Jorge Santiago-Ortiz
Vice President, CMC & Regulatory Affairs Apertura Gene Therapy
Seminars
Wednesday 24th February 2027
Scalable Novel Engineered Capsids: From Platform Manufacturability to First-in-Human
9:00 am
- Demonstrating that a novel engineered capsid can be manufactured on existing platform processes with productivities and yields comparable to AAV9 and confirmed at 500L clinical scale
- Building the capsid access and tech transfer infrastructure that lets partners move quickly: established master cell banks and rep-cap plasmid at CDMOs, plus low-friction material transfer pathways for academic, foundation, and industry programs
- Constructing an FDA-informed, MOA-driven CMC and preclinical package for a human receptor-targeted capsid to support regulatory filings and first-in-human dosing
