Thursday, September 26, 2024 - Day Three of Main ConferenceThursday, 26 September 2024 - ET (Eastern Time, GMT-05:00)

Moore's Law, a foundational principle in the realm of technology, posits that the number of transistors on a microchip doubles approximately every two years, leading to exponential growth in computing power and efficiency while reducing costs. While not directly mirroring technology, the concept finds resonance in the rapid pace of medical advancements and pursuit of new modalities driven by exponential growth in knowledge, capabilities, and understanding. This accelerated pace enables medical breakthroughs but also poses technical challenges, specifically in manufacturing and delivery of new modalities like mRNA and other nucleic acid medicines. We are on the precipice of a revolution in R&D that marries the rapid advancement in modalities with targeted delivery vehicles and streamlined manufacturing to produce the next generation of medicine.

Through leveraging technological advancements in machine learning, at Flagship we have built an open end-to-end platform that harnesses vast knowledge, data, and tools to design and manufacture near infinite medicines with the highest possibility of translational success against target indications across the body. This relentless drive for innovation across the industry has the promise to expand the boundaries of what is possible exponentially to realize future of nucleic acid medicine.

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.

Explore our other tracks to curate your ideal conference experience, and discover exciting new content that sparks your curiosity!

• Co-mixtures add complexity to the assessment of product quality and potency
• Compromises may be required at the formulation stage to enable acceptable product quality/stability for each API
• Agency expectations with regards to stability profiles for the individual API’s within the co-mixture compared to as individual products
• Sorting through the impurity profiles in the co-mixture

• 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.

Welcome To Group Therapy. Let's share the pain. Please join us and come prepared with your bioprocessing related problems. How it will work is we have 4 Round Tables each covering a different area of Bioprocessing. Each will be led by an expert. The expert will guide the discussion allowing participants to share their challenges and time to discuss within the group. These will last for 40 minutes. Included are some examples below:

Round Table Discussions

Cell Line Development & Upstream

• Expanding use of various cells: from Bacteria, yeast, and animal cell platforms to therapeutic cells
• The expanding nature of products: from therapeutic cells, to peptides, proteins, nucleic acids, and viruses
• Upstream sustainability: defining clear goals, establishing unambiguous metrics. and evaluating available options
• Upstream implications of AI: from process control, to supporting personalization, to heightened PAT, to continuous process verification
• Process intensification: Tools for increasing final product yield per cost, time, volume, footprint, personnel, and environmental burden generation
• Process, equipment, and suite flexibility: considering specific goals, performance and financial costs, and the various tools and solutions available

Recovery & Purification

• Standards and Validation: Meeting regulatory requirements and documenting processes for quality assurance
• Product Stability: Maintaining product integrity is essential for safety and efficacy. Degradation or aggregation can compromise the biologic's effectiveness and lead to safety concerns
• Yield & Efficiency: High yield and efficient recovery are crucial for the economic viability of the production process. Low yield or inefficient processes can significantly impact the cost and scalability
• Impurity Removal: Effective removal of impurities is vital to ensure product purity and safety. This includes removing contaminants like host cell proteins, endotoxins, and residual nucleic acids
• Cost and Time: Balancing cost and time with quality is essential for commercial success. High costs or prolonged production times can affect the economic feasibility of the biologic
• Solubility and Precipitation: Maintaining protein solubility and managing precipitation issues

Innovation & Adaptation:

• Compliance: Ensuring that new processes comply with existing regulations and standards can be challenging
• Compatibility: New technologies must integrate seamlessly with existing systems and workflows, which may require significant adjustments
• Data Integration: Managing and integrating data from new systems with legacy systems can be complex
• Economic Justification: Demonstrating the return on investment (ROI) and economic benefits of adopting new technologies
• Operational Disruption: Implementing new technologies can disrupt existing processes and workflows
• Cultural Resistance: Overcoming resistance to change within the organization

Cell & Gene Therapies

• Approval Processes: Lengthy and complex approval processes for new therapies
• Production Scale-Up: Difficulty in scaling up manufacturing processes from lab to commercial scale while maintaining consistency and quality
• Batch-to-Batch Variability: Managing variability between production batches
• Economic Viability: Demonstrating cost-effectiveness and economic viability for widespread use
• Material Sourcing: Securing reliable sources of high-quality raw materials and reagents
• Cold Chain Logistics: Maintaining cold chain logistics for sensitive products
• Customization: Customizing therapies for individual patients, which complicates standardization and scalability
• Long-Term Safety: Monitoring and ensuring the long-term safety of patients receiving cell and gene therapies
• Off-Target Effects: Minimizing off-target effects and unintended consequences of gene editing
• Advanced Analytics: Employing advanced analytics to develop and validate robust assays for measuring potency, purity, and safety.

• How do you avoid greenwashing and ensure your efforts are viewed as credible? What role does third-party verification play in that?
• 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?
• Renewable energy purchasing and PPA's
• 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
  • Challenges in contract manufacturing and M&A
  • Focus on Scope 3 and how to address it: What is one cool industry collective action initiative
  • Focus on product development (80% for design for sustainability), going upstream
  • Complex global value chain- need to work together to solve sustainability and create circularity.

• 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.

• 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.

• How do you avoid greenwashing and ensure your efforts are viewed as credible? What role does third-party verification play in that?
• 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?
• Renewable energy purchasing and PPA's
• 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
  • Challenges in contract manufacturing and M&A
  • Focus on Scope 3 and how to address it: What is one cool industry collective action initiative
  • Focus on product development (80% for design for sustainability), going upstream
  • Complex global value chain- need to work together to solve sustainability and create circularity.

Acknowledged as an emerging cluster for biotechnology across the world, India’s biotech sector is valued at approximately $150 billion and is on track to reach $300 billion. This session will spotlight India’s cutting-edge research capacities, advanced biomanufacturing facilities, and expertise in biomanufacturing, bio-services, and drug development capabilities. The session will explore how tapping into India’s rapid growth and innovation propels advancements in global biomanufacturing.

• 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.

• 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.

• 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 finals competition winner announcement will run from 3:00pm-4:00pm

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.