Cell and gene therapy has crossed a critical inflection point. Scientific feasibility is no longer the limiting question—AAV, lentiviral, and emerging non-viral delivery systems, combined with precise gene editing technologies, have demonstrated the ability to correct disease at its source. The challenge now is translation: transforming bespoke, N-of-1 breakthroughs into scalable, reliable therapies accessible to the many.

This keynote will explore the path from possibility to platform. While AAV and other modalities offer compelling advantages in efficiency, durability, and safety, they also expose persistent barriers—immunogenicity, delivery to the right tissues, limited payload capacity, and the complexity and cost of manufacturing at scale. These challenges are not isolated technical problems; they sit at the seams between research, development, manufacturing, and regulatory execution.

Drawing on insights from the ASGCT “N=1 to N=Many” symposium, Dr. Sadik Kassim will outline a forward-looking framework anchored in platform thinking—where standardized processes, advanced analytics, and scalable manufacturing converge to enable reproducibility and access.

Critically, the keynote will emphasize that progress at this stage is fundamentally collaborative. Cross-industry partnerships—spanning academia, biopharma, technology providers, and regulators—are no longer optional; they are the rate-limiting step in accelerating advanced therapies from breakthrough science to global impact.