De-risking Translation in Rare Ophthalmic Gene Therapy: The Axovia Experience
Time: 2:30 pm
day: Conference Day One
Details:
- Leveraging a disease-specific animal model, single-species toxicology, and dose-ranging studies to assess tolerability and efficacy
- Navigating an adaptable regulatory pathway, pivoting from US to UK-based Phase 1 trials, with a registrational trajectory toward BLA submission
- Driving patient access through close PAG engagement and optimizing recruitment strategies
