Gene Therapy Applied To Mitochondrial Disease: A Guide To Emerging Regulatory Pathways - Part 2

By Kaitlyn Rouillard, PhD, Regulatory Scientist, and Rachel Ward, PhD, Associate Director, Regulatory Affairs

Continuing from our discussion in "Gene Therapy Applied to Mitochondrial Disease: A Guide to Emerging Regulatory Pathways - Part 1," recent breakthroughs in gene therapy have ignited enthusiasm for its use in treating primary mitochondrial diseases (PMDs). Unlike current therapies that solely manage symptoms, gene therapy for PMDs offers the possibility of complete or partial remission, a revolutionary prospect for patients. This promising development aligns with the FDA's priority of delivering innovative and effective treatments to patients with rare diseases as swiftly as possible. Consequently, the FDA has established various incentives and mechanisms to encourage sponsors to develop drugs for rare diseases.

This second part delves into the strategies and FDA recommendations for implementing gene therapy development programs, specifically designed for PMDs.