Creating drug master files for AAV vectors
FDA, NIH leaders want to help the business case for ultra-rare disease gene therapies. FNIH’s Bespoke Gene Therapy Consortium is the vehicle
FNIH has a vision for scaling gene therapy development that involves creating master files for viral vectors that can smooth the regulatory path and improve reproducibility. The hope, among FDA and NIH leaders, is to help the business case for ultra-rare disease gene therapies by not needing to reinvent the whole wheel for each new therapy.
Realizing the vision will require pre-competitive collaboration among public, private and non-profit sectors, which FNIH is facilitating through its new Bespoke Gene Therapy Consortium...