U.S. FDA Grants Platform Technology Designation to the Viral Vector Used in SRP-9003, Sarepta’s Investigational Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophy Type 2E/R4 – Sarepta Therapeutics
In a groundbreaking development in the field of gene therapy, the U.S. Food and Drug Administration (FDA) has granted Platform Technology Designation…
In a groundbreaking development in the field of gene therapy, 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 significant as it recognizes the potential of the viral vector technology to be used in multiple gene therapy products beyond just SRP-9003. Sarepta Therapeutics is a leader in the field of genetic medicine, focusing on developing innovative therapies for rare neuromuscular diseases.
Limb Girdle Muscular Dystrophy Type 2E/R4 is a rare genetic disorder that causes progressive muscle weakness and atrophy. There is currently no cure for this debilitating condition, but gene therapy holds promise as a potential treatment option.
The viral vector used in SRP-9003 is designed to deliver a functional copy of the gene responsible for the production of a critical muscle protein that is lacking in patients with Limb Girdle Muscular Dystrophy Type 2E/R4. By restoring the production of this protein, gene therapy has the potential to improve muscle function and quality of life for patients with this condition.
Sarepta Therapeutics is committed to advancing the development of SRP-9003 and other gene therapy products to address the unmet medical needs of patients with rare genetic diseases. The Platform Technology Designation from the FDA is a significant milestone in this journey towards bringing innovative treatments to those in need.
Source: Sarepta Therapeutics
