• Approaches to reaching high yield downstream purification
• Removing aggregates and contaminates from your titer
• Removal of partially-full capsids from your full-capsid yield

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.

• 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.

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.

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.

• 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.

• The Future of the Pharma Industry: Working Towards a Healthier Planet
• What are the key needs and goals of pharma and biopharma laboratories when it comes to sustainability?
• Biotechnology and pharma are among the world’s largest carbon-emitting industries. What steps need to be taken in the future to lower their position in this list of carbon emitters?
• What are the most significant challenges or barriers that the pharma industry faces in implementing more sustainable practices?
• The health sector generates four to five percent of total global emissions
• Mainly to do with the supply chain and the impact from globally shipping
• Complex global value chain- need to work together to solve sustainability and create circularity

• Highlight the importance of ensuring viral safety in biopharmaceutical products
• Describe methods for viral clearance, such as viral filtration and inactivation
• Present a few real-world examples of successful downstream bioprocessing projects.
• Discuss lessons learned and best practices from these case studies.
• Highlight current trends in downstream bioprocessing (e.g., continuous processing, single-use technologies).
• Discuss future directions and innovations in the field.

• What are CDMOs?
• What are the benefits of working with CDMOs
• Guidance on selecting and working with a CDMO, there are several key considerations to ensure a successful partnership
• Defining Clear Objectives and Expectations
• Risk Management

• Briefly explain the growing importance of digitalization, AI, and machine learning in the biopharma industry
• Key Areas of Digitalization
  • Data Management and Integration
  • Automation and Robotics
  • Real-time Monitoring and Analytics
• Applications in Manufacturing and Supply Chain
  • Use of digital twins and AI to optimize manufacturing processes and improve yield.
  • AI-driven real-time monitoring and anomaly detection in production lines
  • Use of AI for demand forecasting, inventory management, and logistics optimization
• Challenges and Considerations
• Case Studies
• Future Trends and Directions

• 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

The event will showcase the BTI ’24 cohort of best-in-class life science tools companies and a pitch competition featuring the BioTools Innovator 2024 Finalists. In addition to startup presentations, the event includes panel discussions with industry experts, and a Final Competition where one winner will take home the $250,000 Grand Prize, selected by a live audience vote.

Attendees will have the opportunity to network with BioTools Innovator partners, including investors, and senior executives from Life Science tools companies and others.

The event will showcase the BTI ’24 cohort of best-in-class life science tools companies and a pitch competition featuring the BioTools Innovator 2024 Finalists. In addition to startup presentations, the event includes panel discussions with industry experts, and a Final Competition where one winner will take home the $250,000 Grand Prize, selected by a live audience vote.

Attendees will have the opportunity to network with BioTools Innovator partners, including investors, and senior executives from Life Science tools companies and others.

• 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 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.
• Strategies and approaches to best utilise available technologies in the development & production of emerging therapies.
• Lessons and experiences from our panellists.

• Define analytical methods and their purpose in biopharmaceutical development and manufacturing
• Physicochemical Characterization - Techniques such as High-Performance Liquid Chromatography (HPLC), Mass Spectrometry (MS), and Nuclear Magnetic Resonance (NMR)
• Biological Assays - Cell-based assays, enzyme-linked immunosorbent assays (ELISA), and potency assays
• Microbiological Testing - Sterility testing, endotoxin testing, and microbial limits testing
• Role of Quality Assurance (QA) in Biopharma
• Quality by Design (QbD) and Risk Management
• Case Studies and Industry Examples
• Predict future advancements in analytical methods and quality assurance

The event will showcase the BTI ’24 cohort of best-in-class life science tools companies and a pitch competition featuring the BioTools Innovator 2024 Finalists. In addition to startup presentations, the event includes panel discussions with industry experts, and a Final Competition where one winner will take home the $250,000 Grand Prize, selected by a live audience vote.

Attendees will have the opportunity to network with BioTools Innovator partners, including investors, and senior executives from Life Science tools companies and others.