The gene therapy industry is mobilizing to address critical analytical and quality control challenges through two major European summits, as recent regulatory setbacks underscore the urgent need for precise, validated analytics in this rapidly evolving field.
The 6th Gene Therapy Analytical Development Summit Europe, scheduled for May 20-22, 2025 in London, arrives at what organizers describe as "a critical moment for the field." Recent approvals like LYFGENIA and BEQVEZ have raised the bar for analytics, while setbacks such as Rocket Pharma's KRESLADI rejection have exposed CMC vulnerabilities across the industry.
Regulatory Pressures Drive Industry Response
The summit will unite 24+ expert speakers and over 90 industry stakeholders to tackle analytical strategies that can elevate development programs, streamline regulatory approvals, and safeguard therapeutic efficacy. The event positions itself as "the only European forum solely dedicated to tackling the analytical and quality control challenges across the gene therapy lifecycle from Pre-IND/IMPD to BLA."
Key speakers include Uditha De Alwis, VP Analytical Sciences at Sarepta Therapeutics, and Claire Kerridge, Head of Gene Therapy at MHRA, alongside experts from the British Pharmacopoeia, AAVantgarde Bio, Orchard Therapeutics, and GenSight Bio.
Comprehensive Analytical Framework
This year's expanded agenda addresses critical content areas including method validation, characterization, comparability, QC readiness, and potency assays—all tailored to meet European regulatory expectations and harmonize with global frameworks like the FDA and EMA. The program features hands-on workshops on PCR strategy, potency assay challenges, and QC/CMC synchronization.
The summit emphasizes practical solutions for adapting and validating assays under evolving regulations, scaling up with analytical integrity, and navigating tech transfer without compromising consistency. Leading service providers including SGS, Intertek, Sartorius, Abselion, QuTEM and Quatre Lab will showcase cutting-edge innovations in analytics and testing services.
Specialized Focus on Potency Challenges
Complementing the broader analytical summit, the inaugural Gene Therapy Potency Summit (August 29-31) addresses what organizers call "the only industry-led event dedicated to covering all challenges associated with potency for gene therapy drug developers specifically." The event focuses on the "sweet spot" of drug development where potency is most critical—pre-clinical development through commercialization.
The potency summit will unite 60+ analytical, bioassay, Quality Control, regulatory and CMC experts, featuring case studies from major players including Sanofi, Biogen, Sarepta Therapeutics, and Spark Therapeutics. Key focus areas include preventing clinical holds through phase-appropriate adaptations, improving potency assay techniques for products with unknown mechanisms of action, and developing best practices for cell-based assays.
Industry-Wide Collaboration
Both summits emphasize peer-to-peer learning and real-world application through roundtables, fireside chats, and panel discussions. The potency summit specifically addresses bridging differences between assays to reduce biological variability and ensuring continuity throughout the product lifecycle.
Sarepta Therapeutics will present strategies for creating workable long-term program management plans from day one, accounting for changes throughout the development process. AGTC will share insights on applying phase-appropriate adaptations when dealing with regulators, while REGENXBIO and Ultragenyx will focus on improving assay transferability and reducing turnaround times.
