White Paper

Gene Therapy Applied To Mitochondrial Disease: A Guide To Emerging Regulatory Pathways - Part 1

Scientist Using Pipette GettyImages-980092134

Gene therapy holds great promise for addressing the underlying cause of genetically-driven diseases, such as primary mitochondrial diseases (PMDs). PMDs are inherited disorders caused by mutations in mitochondrial DNA and currently have no cure. However, gene therapy offers personalized treatment by targeting the specific genetic mutation. The FDA has introduced expedited programs and incentives to support the development of treatments for rare diseases like PMDs. These programs provide benefits such as expedited regulatory review, market exclusivity, and tax credits for clinical trials. Gene therapies for PMDs may also qualify for the Regenerative Medicine Advanced Therapy designation, which combines the benefits of other expedited programs. The FDA also offers resources to aid in clinical trial design and facilitate communication throughout the development process.

Several gene therapies are currently in clinical development for PMDs, with a focus on Leber's hereditary optic neuropathy (LHON). LHON is a genetic eye disease, and the use of gene therapy has shown promising results in clinical trials. The FDA's Center for Drug Evaluation and Research is actively working to accelerate the development of cures for rare diseases like LHON. Additionally, companies like GenSight Biologics and Neurophth are making significant progress in the development of gene therapies for LHON, with Phase III clinical trials underway.

Exciting advancements are being made in the field of gene therapy, particularly for LHON, and the potential for a cure is within reach. Learn more about how the FDA's expedited programs and incentives, along with the efforts of innovative companies, are paving the way for the development of effective gene therapies.

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