Demonstrating efficacy and value for one-and done therapies
25 Feb 2025
Gene Therapy Development
- What are the key considerations in assessing biodistribution and effective concentration for both known and novel capsids?
- How can we address gene therapy immunogenicity challenges, and what are the most promising strategies for successful re-dosing?
- What are the critical CMC (Chemistry, Manufacturing, and Controls) strategies when working with known and novel capsids to ensure quality and consistency?
- How can a regulatory strategy be optimized to achieve accelerated approval for novel gene therapies?
- What are the most effective clinical endpoints and trial designs for demonstrating safety and efficacy in gene therapy studies?
