Sarepta Garners Platform Technology Designation for Limb-Girdle Muscular Dystrophy Gene Therapy Vector
The platform technology designation is a new designation established by the FDA.
Louise Rodino-Klapac, PhD
Sarepta Therapeutics has received platform technology designation from the FDA for bidridistrogene xeboparvovec (also known as SRP-9003)'s rAAVrh74 viral vector.1
SRP-9003 is an investigational adeno-associated virus (AAV) vector-based gene therapy product in development for limb-girdle muscular dystrophy Type 2E (LGMD2E/R4, also known as beta sarcoglycanopathy). The platform technology designation is a new designation established by the FDA that is intended to allow companies developing a product that uses a well-understood technology that has been previously used in other programs to utilize data regarding that technology from the other programs in support of investigational new drug applications and biologic license applications. Notably, rAAVrh74 is also used in Sarepta’s delandistrogene moxeparvovec-rokl (marketed as Elevidys), an FDA-approved gene therapy intended to treat Duchenne muscular dystrophy (DMD).2
“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,” Louise Rodino-Klapac, PhD, the chief scientific officer and head of research & development at Sarepta, said in a statement.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.”
SRP-9003 is currently being evaluated in a phase 1/2 clinical trial (NCT03652259) and the phase 3 EMERGENE clinical trial (NCT06246513).3 Safety data from the phase 1/2study were recently presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 16-19 in Dallas, Texas.3 The findings, which showed a profile that was sustained over a 4-to-5-year follow-up, came from 6 patients who across 2 cohorts were treated with a one-time intravenous dose of either 1.85x1013 vg/kg (cohort 1; n = 3) or 7.41x1013 vg/kg (cohort 2; n = 3) of the gen therapy product. Over the 5-year period, there were 25 treatment-related treatment-emergent adverse events (TR-TEAEs), most of which were either mild (n = 13) or moderate (n = 10) in severity. TR-TEAEs observed within the first 90 days included vomiting and increased gamma-glutamyltransferase.
The EMERGENE study, which began screening activities in January 2024, is intended to support a BLA for SRP-9003.3,4 The trial, which is using commercially representative process gene therapy product material, is evaluating expression of beta-sarcoglycan, the disease-targeted protein, at 60 days posttreatment as its primary outcome measure. Additional end points include assessments of functional outcomes and safety, and the study includes a 6-month evaluation of natural history for each patient before treatment with the gene therapy.
“We are pleased to share our continued progress in advancing SRP-9003, our investigational gene therapy candidate for LGMD2E, a rare form of LGMD with no treatments beyond symptom management,” Rodino-Klapac said in a statement at the time screening activities for EMERGENE began.4 “Early results from the SRP-9003 clinical development program demonstrated significant protein expression at both 12-weeks and 2 years after treatment as well as functional benefits including slowing progression of this disease, improving mobility, and enhancing the quality of life for individuals living with LGMD2E. In addition to its importance for the LGMD2E community, EMERGENE will inform the clinical development of other programs for LGMD in Sarepta’s pipeline while serving as a pathfinder for viable regulatory pathways to support the development of gene therapies to treat ultra rare diseases.”
