Overcoming manufacturing challenges for gene-enabled cell therapies
This webinar examined how to scale gene-enabled cell therapies, with emphasis on allogeneic programs moving toward commercial manufacture. The panel covered regulatory strategy, comparability, manufacturing capacity, and supply chain resilience, stressing early integration of quality and regulatory functions, consistent analytics across sites, and pragmatic risk mitigation with suppliers. Differences between U.S. and EU expectations, especially around donor requirements and GMP, require tailored global plans. The group also highlighted talent constraints and the value of transparent agency engagement from the start.
12 Jul 2022
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Ashley Alderson
Speakers
Kelly Davis-Claeys – Director, Regulatory Affairs, Catalent.
Tim Farries, PhD – Chief Scientific Officer, ERA Consulting.
Move fast, validate smart: Accelerated development demands fit-for-purpose validation and qualified analytics that still meet GMP.
Comparability is a gatekeeper: Plan for it early, avoid late process changes during pivotal studies, and align assays across sites.
Secure GMP-grade inputs: Dual-source critical materials and pre-plan substitutes to avoid bottlenecks.
Know your regulators: FDA and EU approaches differ; build region-specific strategies and keep communication open.
Capacity remains tight: Blend internal capability with flexible CDMOs and realistic lead times.
Limit multi-CMO complexity: If multiple sites are unavoidable, harmonize methods, data models, and QMS expectations upfront.
Bring QA/RA in early: Cross-functional design from day one reduces costly delays later.
FAQs
Q1. What are the top hurdles for allogeneic programs going commercial? A. Rapid but robust validation, product comparability during scale-up, dependable GMP supply, and managing biological variability across cell sources.
Q2. How is the regulatory picture evolving? A. Agencies are issuing more specific guidance and gaining experience, but expectations are still moving. Early, frequent dialogue helps de-risk filings.
Q3. Why is comparability so hard in cell and gene therapy? A. Small process shifts can change sensitive products. Prove sameness with rigorous, standardized analytics, especially in pivotal phases.
Q4. What risks come with multiple CMOs? A. Inconsistencies in tech transfer, assays, and documentation can slow reviews and jeopardize quality. Strong governance and data sharing are mandatory.
Q5. How should teams prepare for late-stage and launch? A. Build GMP-compliant, scalable processes early, integrate regulatory and quality expertise, stress-test your supply chain, and maintain open lines with regulators.
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