ARTICLE | Regulation
FDA’s positive reinforcement
How FDA’s gene therapy guidances reinforce the paths of the most advanced candidates
July 20, 2018 11:07 PM UTC
Companies are still sifting through a half dozen gene therapy guidance documents issued by FDA, but so far it appears the recommendations support the paths taken by some of the most advanced clinical candidates for hemophilia and retinal disorders. The new documents also could eliminate or reduce the requirements for follow-up studies for some products.
Three of the six draft guidances issued July 11 cover development of gene therapies to treat rare diseases, retinal disorders and hemophilia...