Sarepta Gains Recognition from FDA for its Viral Vector Technology
As a result of the capability to be used across multiple therapeutic programs, the U.S. regulatory body has granted Sarepta’s AAVrh74 platform with platform technology designation.
The U.S. FDA has granted platform technology designation to Sarepta Therapeutics — a company focused on precision genetic medicine for rare diseases — for its viral vector, AAVrh74, that was used in a gene therapy aimed at treating limb-girdle muscular dystrophy (LGMD) type 2E/R4. The company announced the regulatory designation decision in a June 4, 2025 press release (1).
“This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,” said Louise Rodino-Klapac, Ph.D., Chief Scientific Officer and Head of Research & Development, Sarepta, in the company press release (1). “The designation underscores and reinforces the consistency of the data we have seen with this AAVrh74 in multiple clinical programs and is yet another example of Sarepta’s continued commitment to accelerating the development of potentially transformative treatments for patients with rare genetic diseases like LGMD type 2E/R4.”
To receive platform technology designation, a tool must be considered critical and capable of supporting rare disease therapy development — through improving efficiencies in drug development, manufacturing, and review processes. Other considerations for this designation are if the platform is incorporated into an approved drug and has the potential to be used in other drug products without any adverse effects on quality, manufacturing, or safety. Once a platform has been granted with the designation, it is possible for sponsors to use data previously obtained through the platform to support future investigational new drug applications, new drug applications, or biologic license applications.
Sarepta’s viral vector platform is being used across multiple clinical programs, including the gene therapies SRP-9003 for LGMD type 2E/R4, SRP-9004 for LGMD type 2D/R3, and SRP-9005 for LGMD type 2C/R5. In April 2025, the company released an update on these three clinical programs, which are in Phase III, Phase I, and first-in-human clinical trial stages, respectively (2).
“There are no disease-modifying treatments approved for patients with any subtype of limb-girdle muscular dystrophy, and the unmet medical need is significant. Following feedback from U.S. FDA, we are pleased to announce that screening can proceed in Sarepta’s first clinical study for a gene therapy for individuals with LGMD type 2C — the fourth LGMD program that Sarepta has advanced into the clinic,” said Rodino-Klapac, in a company press release (2). “In addition to our progress with SRP-9004 and SRP-9005, we remain on track to share data in the first half of this year from the EMERGENE study with SRP-9003. Our confidence in the potential for gene therapy to bring meaningful treatments to patients with rare, genetic based diseases remains high and the rapid progress across our LGMD pipeline is encouraging.”
References
Sarepta Therapeutics. 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. Press Release, June 4, 2025.
Sarepta Therapeutics. Sarepta Therapeutics Announces Pipeline Progress for Multiple Limb-Girdle Muscular Dystrophy Programs. Press Release, April 15, 2025.