Koji Kabuto
The U.S. Food and Drug Administration (FDA) has granted platform technology designation to the viral vector used in SRP-9003, Sarepta Therapeutics’ investigational gene therapy for the treatment of Limb Girdle Muscular Dystrophy Type 2E/R4. This designation is a significant milestone for Sarepta, as it recognizes the potential of the viral vector technology to treat a range of genetic disorders beyond just LGMD.
SRP-9003 is a gene therapy that aims to address the underlying genetic cause of LGMD Type 2E/R4, a rare and debilitating muscle-wasting disease. By delivering a functional copy of the affected gene to muscle cells, SRP-9003 has the potential to improve muscle function and quality of life for patients with this condition.
The platform technology designation from the FDA acknowledges the versatility and potential of the viral vector used in SRP-9003. This designation could streamline the regulatory review process for future gene therapy products utilizing the same viral vector technology, making it easier for Sarepta to develop and bring new treatments to market.
Sarepta Therapeutics is a leader in the field of gene therapy, with a strong pipeline of investigational treatments for a variety of genetic disorders. The company is committed to advancing innovative therapies that have the potential to transform the lives of patients with rare and devastating diseases.
Overall, the FDA’s platform technology designation for the viral vector used in SRP-9003 is a positive development for Sarepta Therapeutics and for the field of gene therapy as a whole. It paves the way for more efficient development and regulatory approval of future treatments, bringing hope to patients and families affected by rare genetic disorders.
Source: Sarepta Therapeutics
