Regulatory Pathway for Gene Therapy for Rare Diseases: An Illustrative Example from the PaVe-GT Platform Program

Authors

• R Stand
• RM Lomash
• RJ Chandler
• OA Shchelockov
• CP Venditti
• CG Bonneman
• PJ Brooks
• EA Ottinger

Abstract

The Platform Vector Gene Therapy (PaVe-GT) program is an initiative started by a multi-disciplinary group of collaborators to develop adeno-associated virus (AAV) gene therapies for four rare diseases. With approximately 10,000 rare diseases that affect 25 to 30 million people in the US and less than 10% of these diseases having an approved treatment, there is much work to be done. Addressing the need for therapies for rare diseases under a unified platform may serve as a paradigm for reducing process redundancies and increasing efficiencies in the preclinical, regulatory, and clinical activities to broadly enable the development of gene therapies for rare diseases.
Here, we showcase the regulatory strategy for the PaVe-GT program’s first indication. AAV9-hPCCA gene therapy is being developed to treat propionic acidemia resulting from a deficiency of propionyl-CoA carboxylase, alpha subunit (PCCA). The product’s regulatory pathway has thus far progressed successfully through an INTERACT meeting with the FDA and is in pre-IND planning phase. This gene therapy project has also received two valuable designations from the FDA: Orphan Drug and Rare Pediatric Disease, which provide sponsors with financial incentives for research and development of drugs for rare diseases. Lessons learned from our regulatory activities will be presented.
In conclusion, the knowledge achieved by advancing PaVe-GT program’s first product will be applied to the other three indications under development, to reduce process redundancies and increase efficiencies. PaVe-GT represents a strategy for accelerating therapeutic development for rare diseases that can be disseminated to other AAV gene therapy efforts.

Scientific Focus Area: Clinical Research

This page was last updated on Monday, September 25, 2023