Nature Reviews Drug Discovery
Volume 23 Issue 3, March 2024
Comment 06 Feb 2024
The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases
A public–private partnership managed by the FNIH aims to address biological, manufacturing and regulatory challenges to the development of gene therapies for rare diseases.
P. J. Brooks, Timothy M. Miller, Joni L. Rutter