Navigating the highs and lows of manufacturing and scaling an allogeneic cell therapy for clinical trials, successfully managing a CDMO partner and establishing a development process that can be translated into a large-scale commercial product.
- Creating a breakthrough product that will transform the physician experience – one that is less complex and less invasive than corneal transplant (the current standard of care) and minimally invasive for patients.
Aurion Biotech is taking their off-the-shelf, allogeneic cell therapy (AURN001) from the lab to clinical trials with the goal of becoming the first mass-scale cell therapy to help restore vision to 17M+ patients around the world with corneal endothelial disease. Building a mass scale cell therapy means:
- Fulfilling a significant unmet patient need caused by the global shortage of donor corneas for transplant, in ways that are accessible and equitable
- Navigating the highs and lows of manufacturing and scaling an allogeneic cell therapy for clinical trials, successfully managing a CDMO partner and establishing a development process that can be translated into a large-scale commercial product.
- Creating a breakthrough product that will transform the physician experience – one that is less complex and less invasive than corneal transplant (the current standard of care) and minimally invasive for patients.
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
Transposase/Transposon platforms have become increasingly common for the development of robust high-expressing CHO cell lines for protein therapeutic manufacturing. Notably, these techniques use a single transposase/transposon pair to enable such outcomes. ATUM, as part of the Leap In Transposase platform, has developed a number of mutually orthogonal transposase/transposon pairs that can be used to serially engineer CHO, and other, cell lines in a robust manner. Indeed, this engineering can be used to not only increase the expression of transgenes, as is the case for a mAb therapeutics, but also knock-down the expression of endogenous genes to affect cellular physiology and/or product quality attributes … or both. This talk will provide examples of such engineering including a case study wherein three orthogonal Leap In Transposase/Transposon systems were implemented for the creation of a mAb expressing cell line with specific product quality attributes.
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
Glycosylation profiles are essential to the quality and potency of therapeutic biologics produced from CHO cells. Culture medium formulations significantly impact the N-glycosylation of biologics, which is critical for protein structure and bioactivities. In this case study, improvements in the culture medium by supplementing specific metal ion / small molecule components altered the N-glycan profile and enhanced its biological activity in-vivo.
- What Are the requirements across several countries?
- How can we align requirements and gain approval across multiple regions?
- Why do the requirements differ so much?
During this session, leaders from the Biopharma community will discuss their approach to creating, promoting, investing in, and accessing innovation, and ultimately, coming together to bring cures to patients.
In an effort to access cutting edge research and stay ahead of the curve during this unprecedented time, investors and pharma companies are taking less risks. What long term trends have fizzled out and what is on the rise to increase innovation? Are new strategies sustainable as increasing investment rounds requiring more and more funds to obtain new treatments, therapies and technologies?
Leading biopharma experts and VCs will share their insights on how to engage investors, which trends to watch for, how to position technologies and discoveries to attract investors, and navigate this evolving landscape.
An acceleration in the speed at which therapies are breaking through from basic scientific discovery to development is placing a premium on innovative business development models. Biotech innovators, Pharma, and VCs alike are competing for successful win-win deal-making models in the face of novel personalized applications of gene therapies, gene editing, regenerative medicines, vaccines, therapies, and companion biomarkers. This session will cover best practices and strategies in finding the right partnerships to engage.
Whether they’re public-private partnerships, CROs and biotechs or incubators and pharma companies, novel agreements between stakeholders are leveraging the resources and unique expertise to deliver previously only theoretical contributions to science. This session will walk through review successful and impactful partnerships that are shaping the future of drug development.
This past decade has seen an explosion of technologies that are transforming healthcare. From AI to cell and gene therapies to immunotherapies, we’ve seen significant advances in life sciences and the impact on patients, but what can we expect in the next decade? What are investors looking for in the next wave of breakthroughs? What areas of development excite them most? What new business models enable riskier investments to support the development of these novel methods and technologies?
One-to-one meetings will be available to schedule online throughout the duration of the digital event.
Meetings on this day will start at 8:00am EST.
The Startup Spotlight is a pitch competition featuring the most innovative startup biotech companies. This live pitch competition will give a group of hand selected startups the opportunity to pitch in front of the BioPharm America audience.
Startup Spotlight Presenters:
Atom Bioworks Inc.
Enrich Biosystems Inc.
GeneVentiv Therapeutics, Inc.
Opus Genetics
Qprotyn Inc.
SelSym Biotech
| 10:00 – 10:15 | Qatch Technologies |
| 10:15 – 10:25 | Bowhead Health |
| 10:30 – 10:45 | Dignify Therapeutics |
| 10:45 – 11:00 | Microvascular Therapeutics |
| 11:00 – 11:15 | XtalPi Inc |
| 10:45 - 11:00 | EydisBio Inc. |
| 11:00 - 11:15 | Mirimus, Inc. |
| 11:15 - 11:30 | TregTherapeutics |
| 11:30 - 11:45 | Aer Therapeutics, Inc. |
| 11:45 - 12:00 | Lumos Pharma Inc. |
| 12:00 - 12:15 | Shaperon Inc. |
1:00 - 1:10 Praetego Inc
1:20- 1:30 Peri-Nuc Labs
1:30 - 1:40 Adamas Nanotechnologies, Inc.
| 2:30 - 2:40 | ANYO Labs |
| 2:45 - 3:00 | Zucara Therapeutics Inc. |
| 3:00 - 3:15 | Lindy Biosciences, Inc. |
| 3:15 - 3:30 | HDT Bio |
| 3:30 - 3:45 | CellVax Therapeutics |
| 3:45 - 4:00 | Glycan Therapeutics |
| 4:00 - 4:15 | Caeregen Therapeutics |
- What is each step of the journey and the requirements?
- Focus on development, patent procedure, how to make an application to the FDA?
- What to expect?
- Common pitfalls
Examples of methods that are sensitive, specific, and capable of detecting minor differences between the biosimilar and the reference product.
Although many new bioprocessing technologies have evolved over the past 40 years, biosimilars still tend to be made using their legacy production processes. However, the ability to precisely evaluate critical quality attributes in finished drug substances has also evolved. This capability can provide a pathway for adopting powerful new upstream and downstream production methods, where drug impacts can be controlled.
- What Are the requirements across several countries?
- How can we align requirements and gain approval across multiple regions?
- Why do the requirements differ so much?
- Ensuring the quality and consistency of the end product
- Conjugation challenges associated with drug design
- Effective collaboration with CMOs in Preparation for commercialization of ADCs
- Process design and implementation for bi- and multi-specfic ADCs
- Elucidating the drug to antibody ratio (DAR) of your product
- Analytical considerations for the characterisation of complex biologics
- Exploring the unique challenges of T-cell engager manufacturing compared to traditional biologics
- Pre-emptive design and optimization of robust production processes
- Case study example
- Ensuring effective CMC, analytical development, and quality monitoring for the production of safe and consistent therapeutics in line with regulatory advice
- Anticipating potential bottlenecks and addressing these to ensure smooth scalability.
- Working with partners to assist in the manufacturing of next generation therapeutics.
- Applications of generative AI within bioprocessing and next-gen therapeutics manufacturing
- Optimization of process parameters and product design for efficient production
- Ethical considerations and responsible development of AI in biomanufacturing – what to keep in mind.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
Production of biologics require adequate cellular capacity to maintain desired productivity and quality. Topics covered include how tuned energy supply improve secreted productivity in HEK293. How tuning of secretory helper proteins in CHO increase level of active sulfatase product 150-fold. We will also cover cell and protein engineering strategies for improved AAV production.
- Emphasize the advantages of not dealing with cell growth, maintenance, and contamination
- Explore innovations and strategies aimed at making cell-free bioprocessing economically feasible
- Reduce COGs for large-scale cell-free systems
Transposase-mediated semi-targeted transgene integration systems deliver highly productive and genetically stable clones; however, it is sometimes challenging to characterize target gene integration sites for production cell lines with relatively higher gene copy numbers. This presentation will discuss the use of NGS and in silico predictive tools to support the generation of cell line stability data package to meet regulatory expectations.
We developed CRISPR-Cas9 mediated engineered universal stem cells, via elimination of MHC-I/II and targeted insertions of NK and macrophage-resistant factors. We explored the use of a kill switch to remove cells if required. Our precise gene-editing approach generated well characterized GMP compatible clonal hypoimmunogenic lines, minimizing off-target effects. These cells differentiated into human ventricular progenitor cells maturing into cardiomyocytes. This work paves the way for off-the-shelf, allogeneic cell therapy applications.
Roundtable Discussions
Cell Culture Optimization
- Cell Line Development: Selecting and developing an optimal cell line is crucial for achieving high product yields and quality. Identifying a cell line with desirable characteristics, such as growth rate, productivity, and stability, can be a time-consuming and complex process.
- Cultivation Conditions: Maintaining optimal conditions for cell growth and productivity is challenging. Factors like temperature, pH, nutrient availability, and gas exchange need to be carefully controlled to ensure optimal performance. Achieving consistent and reproducible results across large-scale bioreactors adds another layer of complexity
Bioreactor Scale-Up:
- Transition from Lab Scale to Production Scale: Scaling up bioprocesses from small laboratory bioreactors to large-scale production facilities can lead to challenges in maintaining consistent conditions. Factors like mixing, mass transfer, and heat dissipation become more complex as the scale increases. Achieving uniform distribution of nutrients and gases becomes crucial for maintaining cell health and product quality. Take into account Perfusion versus Fed-Batch.
- Bioreactor Design: The design of bioreactors for large-scale production must consider factors such as hydrodynamics, shear stress, and heat transfer to ensure optimal cell growth and product formation. Choosing the right type of bioreactor and ensuring scalability without compromising performance is a significant challenge
Process Monitoring and Control:
- Real-time Monitoring: Continuous monitoring of various parameters, such as cell viability, metabolite concentrations, and product titer, is essential for process control and optimization. Implementing reliable real-time monitoring techniques can be challenging, particularly for complex bioprocesses.
- Process Control: Maintaining tight control over the bioprocess is crucial to achieve consistent product quality. Controlling variables such as pH, dissolved oxygen, and nutrient concentrations requires advanced control strategies. Deviations from optimal conditions can negatively impact cell growth, viability, and product quality
This presentation describes a process characterization (PC) study that evaluated the effect of three vial thaw parameters using a fractional factorial design. This study aimed to identify an optimal thawing process that maximizes cell viability and density. Results from this study have contributed to the standardization and refinement of the vial thaw process, particularly through a DOE approach focused on two molecules, thereby establishing best practices for the vial thaw procedure platform
Considerations when designing a scale up strategy; Tools for scale up calculation and facility fit assessment; Case studies.
This presentation will cover the strategy for clone and process platform selection for a therapeutic fusion protein. The selection process involves generating clonal cell lines, assessing the cell culture process performance, and evaluating product quality attributes in automated microbioreactors. Perspectives from product development, analytical product quality comparability, cost of good reduction, and strategies for further process development will be discussed.
Glycosylation profiles are essential to the quality and potency of therapeutic biologics produced from CHO cells. Culture medium formulations significantly impact the N-glycosylation of biologics, which is critical for protein structure and bioactivities. In this case study, improvements in the culture medium by supplementing specific metal ion / small molecule components altered the N-glycan profile and enhanced its biological activity in-vivo.
We present a digital twin model-based process control (MPC) strategy for the successful glucose feeding in a bioreactor using only daily offline measurements. Our MPC strategy is composed of two kinds of formulars for predicting 1) glucose feeding amounts at daily sample time-points and 2) glucose feeding amounts at unsampled time-points.
Continuous manufacturing has become more prevalent across the biopharmaceutical industry in recent years. The ambr250ht perfusion system stands as a promising option for bench-scale culture work, but first must be proven as a replacement for the more common 3L scale. Through statistical analysis, the differences and similarities between these scales can be investigated to qualify their equivalence
Antibody drug conjugate therapeutics are an increasingly common modality due to their targeted ability to deliver various cytotoxic or immune-modulating payloads to specific cell types or tissues. This presentation covers a case study navigating the added complexity of selecting an appropriate mAb binder, payload, conjugation method, and drug-antibody ratio with emphasis on opportunities and challenges to accelerate pre-clinical development and IND filing.
This presentation will cover the points to consider for enabling multi-use systems in a Next Generation Biotechnology (NGB) facility. Multi-use systems improve operability while reducing cost and facility environmental impact. A framework for enabling multi use systems will be proposed including a review of batch definition, genealogies, hold times, product quality assessments, microbial control, compliance, validation, and regulatory considerations.
Generic Raman spectroscopy models, built using data from multiple processes, offer a method for streamlining Raman model development and integration in upstream bioprocessing. Effective implementation of generic Raman models requires consideration of several factors, such as the presence of any biases in the calibration dataset. The large volume of data used to develop generic Raman models also makes them ideal for creating hierarchical Raman models, which use the outputs of one or more base models as the inputs for a new model—e.g., cell culture pH.
The volumes of data generated by upstream bioprocesses during development, technology transfer and GMP production are only as good as the tools to make the data available for consumption and advanced data analytics. This talk will provide current highlights from our 12-year journey digitizing bench and pilot scale bioreactor processes to enable paperless data aggregation and generation of persona dashboards for daily analysis by data consumers and executives alike.
Disposable technology is being used more each year in the biotechnology industry. Disposable bioreactors allow the user to avoid expenses associated with cleaning, assembly and operations, as well as equipment validation. Disposable bioreactors have played a key role to meet the increasing run rate of the Cell Culture Pilot Plant while maintaining a high success rate, reducing labor costs, increasing efficiency, and lowering the risk of contamination. Recent effort to evaluate the next generation 10:1 turndown single use bioreactor with different cell retention devices to challenges oxygen mass transfer, carbon-dioxide stripping while assessing foaming, and vent filter sizing. This presentation will focus primarily on the advantage of the next generation high turndown single use bioreactor that comes with different sparger options (enhanced DHS and mircrosparger), and tubing for different cell retention device connection. Additionally, the high turndown provided a wide range of working volume allowing us to truly test the true scale down of the cell retention device for its maximum flux and filter throughput. The evaluation provided important system performance, operation experience, and cell culture performance data when comparing the next generation high turndown disposable system with respect to the legacy 2:1 and 5:1 turndown single use bioreactors, and conventional stainless-steel bioreactor system.
- Specific improvements in cell culture media formulations
- The impact of enhanced media on cell growth and productivity
- Challenges and potential solutions in adopting better cell culture media
- Balancing pH and osmolality for an optimal culture environment
- Can scale down models help with implementation?
- How are regulatory agencies approaching the rapid adoption of advanced technologies?
- What will the regulatory framework for the implementation of AI look like?
- How can regulatory agencies and industry work together to make safe and smart manufacturing a reality?
In the slowly evolving landscape of bioprocess development and manufacturing, digital bioprocess-twins have emerged as potential accelerators. While advanced algorithms are at the heart of this endeavor, they are just one piece of the puzzle. The talk delves into key discussion points that are integral to this paradigm shift. The foundation of accelerated process development and automated process control starts with a clever experimental design, in-time data accessibility combined with powerful modeling algorithms. The talk will highlight the advantages of using hybrid modeling, while emphasizing the other critical aspects of his journey. Several industrial relevant upstream showcases for microbial and mammalian cell lines will be highlighted. Thereby, concepts to save experimental effort by up to 70% will be elaborated, and the modeling structure created in the late-stage development will be reused for real-time monitoring and control in the later stages. Additionally, a downstream optimization showcase for UF/DF/SPTFF will be highlighted.
Continuous improvement methodology has been employed for many years to improve manufacturing where effectiveness depends on team experience. Advanced modeling technology for biomanufacturing promises to make continuous improvement methodology more widespread and allow teams to achieve expert results with far less experience. This presentation will illustrate model based continuous improvement across a drug substance biomanufacturing process using an industrial case study.
- How do we define AI/ML?
- Which industries have already implemented AI/ML? How can the Biopharma industry learn from this?
- Is Biopharma prepared for this transition?
- Early adopter’s vs late adopters: Will there be any impact?
- How can small, mid-size, and large companies strategize for ML/AI implementation?
- Can it be beneficial for all areas of biopharmaceutical development (discovery, development, clinical and manufacturing)
- Cost reduction and accelerated timelines for project
Dark Horse Consulting Representative
- Unpacking the current barriers to RNA gene therapy use
- Advancements in the processing of RNA to improve outcomes (remove unwanted cells, viral concentration, purification)
- Scalable processes
- Approaches to reaching high yield downstream purification
- Removing aggregates and contaminates from your titer
- Removal of partially-full capsids from your full-capsid yield
Many cell & gene therapy companies struggle to find the right balance between screening a number of novel viral vector construct designs and moving quickly through development. Often the transition from R&D to Process development (PD) can be bumpy and may result in delayed timelines or, even worse, an underperforming candidate that does not meet critical quality metrics. Building in key manufacturability and quality metrics early on in R&D helps define a successful candidate. This involves employing advanced analytics and characterization testing at an earlier stage in R&D, including cell-based functionality, to help detect any red flags that may not have been found until later in development. Additionally, aligning protocols across R&D and PD early on helps simplify the coordination and allows for more streamlined interpretation of the data. The combination of these aligned workflows result in improved characterization and allows for more viral vector designs to be evaluated in parallel. The ability to screen and characterize more sequence designs helps to understand the impact of different designs improvements such as codon optimization, promoter variants and other sequence elements. We will demonstrate lentiviral vector and AAV workflows with representative data to help highlight a streamlined workflow for accelerated development.
Ultragenyx is developing an investigational gene therapy, UX701, an adeno-associated viral vector-based gene therapy product, for the treatment of Wilson disease. UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene. Ultragenyx has initiated Cyprus2+, a seamless Phase 1/2/3 study of a single intravenous infusion of UX701 in Wilson disease (NCT04884815). UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™. This presentation reviews our first late-stage development activities for our upstream Pinnacle PCL™ platform process including parameter risk assessment, scale-down model qualification, and process characterization, to establish process controls for the upstream unit operations in our 2000L manufacturing process. The process characterization studies help us understand our parameter space and mitigate operational and quality risks which are critical for successful implementation of a robust commercial manufacturing process to provide consistent product quality throughout the product’s lifecycle.
Abstract TBC
Cell-based manufacturing of gene therapy molecules presents multiple challenges in maintaining high product yield and quality. Traditionally, isolating the desired DNA molecules is challenging. Additionally, cell-based approaches require long manufacturing timelines. To address these speed and quality challenges, we developed an enzymatic method capable of producing DNA drug substance at g/L scale and with >99% purity using chromatographic methods. This cell-free process has enabled highly accelerated production timelines compared to cell-based methods and the enzymatic platform facilitates diverse production application with constructs up to at least 7000bp in size.
- Scalable methods to optimize EV Isolation & Purification
- Efficient techniques for manipulating EV properties (size, surface ligands, cargo loading etc.) for enhanced targeting and in vivo delivery
- Challenges and potential strategies for adhering to strict GMP regulations for clinical-grade EV production.
- Leveraging AI to design personalized gene therapies based on individual patients' needs and target new, personalized delivery sites.
- Engineering multifunctional non-viral vehicles that combine gene delivery with imaging, additional therapeutic payloads, and controlled release capabilities.
- Utilizing AI to predict and optimize not just physicochemical properties, but also cellular interactions, biosensing, and response to specific triggers.
Mammalian cell bioprocesses have been monitored traditionally by off-line staining of cell samples. Newer techniques can offer rapid and continuous monitoring without manual sampling. Novel optical and dielectric protocols will be explained and discussed as alternatives for monitoring cells in bioprocesses. These methods can be used to provide growth profiles as well as the metabolic status of cell populations. They offer distinct advantages as in-line or at-line monitoring tools.
A team of BioPhorum members have developed and executed surveys focused on the activities and effort involved in a typical Cell Line Development (CLD) campaign. An average of 27 members from different companies that participate in the BioPhorum CLD working group answered surveys covering a standard CLD workflow from vector design to single cell clone selection. The surveys were very extensive, including a total of 341 questions split between antibody and complex molecule CLD processes. They provide a comprehensive industry perspective on the typical time and effort required to develop a CHO production cell line
Transposase/Transposon platforms have become increasingly common for the development of robust high-expressing CHO cell lines for protein therapeutic manufacturing. Notably, these techniques use a single transposase/transposon pair to enable such outcomes. ATUM, as part of the Leap In Transposase platform, has developed a number of mutually orthogonal transposase/transposon pairs that can be used to serially engineer CHO, and other, cell lines in a robust manner. Indeed, this engineering can be used to not only increase the expression of transgenes, as is the case for a mAb therapeutics, but also knock-down the expression of endogenous genes to affect cellular physiology and/or product quality attributes … or both. This talk will provide examples of such engineering including a case study wherein three orthogonal Leap In Transposase/Transposon systems were implemented for the creation of a mAb expressing cell line with specific product quality attributes.
In the biopharma industry, various techniques are utilized to enhance yield and quality of the target protein produced by stable cell pools and accelerate overall CLD timeline. In this presentation, we will show a case study of a method for minipool productivity enrichment via co-expression of the target protein with a fluorescent biosensor protein using an IRES, combined with state-of-the-art automation tools to allow productivity enhancement and reduce timeline for overall cell line development efforts.
Cell Line Development plays a crucial role in establishing Master Cell Banks for clinical and commercial biomanufacturing. This involves creating subclones and undergoing multiple stages of rigorous assessment, leading to the selection of a final clone used for the project's entire duration. Decision-making in this process hinges on extensive datasets obtained from advanced analytical methods. The introduction of high-throughput platforms like the Berkeley Light Beacon and automated micro-bioreactor systems has resulted in generating vast datasets, which often consist of thousands of data points in each experiment. Moreover, the need to integrate process and performance data from various scales, including deep-well plates, shake flasks, and bioreactor processes, is essential for a thorough analysis. Collectively, these factors pose significant challenges in data processing and analysis, which are critical for informed decisionmaking in Cell Line Development. Here, we propose a holistic method for digitizing the entire cell line development and selection process. Our approach begins with implementing laboratory and data automation tools to streamline the generation and handling of raw data. We then establish automated data pipelines using the Databricks platform, enabling the integration of various data types and data of different scales into a specially designed database. This database comprehensively encompasses data on cell line creation, assessment, and selection. Additionally, we develop visualization dashboards linked in real-time to the database, significantly reducing time spent on data processing. Finally, we leverage this streamlined data to build predictive models using open-source Python machine-learning algorithms, enhancing the cell line selection process. Our proposed digital framework ensures a data-driven approach, optimizing the selection of highquality cell lines for clinical and commercial manufacturing purposes.
Chinese hamster ovary (CHO) cells are the most widely used mammalian host for industrial-scale production of mAbs and other protein biologics. Selection of high-producing cell lines is a major endeavor in the process of manufacturing a novel biologic and requires an extensive and lengthy screening campaign of several hundreds of clonally-derived cell lines. We have previously reported the development of an efficient cumate-inducible expression system. Here, we present a new GMP-banked parental cell line, amenable to both constitutive or cumate-inducible expression. We first present our process for selecting CHO pools in suspension culture and then cell lines using a semi-automated approach, where imaging analysis provides >99% probability that selected cell lines are single-cell derived. Recent plasmid engineering efforts allowed to increase cell line productivity by 75% where~70% of selected clones show stable expression after at least 60 generations in culture. We also present recent development of a selection approach allowing to screen for more productive CHO minipools prior to single cell cloning. Optimizing several parameters such as cDNA sequence, signal peptide sequence, and suspension minipool selection process, we were able to increase titer for IgG1s from 2.5-3.0 g/L to 5.5 g/L using commercial medium and feed. Finally, we present data supporting the use of stable cumate-inducible CHO pools for clinical development of trimeric SARS-CoV-2 spike subunit vaccine antigens.
The Cell Line Development (CLD) group within the Genomic Medicine Unit CMC department at Sanofi is dedicated to the establishment of best-in-class Producer Cell Lines (PCLs) for manufacturing of life-changing gene therapy products. The development of a robust PCL requires optimization of a multitude of process and operating factors; however, additional functionality can also be unlocked through the implementation of an automation-enabled and data-driven workflow. We have implemented several enhancements in our PCL development process, including liquid handling automation and state-of-the-art automated cell imaging and seeding systems. This newly developed automation-enabled next-generation process will lead future high-throughput capabilities for PCL development.
Cell lines derived from the caterpillar, Spodoptera frugiperda (Sf), are the most commonly used hosts in the baculovirus-insect cell system (BICS), but they can harbor Sf-rhabdovirus (Sf-RV) contaminants. In this presentation, I will review our efforts to isolate the first Sf-RV negative (Sf-RVN™) Sf cell line, as well as the characteristics of this cell line indicating it is an improved, seamless alternative for Sf-RV-contaminated lines.
- Summarize key takeaways from discussions on accelerating CLD and FIH studies
- Encourage further exploration of innovative technologies and strategies for bioprocess development
- Highlight the importance of collaboration and shared learnings in advancing the field
The focus on accelerated development of new modalities for advanced therapies and challenges with complex drug candidates has driven adoption of novel concepts and technologies in biopharmaceutical development and manufacturing. The active drug substances have become more structurally complex and R&D timelines continue to be compressed, the technologies necessary to provide safe, robust and economical access to these molecules needs to keep pace. Emerging technologies such as continuous processing, automation, high throughput experimentation, predictive modeling in an integrated fashion can expedite process development and commercial manufacturing while ensuring efficiency and delivering quality products. All these aspects help to minimize the time and cost associated with development and manufacture of drugs and bring medicines to patients more effectively. This presentation will share AbbVie’s strategy and experiences in integrating such end-to-end technologies into process development and manufacturing facilities and discuss the challenges and opportunities associated with our approaches.
- Specific and strategic considerations for process and analytical development of advanced therapies
- Optimising and scaling-up of manufacturing operations post approval
- Preparing for regulatory filing
Given the complexity of biological products, effective control strategy and life cycle management are critical to delivering quality products with intended efficacy. This presentation will focus on an overview and guidelines to develop and implements analytical control strategy approach to streamline and harmonize quality control testing and method life cycle.
Control and management of materials is a key pillar of GMP manufacturing. An initiative is in progress which aims to harmonize the approaches of material management between clinical and commercial Drug Substance production sites to increase efficiency and supply chain sustainability within the organization. We will present the background and drivers, strategies, and current status of this initiative.
- An update on NIIMBL’s activities working towards key fundamentals
- Security of supply chains
- Flexibility of facilities to match varied and changing demand across portfolios of products
- Faster development with supply chains that can match this
- Sustainability for raw materials, components, and energy
- Addressing unique needs for vaccine modalities
- Optimization of PAT and tech transfer
- Scalability considerations
- Managing internal and external manufacturing operations
- The importance and advantages of microbial systems in biopharmaceuticals
- Platform for non-mAb biopharmaceutical development
- High Throughput process development
- CASPON technology a solution for the development of therapeutic peptides
- Future prospects, such as sustainability and competitiveness
Next generation manufacturing through implementation of advanced technologies is one of the current focus areas to enable agile, reliable, cost-effective, and timely production of biopharmaceuticals at highest quality standards. New modalities beyond standard mAB characteristics and complex supply chain logistics in a VUCA environment are factors to consider when designing the plant of the future and will impact the competitive advantage through application of advanced technology concepts. The talk will provide examples of deployment in context with business needs and added value.
Shifting from batch to continuous manufacturing is a promising way of lowering manufacturing costs of by allowing operations to run simultaneously with reduced downtime, higher productivity, and at several-fold smaller scale. This is particularly promising for gene therapy products using recombinant adeno-associated viral vectors (AAV), as treatments currently cost up to USD 3.5 million per patient and have high cost-of-goods ranging up to USD 1 million per dose. In this talk, we present approaches for intensification of downstream AAV manufacturing processes using principles of continuous processing.
The biopharmaceutical industry is increasingly confronted with the dual challenges of complex product demands and a volatile market landscape, necessitating unprecedented levels of supply chain agility and resilience. This presentation addresses the critical role of digital innovation in transforming biopharma supply chains, enabling them to adapt swiftly to market fluctuations and unexpected disruptions. We explore the integration of advanced technologies such as artificial intelligence (AI), and machine learning (ML) to enhance visibility, optimize operations, and improve decision-making processes. Through case studies and industry insights, we demonstrate how digital tools can facilitate precise inventory management, robust cold chain logistics, and regulatory compliance, thereby reducing waste and ensuring timely delivery of vital products.
- The development of first in class novel complexed miRNA-based cellular reprogramming therapeutics
- Exploring the use of miRNA for the treatment of Alzheimer’s Disease, Diabetes and Heart Disease
Aurion Biotech is taking their off-the-shelf, allogeneic cell therapy (AURN001) from the lab to clinical trials with the goal of becoming the first mass-scale cell therapy to help restore vision to 17M+ patients around the world with corneal endothelial disease. Building a mass scale cell therapy means:
- Fulfilling a significant unmet patient need caused by the global shortage of donor corneas for transplant, in ways that are accessible and equitable
- Navigating the highs and lows of manufacturing and scaling an allogeneic cell therapy for clinical trials, successfully managing a CDMO partner and establishing a development process that can be translated into a large-scale commercial product.
- Creating a breakthrough product that will transform the physician experience – one that is less complex and less invasive than corneal transplant (the current standard of care) and minimally invasive for patients.
- How can early-stage/research professionals best prepare for GMP
- Critical aspects to keep in mind transitioning from early-stage development into more commercial manufacturing.
- Examples from experiences and lessons learnt
- Cross-team collaboration and pre-emptively preventing down the road bottlenecks
This presentation will focus on the technical and manufacturing capabilities for cell therapy commercialization with discussion of CMC requirements, manufacturing launch readiness, and supply considerations. Specific considerations for a cell therapy and partnership with contract manufacturing organizations will also be discussed including lessons learned.
IMPT-314 and IMPT-514 are autologous CAR T products that use the same CD19/CD20 bispecific tandem CAR, and the same cell manufacturing process selecting for desirable naïve/memory T cells. The process was developed by Professor Yvonne Chen at UCLA and was successfully transferred to ImmPACT Bio, a clinical-stage biotech company.
IMPT-314 is being studied in a Phase 1/2 clinical trial in aggressive B cell lymphoma. IMPT-514 is being studied in a Phase 1/2 clinical trial in lupus nephritis and systemic lupus erythematosus. Both trials are actively enrolling patients.
While the efficacy and durability of autologous CAR T therapies remain unparalleled, a key challenge for scalability and continued adoption is the high cost of manufacturing. Constrained by single patient batch manufacturing, resource-intensive manual processes with open steps, these therapies require highly skilled direct labor, expensive GMP cleanroom infrastructure and controls, with limited opportunities for scale-up.
We present a case study of transition from an established GMP manual process to an automated, closed-processing manufacturing platform, and address the following topics:
- Selection of automated platforms
- Process development and product comparability,
- Converting GMP operations to automated & paperless systems,
- Benefits and challenges of automation,
- Aligning manufacturing process changes with clinical development phases.
- Reducing wait times and logistical magnitude to reduce overhead costs
- Increasing accessibility, distributing to multiple locations closer to patient populations and remove transportation cost barriers
- How QC release testing and material kitting and management will be handled for POC manufacturing in this ecosystem.
- Establishing harmonized regulatory standards across different regions
- Implementing robust oversight mechanisms to ensure compliance with regulatory requirements and maintain product in a distributed manufacturing model.
- EMA vs FDA approach
This presentation delves into the evolving landscape of supply chain management for CGTs, offering insights into the latest developments that are reshaping the industry. The session begins with an overview of traditional supply chain practices, highlighting their limitations and the pressing need for adaptation in an ever-changing global market. From there, it transitions into an exploration of cutting-edge innovations such as artificial intelligence (AI), machine learning, blockchain technology, and Internet of Things (IoT). These technologies are revolutionizing supply chain processes, enhancing efficiency, visibility, and responsiveness across the entire network. We will also consider predictive analytics to digital twins and autonomous systems, which can empower organizations to anticipate disruptions, optimize inventory management, and streamline operations for maximum effectiveness. In addition, we will consider sustainability initiatives, circular economy practices, agile supply chains, and the integration of advanced robotics and automation. By offering a comprehensive overview of these innovations, tools, and trends, attendees will be equipped with the knowledge and insights necessary to navigate the complexities of modern supply chain management.
- Navigating diverse region-specific frameworks to expedite approvals.
- Build strategic partnerships with local distributors to facilitate market entry.
- Tailor marketing and patient education strategies
- Collaborating with local health services/distributors in preparation for product launch
- Ensuring preparedness for your supply/logistics chain across goal market
- E.g. Cryopreservation facility/availability when transporting
*This event requires a separate ticket which can be purchased at a discount during your conference registration*
The BWB Awards @ Biotech Week Boston brings together the faces and names that make the Boston community the beating heart of the biotech world.
This evening celebratory gala features a night of networking, food, drink, entertainment and recognition on September 23rd, 2024 in Boston. We're excited to roll out the red carpet and honor the top individuals, teams, and organizations that make the life sciences ecosystem vibrant and innovative.
Full details on the BWB Awards 2024 can be found at https://informaconnect.com/bwb-awards/
- Case study
- Working with components with short half life's
- Sourcing and ensuring traceability for radioactive supply
- Preparing a secure and reliable network of suppliers by analysing the vulnerabilities of complex bioprocessing supply chains
- Best practices for successful collaboration and transparency with stakeholders/vendors
- Strategies to mitigate risk and ensure reliability.
- Utilizing AI-aided design to anticipate potential manufacturing challenges
- Implementing AI into pre-existing bioprocessing workflows
- Case study examples
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
Objective: This comprehensive tutorial will provide an overview of the regulatory and quality principles that guide the analytical studies for all biological products, with emphasis on the specific elements applicable to complex MODALITIES such as gene and cell therapy. Emerging best practices in analytical methods for characterization, comparability, release and stability testing of gene and cell therapy will be presented. The rationale behind the requirements, with supporting references, will be provided. Attendees to this class will receive electronic copies of all reference guidances and publications discussed in the class.
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am - 4pm.
Through a series of presentations, case studies and interactive discussions and exercises this course will focus on CDMO oversight- selection, negotiation, tech transfer and life cycle management specifically for cell and gene therapy products.
Some of the topics to be discussed include:
CDMO Selection: Models and Process
Contract and Quality Agreement Negotiation
Tech Transfer: Best practices and risk management
Quality Assurance and Quality Control
On- Going Manufacturing: Organisational models and interactions
Life Cycle Management
Relationship Management
Who should attend?
Process Development Scientists/Engineers
Business Development Managers
Project Managers
Regulatory Affairs Professionals
Quality Assurance/Quality Control Managers
Manufacturing Operations Managers
Supply Chain Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
What is this track about?
This is a full day course from 9am – 4pm
- Introduction to Tech Transfer
- Definition of TT
- Tech transfer: a Project with Challenges
- Session 1: Formal Aspects including Regulatory concerns ; product comparability, Tech Transfer Procedure, Protocol & Report
- Session 2: Tools for Tech Transfer including Transfer of Information, Dealing with Scale up and changes, Analytical Transfer
- Session 3 : Complexities of Tech Transfer including Complexity and Risk Factors, The importance of communication, Optimising the transfer programme
- Session 4: Workshop Case Study and Group Activity
Who Should Attend?
Process Development Scientists/Engineers
Manufacturing Operations Managers
Quality Assurance/Quality Control Managers
Regulatory Affairs Professionals
Project Managers
Supply Chain Managers
Analytical Development Scientists/Engineers
Validation Specialists
Operations and Production Personnel
Research and Development Scientists
Business Development Managers
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
This workshop will explore the current initiatives, innovative strategies and new technologies designed to drive sustainability of biologics process development and manufacturing.
- Define the Current State of Sustainability in 2024
- Case Studies on Current Initiatives from End Users and Vendors
- Help Individuals develop sustainability strategies and solutions best suited for their companies
- Interactive Discussions on Challenges with Single Use and Environmental and Economic Modeling
- A Look Ahead - What’s Next and What’s Needed?
- Approaches to reaching high yield downstream purification
- Removing aggregates and contaminates from your titer
- Removal of partially-full capsids from your full-capsid yield
Increasing demand for recombinant adeno-associated virus (rAAV) based gene therapies necessitates increased manufacturing production. Transient transfection of mammalian cells remains the most commonly used method to produce clinical-grade rAAVs due to its ease of implementation. However, transient transfection processes are often characterized by sub-optimal yields and low fractions of filled-to-total capsids, both of which contribute to the high cost of goods of many rAAV-based gene therapies. Analysis of our previously developed mechanistic model for rAAV2/5 production suggested that the inadequate capsid filling is due to a temporal misalignment between viral DNA replication and capsid synthesis within the cells and the repression of later phase capsid formation by Rep proteins. We experimentally validated this prediction and showed that performing multiple, time-separated doses of plasmid increases the production of rAAV. In this study, we utilize the insights generated by our mechanistic model to develop an intensified process for rAAV production that combines continuous perfusion, high cell density transfection, and re-transfection which resulted in increased titer and plasmid efficiency. Our results establish a new paradigm for continuously manufacturing rAAV via transient transfection that improves productivity and reduces manufacturing costs.
AAV has emerged as a significant therapeutic modality in gene therapy. Challenges such as poor yield and variable product quality persist in the viral vector manufacturing space and we have addressed these problems using our cell engineering technology. Our cell engineering platform for improved adeno-associated virus (AAV) manufacturing addresses the critical challenges in gene therapy manufacturing, and presents an innovative modality for improving cells for production of viral vector therapeutics. Our platform, yielding cells with improved AAV production and CQAs, can significantly bolster the efficiency and cost-effectiveness of gene therapy manufacturing, and can accelerate current development timelines. We used a directed-evolution approach based on repeated cell fusions to shuffle the cell genome, and amplify chromosomes of HEK-293 host cells. Engineered clones enriched for mitochondria phenotypes were isolated, then used as transient-transfection hosts, and for creating stable packaging and producer cell lines. For generation of stable packaging and producer cell lines, we developed a novel inducible system that maximizes the capabilities of the inherent viral production machinery. Engineered HEK-293 clones grown in suspension culture exhibited up to 15-fold productivity improvement via triple transient transfection for AAV1, AAV2, AAV5, and AAV9 serotypes with capsid titers as high as 1017 viral particles/L (vp/L)—at least 10-fold higher than current industrial manufacturing processes. Selection for certain mitochondria phenotypes resulted in a 2-fold improvement in full-to-empty ratio—up to 55% full in crude supernatants. Finally, our engineered stable packaging and producer cell lines achieved capsid titers of up to 1016 vp/L. We demonstrated a multi-modal cell-engineering platform that has significantly improved yield and manufacturability for transient transfection and for stable packaging and producer cell line methods. We further propose a model regarding the role of mitochondria for enhancing capsid percent-full. Taken together, our disruptive platform technologies provide solutions for meeting current—and future—gene therapy manufacturing challenges.
- Translating exciting novel vectors into scale – overcoming the production challenges hindering advancement of vector therapies
- Deep dive into complex purification processes and potency issues
- Balancing efficacy, safety, and manufacturability in vector design
- Selecting the appropriate vector platform (adenovirus, lentivirus, AAV) based on therapeutic application.
- Incorporating novel vector design elements for enhanced targeting and gene delivery
- Utilizing CRISPR/Cas9 technology to facilitate gene editing and therapeutic development.
- What are the key criteria for choosing a cell or gene therapy manufacturing partner? (Expertise, capacity, regulatory knowledge, technology fit)
- Approaching start-up build from the ground up to develop new therapeutics
- Ensuring preparedness for CDMOs
- How can different stakeholders identify complementary strengths to build mutually beneficial partnerships?
- Best practices to ensure quality control and compliance throughout manufacturing.
- Building trust and transparency in long-term CGT manufacturing partnerships
- Tailored design and engineering of polymeric nanoparticles for optimal cell interaction, targeting, and therapeutic delivery.
- Diverse delivery strategies and tissue targeting approaches for specific disease applications
- Effective encapsulation techniques to preserve cell viability, functionality, and differentiation potential during delivery
- Case study: how are these being explored/used
- Emergence of EVs as a promising non-viral delivery vehicle
- Exploring the potential of using engineered EVs with controlled release mechanisms for sustained therapeutic delivery
- Advancements in EV isolation, modification, and loading techniques
- Challenges regarding therapeutic EV manufacturing and developing clinical-grade EVs
- Scalable methods to optimize EV Isolation & Purification
- Efficient techniques for manipulating EV properties (size, surface ligands, cargo loading etc.) for enhanced targeting and in vivo delivery
- Challenges and potential strategies for adhering to strict GMP regulations for clinical-grade EV production.
In the last 3 decades, diverse research shreds of evidence suggested that Mesenchymal stem cells (MSCs) can regulate the immune niche through the secretion of various cytokines, showing promise in treating immune disorders. However, conventional MSCs face limitations such as limitations in large-scale manufacturing and constraints on donor diversity and necessitate overcoming these challenges innovation in gene delivery and production technologies.AffyXell Therapeutics is developing next-generation cell & gene therapies (CGT) for immune diseases by overcoming these limitations with the AffyXell platform technology (AFX Platform). This novel approach utilizes the Affimer® (non-IgG scaffold) protein gene introduced into allogeneic pluripotent stem cell-derived MSCs to synergistically enhance their immune regulatory functions. In addition, the established cell bank system of genetically engineered MSCs enables the production of consistent quality, and the ease of clinical application and global logistics are possible through cryopreservation technology.In the development of the first pipeline, AFX001, introducing the hCD40L antagonist Affimer® gene into MSCs, demonstrated an excellent binding affinity and effective inhibition of the activity of hCD40L. This resulted in the suppression of T cell and B cell activation both in vitro and in vivo, as evidenced by improved clinical scores and histological improvements in a xenograft GVHD mouse model.This AFX platform technology represents a new direction for optimized next-generation CGT for immune diseases overcoming the limitations of conventional MSC therapies through innovation in genetic engineering and production technologies.
- Overcoming hurdles of viral vectors for Gene Therapy by harnessing the power of non-viral DNA-based platforms for Advanced Therapeutics
- Advancing Beyond Lipid Nanoparticles: Innovative Strategies in Delivery Vehicles Technology
- Exploring the use of DNA-based therapeutics for the treatment of diabetes, anemia and cancer in animal and human health.
- Case Reports
Since FDA's and EMA's market approval of the first oncolytic viral (OV) therapy in 2015, immuno-virotherapies have developed with increasing importance and unprecedented speed. More than 50 clinical OV studies have already been completed, more than 100 are currently ongoing in different clinical phases, either as monotherapy or in combination with other therapies.
To enable these new immuno-virotherapies, numerous virus species from different families, widely varying in type, shape, size, size distribution, particle-to-plaque ratio, etc. entered the virus manufacturing facilities over the last years. Many new facilities were built to fully cope with the specific requirements of these new therapeutic entities.
In contrast to many other viral vaccines, virus-based immuno-therapeutic products are replicating biological entities produced, stabilized, and stored at titers which can easily exceed those of vaccines by several orders of magnitude. Since administration of immuno-therapeutic viruses is either intratumoral, or - to an increasing extent in new applications – intravenous, these viruses represent parenteral products with high specific requirements regarding safety. In contrast to other protein-based parenteral products, immuno-therapeutic viruses are particles that need adequate manufacturing technologies in both upstream (virus amplification and clarification) and downstream (purification, concentration and formulation) processing able to cope with the requirements for manufacture and stable storage of actively replicating virus particles at high titers without product loss due to aggregation, degradation or loss of infectivity.
ABX-001 is a lymphocytic choriomeningitis virus (LCMV)-derived non-oncolytic arenavirus which is currently in preclinical development to treat cancer. In contrast to oncolytic viruses, infection with LCMV does not kill host cells by direct lysis but results in a strong combined innate and adaptive immune response directed against the tumor.
A clinical manufacturing process has been developed for ABX-001 from lab-scale to clinical scale in a BSL-2 facility using fully disposable manufacturing concepts for upstream and downstream operations. The virus is amplified by infection of a human host cell line, expanded from a GMP Master Cell Bank in suspension culture, with virus from a GMP Master Virus Seed in a stirred single-use bioreactor (SUB) operated in batch mode. For subsequent downstream processing, two substantial single-use technologies are applied: (a) hollow fiber-based tangential flow filtration is used for initial clarification of virus at full recovery, and for final formulation by ultra-diafiltration, and (b) monolithic chromatography is used in a two-stage chromatography process for efficient removal of process-related impurities (polish) followed by a fast capture chromatography concentrating the enveloped >200 nm virus particles. The purified and concentrated virus (>10^11 particles/mL) is further formulated in a buffer that allows for long-term liquid frozen storage without appearance of visible particles or any significant increase of subvisible particles, even when thawing after months of storage. Challenges during process development and scale-up have been manifold and will be discussed together with the solutions we applied to implement a safe clinical manufacturing process for this promising immuno-virotherapy.
- Navigating the complex grey area of manufacturing for Phase 1 and 2 trials
- Keeping efficiency while complying with limited guidance
- Need conversation between regulators and developers
- Lessons learnt and case studies from companies moving between clinical phases
- Therapeutic Developer/CDMO dynamics in the phase 1 and beyond
- How to best leverage CDMO expertise and capital efficiency
This presentation will provide insight into the bit.bio team's recent work to scale-up from 2-D adherent culture to 3-D suspension culture of iPSC-derived cell therapies and commercial research products. The unique characteristics of bit.bio's foundational opti-ox technology allow for precise control of homogenous 3-D suspension cultures.
Automation plays a key role in improving the quality and lowering the cost of cell therapy products. As new or improved processing and testing technologies enter the market, it’s important for firms that build these products to design and provide both digital and physical integration touchpoints for their systems and consumables. This overview will discuss some of these important design considerations that will allow for more effective implementation of automation and make it easier to adopt new technologies.
Allogeneic cell therapies present a unique challenge for drug product process development by combining aspects of autologous cell therapies and conventional biologic products. As production scale increases to that of a commercial, off-the-shelf therapy, new challenges emerge for expanding fill finish and cryopreservation capacity. This presentation will discuss the developments and considerations that are necessary for pursuing large scale, allogeneic cell therapy manufacturing.
Many pharma and biotech companies onboarded thousands of materials and increased inventory during the pandemic. With the endemic in full swing, organizations have a surplus of material that is about to or has already expired. This presentation seeks to explain how the presenter worked to extend the shelf life of various materials for continuity improvement and financial savings.
*This event requires a separate ticket which can be purchased at a discount during your conference registration*
The BWB Awards @ Biotech Week Boston brings together the faces and names that make the Boston community the beating heart of the biotech world.
This evening celebratory gala features a night of networking, food, drink, entertainment and recognition on September 23rd, 2024 in Boston. We're excited to roll out the red carpet and honor the top individuals, teams, and organizations that make the life sciences ecosystem vibrant and innovative.
Full details on the BWB Awards 2024 can be found at https://informaconnect.com/bwb-awards/