Demonstrating efficacy and value for one-and-done therapies
15 Apr 2025
Gene Therapy Development
- Control arm in pivotal studies – is Placebo necessary and/or ethical?
- Clinical endpoints borrowed from non-rare diseases: do they truly apply to the rare diseases for gene therapy?
- How are doing in terms of managing toxicities that are inherent for AAV delivery e.g. liver injury?
- Immunosuppressive regimen – are we close to a standardized regimen by age?
- Dose selection for gene therapy in clinical trials – is higher always better?
