At Advanced Therapies Week 2025, the video presents a comprehensive discussion on critical aspects of cell and gene therapy manufacturing, focusing on strategic planning, quality control, process development, and potency assay development. The speakers emphasize the importance of beginning with the end in mind, mapping out a clear pathway from early research through clinical trials to commercialization, ensuring regulatory compliance, and integrating quality systems from the outset. They highlight challenges unique to cell and gene therapies, such as variability in starting material, assay development complexity, and the need for flexible, scalable manufacturing platforms.
Donna opens by stressing the necessity of early strategic planning, including understanding regulatory expectations for Biologics License Applications (BLA), especially the Chemistry, Manufacturing, and Controls (CMC) section, which often causes delays. She advocates building quality into processes from day one, including robust quality systems, data integrity, and documentation. Donna underscores the value of internal controls and early assay validation to avoid costly rework later.
David then delves into process development, encouraging a thorough understanding of available manufacturing technologies and the importance of choosing partners who can scale and evolve with the therapy. He stresses designing simple, scalable, and flexible manufacturing processes that maximize equipment utilization and minimize fail points. He discusses the evolution of cell therapy manufacturing platforms and reagents, highlighting the increasing complexity and variety of options compared to a decade ago.
Kasia shares insights from Quell Therapeutics’ journey in manufacturing autologous T-regulatory cell therapies. She emphasizes the critical role of knowledge management and manufacturing feedback loops to reduce operational variation and improve process stability. Kasia illustrates how integrating data across clinical, manufacturing, and development teams enables better process control and product consistency, ultimately leading to scalable, tech-transferable manufacturing.
Scott focuses on potency assay development, advocating for early initiation of assay design, risk assessment, and phased validation. He introduces an innovative physiologically relevant miniaturized in vitro assay platform (PRIMA) that mimics human circulatory conditions to assess potency and mechanism of action more accurately. Scott emphasizes that potency assays must be specific to the therapeutic and that multiple assays should be developed initially, then refined through qualification and validation.
In the concluding panel discussion, speakers agree that flexibility and a deep understanding of the product’s biology are essential for integrating new technologies and ensuring long-term success. They recommend leveraging internal process controls and robust knowledge management systems to facilitate comparability and process improvements. Early engagement with regulatory requirements and pragmatic decision-making in technology adoption are also highlighted as key factors.
Overall, the Advanced Therapies Week 2025 session provides a detailed roadmap for biotech companies developing advanced therapies, from early clinical stages through to commercialization, with a strong focus on quality, flexibility, and strategic foresight.
Highlights
- Begin with the end in mind: map out the strategic pathway early to meet regulatory and investor expectations.
- Integrate quality systems and documentation from day one to ensure data integrity and regulatory compliance.
- Develop and validate potency assays early, using a phased approach aligned with clinical development.
- Choose manufacturing partners who are flexible and can scale with your therapy’s evolving needs.
- Implement manufacturing feedback loops and companywide knowledge management to reduce variation.
- Simplify manufacturing processes to minimize fail points and maximize equipment utilization.
- Leverage innovative assay platforms, such as physiologically relevant miniaturized systems, for better potency assessment.
Key Insights
- Strategic Mapping Drives Success: Donna’s insistence on mapping the pathway from early stages to BLA submission is crucial. Early understanding of CMC requirements, which drive many BLA issues, prevents delays and aligns resources with commercial realities.
- Quality Systems Are Foundations, Not Afterthoughts: Integrating quality systems and documentation early protects data integrity and strengthens regulatory submissions and investor confidence. Building a quality culture from the start reduces rework and pushback.
- Knowledge Management and Feedback Loops Enhance Process Control: Kasia’s focus on companywide data integration across development, manufacturing, and clinical functions enables continuous improvement and supports consistent, scalable products and smoother tech transfer.
- Flexibility and Scalability in Manufacturing Platforms Are Vital: David highlights the need to select simple, modular platforms that scale up and scale down efficiently, avoiding unnecessary complexity and fail points while enabling pivots as the field evolves.
- Potency Assay Development Requires an Early, Phased Strategy: Scott’s phased approach moves from multiple candidate assays to qualified and validated methods as programs advance, with PRIMA illustrating how physiologic models can improve relevance and predictiveness.
- Strategic Partnering Underpins Manufacturing Success: Selecting partners for today’s needs and tomorrow’s evolution is essential. GMP-fit reagents, closed-system compatibility, and responsive collaboration reduce risk as programs scale.
- Purposeful, Timed Automation: Automate where it adds value and plan for future automation without over-engineering early. This balances speed, cost, and quality as programs progress.
Conclusion
This Advanced Therapies Week 2025 discussion offers a practical framework that ties early CMC strategy, rigorous quality, flexible platforms, and potency assay planning to downstream success. With strong knowledge management, thoughtful partner selection, and phased technology adoption, teams can deliver consistent, scalable therapies that meet regulatory standards and reach patients efficiently.
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