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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?
Industry Expert
Dolly Parasrampuria, Head, Clinical Pharmacology, Cell & Gene Therapy - Astellas