Tuesday, September 22, 2020

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

1:00 - 1:10 Praetego Inc

1:20- 1:30 Peri-Nuc Labs

1:30 - 1:40 Adamas Nanotechnologies, Inc.

• Introduction
• Objectives and outline of course
• Main development stages and regulatory cadre
• The main streams of activity from cell line to drug product
• The CTD for Regulatory filings. Quality Module – overview of main sections
• The importance of CMC : Quality (and Safety and Efficacy)
• Supply
• The pillars of pharmaceutical development
• Criticality Assessment
• Control strategy & Validation
• QBD Principles
• Most relevant regulatory Guidance and where to find it MH

Quiz

• Review of the main streams and initial status
• Broad requirements for Tox and for First in Human Studies
• Cell banking
• Other Raw materials
• Drug Substance Process and Manufacture
• Minimum requirements
• Understanding your process :
• Impurities : identity, clearance, control
• First steps towards a control strategy
• Adventitious contamination and Viral Clearance Studies
• Drug formulation and Drug Product Processing
• Analytical package
• Release methods definition and development
• From method performance to method validation
• In Process Controls (else cover under process?)
• Batch data in the submission
• Product Characterisation and Reference standard
• Stability ( DS and DP)
• Forced degradation studies : necessity and importance
• Why is stability important ?
• Different type of stability studies and typical package for PhI
• Shelf life assignment

• Review of the main streams and initial status
• Broad requirements for Tox and for First in Human Studies
• Cell banking
• Other Raw materials
• Drug Substance Process and Manufacture
• Minimum requirements
• Understanding your process :
• Impurities : identity, clearance, control
• First steps towards a control strategy
• Adventitious contamination and Viral Clearance Studies
• Drug formulation and Drug Product Processing
• Analytical package
• Release methods definition and development
• From method performance to method validation
• In Process Controls (else cover under process?)
• Batch data in the submission
• Product Characterisation and Reference standard
• Stability ( DS and DP)
• Forced degradation studies : necessity and importance
• Why is stability important ?
• Different type of stability studies and typical package for PhI
• Shelf life assignment

• Anticipating the needs on the work streams
• Process Understanding and Design
• Technology Transfer
• Dealing with changes and Comparability

• Anticipating the needs on the work streams
• Process Understanding and Design
• Technology Transfer
• Dealing with changes and Comparability

Intensified and continuous bioprocessing place fundamentally different demands on production cell lines than traditional fed-batch manufacturing. High-density perfusion cultures require cell lines engineered for long-term stability, robustness to shear and nutrient gradients, and controlled growth at extreme cell concentrations, rather than short-term peak productivity alone. This presentation will explore what defines a “perfusion-ready” cell line, highlighting engineering strategies to improve metabolic efficiency, reduce waste accumulation, and sustain consistent product quality over extended culture durations. The interplay between cell line behavior and cell retention technologies such as ATF and TFF will be discussed, along with the implications for process robustness and scalability. Finally, the talk will address how cell line development workflows must evolve for continuous manufacturing, emphasizing early exposure to perfusion conditions and selection criteria aligned with long-term intensified process performance.

• What are the scale up risks: Cell retention, fouling, oxygen transfer, media logistics, sampling/sterility strategy.
• Practical approaches to managing large media volumes/ logistics in continuous operations.
• How do ATF and TFF systems improve cell retention and productivity

• Exploring the potential for process intensification through continuous membrane steps.
• How can continuous processes be scaled effectively for GMP manufacturing?
• What are the risks and limitations of adopting continuous chromatography?

TBC

• Online biomass, metabolites, product titre
• Inline chromatography analytics
• Control strategies for steady-state operations
• Data integrity and batch definition in continuous processes

We demonstrate the first end-to-end concept downstream continuous processing train for AAV vectors at pilot 50 L scale, addressing key bottlenecks in traditional batch processing. The process utilizes novel enabling technologies for each unit operation, including multi-column affinity capture with fast-flow loading, dual-tank low pH viral-inactivation, rapid-cycling anion exchange chromatography using weak partitioning AEX for full-empty separations, and countercurrent hollow fiber filtration for single-pass UFDF. The continuous train was run for 72 hours of continuous operation and reduced resin volumes by >90% while matching or exceeding batch process CQAs. The continuous purification process as demonstrated can be linearly scaled up from 50 L to be compatible with 500-2,000 L scale staggered batch harvests as well as upstream perfusion systems.

TBC

An honest assessment of continuous processing and integrated processes – what works, what doesn’t and what CDMOs must consider before investing.

• Realistic cost-benefit analysis: footprint, CapEx, development time
• Is an end-to-end continuous process feasible for a CDMO customer mix

With pressure to reduce COGs and time-to-market, process intensification has re-emerged as a strategic priority.

• Practical strategies to intensify processes without increasing risk
• Understanding where intensification truly delivers value
• Insight into future manufacturing models for biologics

• What problems AI is actually good at solving?
• Pattern recognition versus mechanistic understanding
• Why AI does not replace process understanding
• The three types of AI: Descriptive, Predictive, Prescriptive – Where does bioprocessing mostly sit today?
• What AI can realistically deliver in the next 12-24 months? (where it is working, where it is still experimental, where expectations need resetting)

TBC

• What AI needs to work – data volume versus relevance, why garbage in is dangerous?, Why AI magnifies process misunderstandings?
• How to diagnose data readiness
• What “good enough” looks like
• A practical checklist for AI readiness
• How to spot red flags in your own datasets
• When not to start an AI project

• Why pure AI fails in bioprocessing?
• What mechanistic modelling actually is?
• Where mechanistic models outperform AI?

• Combining first-principles understanding with machine learning
• How mechanistic and AI models are combined in practice?
• What hybrid architectures look like?

• Maintaining, retraining and governing ML models in GMP environments.
• What happens after the model is deployed in a real GMP environment?
• What drift actually looks like in QC and analytical models, PAT and process models, predictive stability models? Early signs? How often does drift occur?
• What happens when a model is wrong once?
• Why users stop trusting models?
• Who owns the model after deployment?
  
  

TBC

• If you can’t explain it, you can’t use it.
• Making AI transparent for quality, manufacturing and regulators.
• How to document models
• Managing model updates
• When retraining becomes re-validation
• When to freeze a model versus evolve it?

• What surprised you most after deploying AI or models?
• Where should companies stop experimenting and start standardizing?
• What decisions are we still not ready to trust to models?
• What gaps are holding teams back more than technology?

• Introduction to Biopharmaceutical Life Cycle.
• Explain what upstream bioprocessing involves: the early stages of production, including cell culture and fermentation.
• Outline the key objectives: generating the desired biological product through cell growth and expression.
• Discuss the selection of cell lines (e.g., CHO cells, microbial cells).
• Introduce bioreactors and their role in providing a controlled environment for cell growth.
• Discuss different types of bioreactors (e.g., stirred-tank, wave, single-use) and their applications.
• Explain the fermentation process and its parameters (e.g., pH, temperature, oxygen levels).
• Explain the importance of culture media in supporting cell growth and productivity.
• Describe the process of scaling up from lab-scale to commercial-scale production.
• Highlight current trends in upstream bioprocessing (e.g., single-use technologies, continuous processing).
• Discuss future directions and innovations in the field.

• Explain what downstream bioprocessing involves: the purification and formulation of the biological product after cell culture and fermentation.
• Outline the key objectives: ensuring product purity, quality, and stability.
• Describe the process of harvesting cells or extracellular products from the bioreactor.
• Explain the methods used for cell separation (e.g., centrifugation, filtration).
• Introduce the main purification methods: chromatography, filtration, and precipitation.
• Describe different types of chromatography (e.g., affinity, ion-exchange, size-exclusion) and their applications.
• Explain the principles and applications of ultrafiltration and diafiltration.

• Growing importance of digitalization, AI, and machine learning in the biopharma industry.
• Key pillars of digital transformation in biopharma.

Key Areas of Digitalization

• Data Management and Integration (from Development to Manufacturing).
• Automation and Robotics in bioprocess workflows.
• Real-time Monitoring and Advanced Analytics for process optimization

Applications in Bioprocessing

• Use of digital twins and AI to optimize upstream and downstream unit operations.
• Role of ML/AI-driven tools for Advanced Therapy Medicinal Products (ATMP) manufacturing.AI-driven real-time monitoring, predictive maintenance, and anomaly detection in production lines.
• AI-driven real-time monitoring, predictive maintenance, and anomaly detection in production lines.
• Simulation-based process development for rapid scale-up.

Challenges and Considerations

• Overcoming data silos and ensuring system interoperability.
• Addressing regulatory requirements for AI and digital tools in GMP environments.
• Ensuring data quality, integrity, and security in digitalized workflows.
• Bridging talent gaps and fostering a digitally skilled workforce.

Case Studies

• Real-world examples of digital transformation in bioprocessing.
• Lessons learned from integrating AI-driven tools in ATMP production.

Future Trends and Directions

• Adoption of Industry 4.0 principles in biopharma manufacturing.Emerging technologies such as edge computing and IoT for bioprocessing.
• Emerging technologies such as edge computing and IoT for bioprocessing.
• Sustainability and digitalization: How to?

• Understanding emerging therapies: distinctions between cell therapy, gene therapy, etc.
  • Therapeutic potential and current clinical landscape of different emerging therapies, unique challenges and opportunities presented.
  • Differences and similarities from ‘traditional’ biologics:
  • What learnings can we take from traditional modalities to approach novel modalities?
• Understanding the Cell Therapy and Gene Therapy manufacturing processes.
  • Best practices when entering/transitioning into the advanced therapy industry.
  • Leveraging experiences from your background into industry.
• Strategies and approaches to best utilise available technologies in the development & production of emerging therapies.
  • Moving and translating research from academia, to start up, industry, and beyond.
  • Understanding the difference between these, how to transition, pros and cons.

Lessons and experiences from our panellists.

• The evolution of biopharmaceutical modality
• Analytical methods and their purpose in biopharmaceutical development and manufacturing
• Analytical method development and validation
• Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
• Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
• Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
• Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
• Role of quality control (QC) and quality assurance (QA) in biopharma
• Case studies and industry examples
• Latest and future advancements in analytical methods and quality assurance
  
  

• The evolution of biopharmaceutical modality
• Analytical methods and their purpose in biopharmaceutical development and manufacturing
• Analytical method development and validation
• Product physicochemical characterization - high-performance liquid chromatography (HPLC), capillary electrophoresis (CE), spectroscopy, imaging, and post-translational modification (PTM)
• Product biological assays - cell-based assays (CBA), enzyme-linked immunosorbent assays (ELISA), and potency assays
• Microbiological contaminants - sterility testing, endotoxin testing, and microbial limits testing
• Process impurity testing - host cell DNA, host cell proteins, chromatography ligand
• Role of quality control (QC) and quality assurance (QA) in biopharma
• Case studies and industry examples
• Latest and future advancements in analytical methods and quality assurance

Final panel with all presenters for one last Q & A opportunity!

Explore the latest innovations in bioprocessing while networking with industry experts, solution providers, and fellow attendees. Enjoy complimentary snacks and refreshments as you browse cutting-edge technologies, discover new solutions, and connect with exhibitors who are shaping the future of the industry. Don’t miss this opportunity to start your conference journey with great conversations and valuable insights in a dynamic setting!

Join us for an invigorating start to your day with our special wellness combo event!

Fun Run

Lace up your running shoes and hit the trail with colleagues for our energizing Fun Run. Whether you're a seasoned runner or prefer to walk, this inclusive event welcomes participants of all fitness levels. Enjoy the camaraderie, fresh air, and endorphin boost as we move together through scenic surroundings.

Morning Yoga Session

Happening simultaneously, our rejuvenating yoga session offers a peaceful alternative. Our experienced instructor will guide you through gentle poses designed to increase flexibility, reduce stress, and center your mind for the day ahead.

How To Sign Up: Please ask at registration onsite
What to Bring: Comfortable athletic wear, water bottle, sunscreen

Through a series of presentations, case studies, interactive discussion and exercises this workshop will delve deeply into the underlying science of developing potency assays.

Some of the topics to be discussed include:

• Overview of what potency is, and what it is not
• Considerations when qualifying and validating bioassays
• From initial ideas, through characterisation to the final potency method
• Clinical correlation
• Regulator objections and why
• Examples of potency assays for approved products
• Examples of a cell and gene therapy potency assays for various product types

Some of the topics to be discussed include:

• Overview of what potency is, and what it is not
• Considerations when qualifying and validating bioassays
• From initial ideas, through characterisation to the final potency method
• Clinical correlation
• Regulator objections and why
• Examples of potency assays for approved products
• Examples of a cell and gene therapy potency assays for various product types

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 for external tech transfers
• Knowledge Management and Digital Transformation in Tech Transfer
• Quality Assurance and Quality Control
• Life Cycle & Relationship Management

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 for external tech transfers
• Knowledge Management and Digital Transformation in Tech Transfer
• Quality Assurance and Quality Control
• Life Cycle & Relationship Management

Comprehensive vein-to-vein data analyses can yield profound insights into biomarkers predictive of clinical outcomes, CMAs and CDAs predictive of manufacturability, and CPPs to enable adaptive manufacturing. However, statistically meaningful analyses require large data sets atypical for innovative modalities. This presents a unique opportunity to accelerate progress in cell therapies with a sector-wide precompetitive collaboration that maintains participants’ data confidentiality.

• What to start early in the process?
• How early is too early to consider strategy for scaling up?
• How to define the right commercial scale early in development
• Best practices for locking in scalable processes from the start
• Designing therapies with long-term success in mind—beyond Phase 1
• Key considerations for commercial scale up, reimbursement, raw material availability, and regulatory approvals
• Pathways to developing safe, reimbursable therapies with broad patient access

Delivery of plasmid DNA enables systemic expression of therapeutic molecules, including but not limited to monoclonal antibodies, proteins and peptides. However conventional delivery techniques are limited by constraints of redosability and toxicity in their ability to delivery DNA effectively. Polymeric systems can overcome these constraints but have a very large design space. This presentation will describe how machine learning can leverage large design spaces for the design of polymeric delivery vehicles for a broad range of therapeutically relevant molecules in vivo.

Engineered viral-like particles (eVLPs) are a promising delivery technology for genetic medicines addressing hematologic malignancies but lack natural tropism to HSPCs in vivo. Here, we have engineered eVLPs with next-generation viral fusogens that enable in vivo editing of up to 68% of human HSPCs in a humanized mouse model with minimal editing of human liver.

LNPs with multiple payloads are very sheer sensitive, therefore the TFF purification should be replaced with CIM monolith column chromatography deprived of shear forces.

· LNPs with multiple payloads are very heterogenous, chromatography approach is required to purify the LNP product.

· To enable the batch-to-batch product manufacturing consistency new analytical method to characterise the pDNA, mRNA and LNP should be used.

KRAS mutations drive pancreatic cancer, an indication with high unmet medical need. Anocca's platform has discovered, validated, and developed novel TCR-T products targeting mutant KRAS pHLAs that show high potency, specificity, and effective cell killing preclinically. The first product in this product library, targeting mutKRAS G12V, has entered clinical development as part of our Phase I/II VIDAR-1 trial in advanced pancreatic cancer, and is the first non-viral edited TCR-T approval in Europe.

Onechain Immunotherapeutics developed a stromal-free bioprocess for the novo generation of γδ T cells from cord blood (CB) CD34+ HSPC or iPSC-derived CD34+, using recombinant Notch ligands, eliminating the need for expanding these cells from peripheral blood or other sources. The process can yield functional γδ T cells that can be armed with CARs with cytotoxic activity against multiple cancer cells. This strategy is highly scalable, consistent with GMP guidelines, and represents a step forward in the field of allogeneic, off-the-shelf CAR-T cell therapies.

To date, multiple in vivo gene editing clinical trials have demonstrated significant benefits to patients due to the “one and done” nature of these therapies. However, all of this work has been done in the liver using LNPs. Arbor’s technology is very small and enables us to move beyond the liver to target other regions of the body, in particular, the CNS.

The rise of advanced therapies has led to a demand for small-scale, GMP-ready mixing solutions. The Cytiva 10 L LevMixer™ single-use mixing system extends the proven LevMixer technology to shear-sensitive applications including viral vectors and personalized medicine. This presentation shares in-house mixing performance data and computational fluid dynamics (CFD) modeling to demonstrate the strong performance and scalability of the LevMixer system at larger volumes. With consistent, gentle mixing, the 10 L LevMixer system enables efficient mixing from process development to clinical manufacturing for advanced therapies.

CREATe Tx developed a proprietary platform to generate designer receptors from virtually any GPCR. Combining gene therapy with pharmacological control, it allows selective, tunable modulation of neurons defined by location and neurochemistry. Initially targeting drug-resistant focal epilepsy, the platform shows broad CNS potential, enabling a new class of CNS therapeutics with unprecedented efficacy.

Recombinant adeno-associated virus (AAV) vectors are well-established in the gene therapy field providing a safe and effective means to treat a range of genetic disorders. The therapeutic potential and safety of the final product relies on the purification of AAV capsids containing the full intended gene product. The separation of empty from full AAV capsids is a subject of significant process development often requiring substantial time and resources to optimize. Analytical Ultracentrifugation (AUC) is considered the gold standard to characterize empty and full capsids in rAAV products. However, the method is difficult to implement in a routine QC environment, requires large sample volumes, and is sensitive to certain matrix contaminants, limiting its capability to characterize upstream processing steps. Additionally, the method cannot discriminate between capsids filled with the intended product or contaminating sources of DNA. Commonly for in-process sampling steps, a PCR-based assay is used to quantify the genomic titer and an ELISA-based assay is used to quantify capsid concentration. These data can also be used to characterize the ratio of empty to full capsids however this method suffers from compounding variability emerging from the two independent assays. Here we present a novel method for the determination of genomic titer, capsid concentration and empty/full capsid ratio within a single ddPCR reaction. The kit features a primer and probe set specific to the Inverted Terminal Repeat (ITR) region of the rAAV vector to determine genomic titer and allows for the substitution with gene-of-interest (GOI) specific primer and probe sets. Capsid concentration is determined through Proximity Ligation Assay (PLA) detection using two antibodies specific to the AAV capsid. We will cover the principles behind the kit’s functionality, demonstrate its reproducibility in a quality control environment, and highlight examples of its utility in the characterization of manufacturing processes and process improvement. We will show that the method can provide not only suitable genomic titer and capsid concentration values but can also provide datasets comparable to AUC across multiple matrix types. We will address how this tool can be used to optimize the yield of full, functional AAV particles while minimizing the impact of empty particles, ultimately improving the overall efficiency and scalability of AAV vector manufacturing.

Satellite Bio is aiming to treat liver disease with a hepatocyte cell therapy. In this presentation, we will discuss the development of a robust scalable process for expanding primary human hepatocytes while preserving their identity and function. By leveraging heterocellular aggregation with stromal cells, we also developed cryopreserved “seed” constructs that maintain viability and are reproducibly functional post-thaw.

Prime editing is a versatile genome editing technology with the potential to correct a broad range of disease-causing mutations. Establishing a modular and scalable manufacturing process is essential for translating Prime Editor Lipid Nanoparticles (PE-LNPs) into clinically viable therapies for liver diseases. A key challenge lies in ensuring the quality and reliability of PE-LNP components - particularly ionizable lipids, mRNA, and guide RNAs-to support the safety and efficacy of the final drug product. This presentation will highlight the transition from early development to scalable PE-LNP manufacturing, focusing on key process development strategies and CMC considerations.

• Internalising or externalising manufacturing – What are the decision points?
• How to select CDMO partners? – Industry criteria for selection
• When / what stage of development should you bring on second CDMO?
• Understanding CDMO capacities, timelines, and capabilities
• Qualification, and quality agreements for commercial manufacturing
• Tech transfer: best practices and risk management
• Strategies for transferring product from development phase to CDMO for large scale production: How to scale up?
• Lessons learnt from transferring to CDMO and vice versa
• Increasing skills and experience in manufacturing of next generation CGT products
• Partnership and communication – how to partner most effectively with CDMOs?

• Overcoming challenges at manufacturing scale
• Applying automation to meet manufacturing end points
• Achieving maximum doses per lot of drug product to drive patient cost down

• Precision genome engineering with chRDNA technology
• Strategies for armoring allogeneic cells to enhance potency and durability
• Scaling CAR-T production to broaden patient access

• Developing a closed system GMP-compliant manufacturing process for allogeneic NK cell therapies
• Cost-effective solutions in process automation
• Scale-up strategies
• Developing rigorous product quality analytics

TCR-T therapies offer immense promise for solid tumors, expanding beyond CAR-T. Realising this potential, demands scalable solutions addressing biological and manufacturing challenges. Anocca's end-to-end platform provides this through standardisation, high-throughput, and integrated, efficient manufacturing, including non-viral editing. This delivers a pipeline of personalized product libraries, with the first program in clinical development, making widespread TCR-T application feasible.

• Defining point-of-care manufacturing and decentralized manufacturing
• Scaling with consistency: key elements for global standardization
• Tech & innovation: cutting-edge solutions for safe, efficient, and cost-effective decentralized manufacturing
• Overcoming obstacles to decentralised manufacturing to enable access to approved CGTs
• Differences in systems across decentralized cell therapy manufacturing models - how can we harmonize?
• Standardization vs a modular approach
• Equipment, data, and technologies
• Dialogue and collaboration between developer/practitioner to streamline eventual implementation
• Improving worldwide access: how can local manufacturers and supplier help?
• Real world experience with a decentralized cell therapy manufacturing platforms supporting Phase I/II clinical trials

• Cryopreservation scientific principles and its applications in process development
• Development guidance in minimizing variation within and across batches from freezing to shipping
• Scale up challenges in fill-finish/cryopreservation and solutions

Presentation covers the current trend in automation used in CART cell therapy, manufacturing process and the unique consideration of supply chain. Also the challenges of new automation technology implementation and consideration for consumables design for automation systems are discussed.

The Biophorum ATMP Visible Particles workstream has proposed a holistic, lifecycle approach to reduce and de-risk visible particulates in cell therapy (CT) drug formulations. This involves characterizing and detecting particulates in the manufacturing process and formulation, then improving material quality and process controls to minimize them.

CTs face unique challenges in particulate control and detection compared to other injectables, including difficult-to-inspect formulations and containers. CTs, with inherent cell-related particulates, complicate the detection of other particles. Terminal sterilizing filtration isn't applicable due to cell size and formulation needs. Small batch volumes, especially in autologous therapies, make rejecting units with particle defects critical. Regulatory guidance and health authority expectations are not aligned with the unique characteristics of cell and gene therapies, posing challenges for sponsors in meeting particulate specifications.

Scalable and cost-effective manufacturing of pluripotent stem cells is essential for advancing regenerative medicine and cell therapy. We have developed a commercial-scale, automated, and closed-system expansion platform capable of producing billions of cells from a single vial with a 10-fold cost reduction. Our system integrates real-time, in-line process analytical tools to continuously monitor cell growth and health, ensuring optimal expansion conditions and consistent product quality. Additionally, we have implemented and validated analytical metrics to meet regulatory and commercialization standards. By addressing scalability, automation, and regulatory compliance, our platform significantly reduces manufacturing costs and batch-to-batch inconsistencies and accelerates the path to clinical and commercial applications. This poster presentation will cover the system’s design, analytical validation, and its impact on the future of stem cell manufacturing.

At Fujifilm Diosynth Biotechnologies, our teams understand that along with cutting edge science there is trepidation in choosing a partner. When your technology and science is advancing quicker than CMDOs can build experience, it can be hard to feel confident that a contract manufacture can get it right. That is why we have created an early access program which builds a strategy alongside our customers, establishing a roadmap to manufacturing through quicker data generation and collaborative consulting mentality. By showing our customers that we can reproduce their results, optimize their process, or simply grow their cell type, our teams build trust and provide a data set that customers can leverage to raise capital, show their leadership, and design the tech transfer that meets all of their program goals.

Timely and successful Investigational New Drug (IND) applications in cell and gene therapy (CGT) require manufacturing partners with deep technical expertise, regulatory foresight, and the ability to balance platform efficiency with program-specific customization. ProBio has supported over 77 IND-enabling programs across plasmid DNA, adeno-associated virus (AAV), and lentiviral vector (LVV) modalities, helping innovators streamline Chemistry, Manufacturing, and Controls (CMC) processes while ensuring regulatory compliance.

This session will share key insights and technical strategies from ProBio’s experience in accelerating CGT CMC development, including:

• Enhancing vector productivity: Leveraging platform-based packaging systems, optimized helper plasmid design, and scalable upstream processes to improve AAV and LVV titers and reduce batch-to-batch variability.
• Strengthening product quality: Implementing rigorous release specifications, robust analytical method development, and phase-appropriate assay strategies to align with global regulatory expectations.
• Compressing development timelines: Facilitating efficient technology transfers and phase-specific CMC documentation to mitigate risk and support accelerated IND submissions.

Through real-world case studies, we will demonstrate how ProBio’s integrated manufacturing platform, scientific expertise, and flexible partnership model enable CGT developers to progress from process development to GMP manufacturing with speed, quality, and regulatory confidence.

• What is required for product characterization by regulatory authorities?
• Use of risk-based assessment to identify product CQAs
• Development of rapid process characterization assays
• How analytics can be used to support the move to “the product is the product”
• Design potency assay qualification studies

• General information on phase appropriate assay development
• Functional characterization as base for the commercial potency assay
• Examples of two potential potency assays
• Example of how to select one of the two assays as the commercial product potency assay

Quality control in cell and gene therapy (CGT) involves large molecule characterization, vector insertion mapping, and adventitious virus detection using next-generation sequencing (NGS). These tools enable real-time genome monitoring, verification of vector integration, and early detection of contaminants, which are essential steps to ensure product quality throughout development. Epigenetic testing, which evaluates chromatin structure and gene expression, adds critical insight into therapeutic performance and manufacturing consistency. Demonstrating how these integrated genomic and epigenetic approaches address common challenges in CGT manufacturing can help strengthen product reliability and fulfill the growing expectations of regulators, development teams, and patients for the long-term success of CGT.

UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene for the treatment of Wilson Disease. UX701 leverages Ultragenyx’s proprietary producer cell line platform, Pinnacle PCL™, to produce rAAV at 2000L scale.

Process characterization of UX701 is an essential aspect of the process validation lifecycle, aimed at defining manufacturing process ranges to maintain the target product profile (TPP). A risk-based approach was applied to the late-stage development activities for UX701 upstream and downstream process including process parameter and raw materials risk assessment, scale-down model (SDM) qualification, and process characterization (PC) studies, to establish process controls and inform parameter criticality for the upstream and downstream unit operations in the 2000L manufacturing process. We assessed over 400 upstream and downstream process parameters for their impacts to process performance indicators (PIs) and critical quality attributes (CQAs) and classified them into high, medium or low risk parameters. Of these we studied 11 high and medium risk upstream process parameters and 2 raw materials, and 14 high and medium risk downstream process parameters in PC studies.

We identified 8 process parameters in upstream and 3 downstream process parameters to be critical (CPP) or key (KPP). In the upstream process, temperature and pH, seeding density, and helper virus concentration were all found to be CPPs during production. In the downstream process, viral heat inactivation step, temperature and time affect the inactivation kinetics of the helper virus. Characterization of our anion-exchange (AEX) polishing chromatography step identified an upper threshold which the load conductivity should stay below to ensure binding onto the AEX column.

In this work, we have characterized the cell culture and purification manufacturing process and demonstrate a robust and high yielding process for the manufacture of UX701. This is the first PC campaign for our Pinnacle PCL™ rAAV gene therapy manufacturing platform.

In the rapidly evolving field of gene therapy, technology transfer for a production process from research or development to cGMP manufacturing is a crucial activity that requires effective communication from planning stages throughout execution. This presentation will explore the general approach for technology transfer to Contract Manufacturers, with a specific focus on best practices for the gene therapy sector.

• Developing and adopting simplified, scalable state-of-the-art platforms to fulfill demand needs for commercial gene therapy manufacturing.
• Evaluating innovative approaches to address the current lack of robust analytics to achieve comprehensive characterization of gene therapy product.
• Introducing potential alternative gene therapy platforms to counter concerns around viral vector safety and toxicity.

Production of viral vectors for gene therapy is focused on providing pure, safe, and effective products. The absence of impurities and the presence of a complete vector genomes play a crucial role. Information obtained by several different capsid content characterization methods might be incomplete or even misleading. For this reason, we have focused ourselves on evaluation of viral vector genome integrity. The recombinant viral genome contains all the information required for the therapeutic effect of the manufactured construct (e.g., promoter, enhancer, gene of interest, polyA tail). Incomplete viral genome leads to no therapeutic effect or a much lower therapeutic effect than expected. In our case study we have analyzed several fractions of AAVs after being eluted from AEX (including several fractions of peaks of empty and full particles). For each of these fractions we have quantified the viral vector genome integrity by multiplex digital PCR and obtained detailed information on titters of complete genomes and different genome fragments. Additionally, we have transduced one human cell line with those samples and determined the transduction efficiency by monitoring the GFP production in the cells. Finally, we were able to make correlations between genome fragment content and the GFP expression and intensity. With this correlation we have pointed out that viral vector genome integrity evaluation is a very useful tool in the process development and for sample characterization, as it provides a direct link to the final efficacy of the rAAV sample.

In order to achieve development timelines, rapid process development can result in the sacrifice of capsid recovery in order to meet specifications. By establishing a strategy focused on critical process parameters, leveraging design of experiments strategies, and using statistically sound analytics, genome-containing capsid recovery can be optimized in recombinant AAV downstream unit operations.

Comprehensive characterization of AAV8 critical quality attributes (CQAs), specifically empty and full capsids, was performed using orthogonal techniques including AUC, cryo-TEM, MADLS, ELISA, CE, and SEC. Each method provided unique insights into capsid composition and product quality. Together, this analytical strategy allowed for a robust assessment of vector quality to support regulatory submissions.

Gene therapy has emerged as a cutting-edge modality with transformative potential for treating a broad range of genetic disorders. Despite significant advancements, the purification and separation of the product of interest from process- and product-related impurities remains a major challenge in the development, particularly for adeno-associated virus (AAV) vectors. Leveraging Quality by Design (QbD) principles, we have developed a high-throughput downstream development platform for viral vectors, increasing the throughput of downstream purification operations to over 100 purifications per week, supported by at-line analytical capabilities. This platform enables rapid process characterization and optimization, significantly reducing development timelines while supporting robust process understanding and consistent product quality. The results presented will highlight advancements in high-throughput downstream purification for AAVs as well as formulation development for process intermediates. Together, these innovations demonstrate a path toward faster, more cost-effective, and higher-quality AAV process development, aligning with QbD frameworks to enable more efficient delivery of gene therapy products to patients.

We will explore how novel clarification technology, based on advanced synthetic fibrous chromatography materials, will enable new bioprocess strategies to address critical challenges in process simplification and intensification. We will illustrate how this platform can offer seamless implementation of chromatographic clarification from discovery to clinical and commercial manufacturing, providing consistent and high-quality clarified fluid and enhancing commercialization productivity.

What does MVM infectivity in HEK cells mean for your viral safety profile? Here we discuss detectability of MVM using adherent HEK cells and how to integrate MVM detection into your AAV Viral Safety Strategy.

AAV and Lentiviral vectors are widely utilized tools in cell and gene therapy due to their unique characteristics. For different applications, it's crucial to ensure the product's purity and demonstrate the elimination of impurities. CIM® monoliths play a vital role in achieving high purity standards of the final product. When paired with PATfix® on the analytical side, they allow for the assessment of recovery, monitoring of impurity removal, and act as complementary analytics to traditional methods, offering a more comprehensive understanding of the sample's composition.

Host cell DNA (hcDNA) levels in AAV-based in vivo gene therapies have substantial implications for a product’s safety profile, however the current industry baseline is very broad due to order-of-magnitude differences in dose level from product to product. To obtain a more valuable baseline, members of a BioPhorum industry collaboration were surveyed to assess normalized hcDNA levels to establish a range for current manufacturing practices. Based on the BioPhorum industry survey, health authority guidelines, and literature, this presentation will cover the challenges with hcDNA and summarize the survey results, including current practices from industry on hcDNA analysis, control and reporting. An approach for harmonized reporting of hcDNA for AAV, based on normalization, will be proposed, with the collective benefits outlined.

Wrap up an insightful first day of the conference with a relaxed networking session over drinks and nibbles. Join fellow cell and gene therapy professionals, industry leaders, and innovators for an evening of meaningful conversations, idea-sharing, and collaboration opportunities. Whether you're looking to strengthen existing connections or forge new ones, this informal gathering provides the perfect setting to engage with peers in a friendly and welcoming atmosphere. Don't miss this chance to unwind, expand your network, and set the stage for productive discussions on Day Two!

Join us for an unforgettable evening of networking, innovation, and discovery at the iconic Museum of Science, Boston! Mingle with fellow cell and gene therapy professionals over delicious food and drinks while exploring fascinating museum exhibits. Engage in lively conversations, make valuable connections, and immerse yourself in an inspiring atmosphere where science and industry meet. This exclusive event offers the perfect blend of socializing and discovery—don’t miss this chance to connect, unwind, and experience the wonders of science in a truly unique setting!

The CGT C-Level Forum provides a unique, powerful and valuable space for candidly sharing ideas and experiences between executives of ~20 CGT therapeutic development companies. The forum is designed to foster new ideas and create new relationships between attendees so that they can leave with tangible outcomes and that will drive their businesses and the sector forward.

An exclusive invite-only session, in a closed room setting, the forum will focus on discussing the right approach to engage and partner with pharma and industry. It will also include general discussion focused on brainstorming solutions to the most pressing and current challenges of CEO/CSO/CBO/CMOs in the CGT sector.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event a Cell and Gene Therapy International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at Cell and Gene Therapy International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join us for an engaging Lunch & Learn session where leading technology, product, and service providers showcase the latest advancements in bioprocessing. Enjoy your meal while exploring cutting-edge solutions designed to enhance efficiency, scalability, and innovation in biomanufacturing.

This interactive session offers a unique opportunity to gain insights into breakthrough technologies, ask questions directly to industry experts, and network with peers—all in a relaxed, informal setting.

Whether you're looking to optimize your processes, explore new tools, or simply stay ahead of industry trends, this is a must-attend event at BioProcess International.

Join industry leaders as we explore how AI, digitalization, and advanced analytics are transforming cell and gene therapy manufacturing. This dynamic panel will dive into:

• AI-Powered Manufacturing: How AI-driven models, predictive analytics, and digital twins are optimizing process control
• Process Analytical Technologies (PAT): The latest breakthroughs in real-time monitoring, automation, and data-driven decision-making
• Overcoming Data Challenges: Strategies to build robust AI models despite limited datasets

Success Stories: Case studies showcasing AI and PAT integration for improved yield, efficiency, and product quality in cell and gene therapy manufacturing and process development

• Snapshot into the investment landscape for CGT products and enabling technologies for 2025/ 2026 and beyond
• How to drive investment in CGT?
• Positioning for success in a competitive funding environment: What do investors need to see?
• “Ask the Investor” interactive Q&A open discussion with our investor panel to get tips and tricks when pitching and their views on where innovation lies in the CGT field

At Fujifilm Diosynth Biotechnologies, our teams understand that along with cutting edge science there is trepidation in choosing a partner. When your technology and science is advancing quicker than CMDOs can build experience, it can be hard to feel confident that a contract manufacture can get it right. That is why we have created an early access program which builds a strategy alongside our customers, establishing a roadmap to manufacturing through quicker data generation and collaborative consulting mentality. By showing our customers that we can reproduce their results, optimize their process, or simply grow their cell type, our teams build trust and provide a data set that customers can leverage to raise capital, show their leadership, and design the tech transfer that meets all of their program goals.