Cambridge Healthtech Institute’s 12th Annual
Gene Therapy CMC
Improving Quality, Control, and CMC of Genetic Medicines and in vivo CAR Ts
June 15-16, 2027
Cambridge Healthtech Institute’s 12th Annual Gene Therapy CMC conference explores the quality, control, and analytical strategies advancing the next generation of AAV, lentiviral, LNP, and gene-edited therapies. Key sessions address CMC readiness for BLA submission, lessons from recent approvals and setbacks, evolving FDA expectations, platform- and cargo-based pathways for ultra-rare and N-of-1 personalized therapies, and CMC considerations for in vivo CAR T therapies. Additional topics include potency assessment for complex mechanisms of action, structure–function relationships, process development for engineered capsids, large-payload characterization, and functional particle analytics. Sessions also examine formulation, LNP targeting, heterogeneity, and redosing; integration and genome profiling; compendial methods; device compatibility; reusable control strategies; AI-enabled documentation; and heightened inspection expectations. Attendees will gain practical insights to strengthen CMC strategies, advance development, and prepare the next generation of genetic medicines.
Coverage will include, but is not limited to:
- Sharing CMC lessons from recent approvals and setbacks
- Addressing CMC challenges for in vivo CAR T (viral and non-viral)
- Process development and characterization of next-generation engineered capsids, large payloads, truncated genomes, empty and full ratios, and functional particles
- Navigating evolving FDA expectations, platform guidance, and pathways for ultra-rare and N-of-1 therapies
- Evaluating potency for complex mechanisms and gene-edited therapies, with structure-function correlations
- CMC and analytical strategies for viral vector, in vivo CAR T, and gene-edited therapies
- CMC for exosomes
The deadline for priority consideration is November 20, 2026.
All proposals are subject to review by session chairpersons and/or the Scientific Advisory Committee to ensure the overall quality of the conference program. Additionally, as per Cambridge Healthtech Institute’s policy, a select number of vendors and consultants who provide products and services will be offered opportunities for podium presentation slots based on a variety of Corporate Sponsorships.
Opportunities for Participation:
- To submit a podium presentation, please click here.
- To become a sponsor, click here.
- To present a poster, click here.
- To sign up to attend, click here.