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SRP-9005
- Development stage
- Discontinued
- Lead developer
- Sarepta Therapeutics
- Modality
- Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
SRP-9005 is an investigational gene therapy developed for the treatment of limb-girdle muscular dystrophy type 2C (LGMD2C), also known as LGMDR5 or gamma-sarcoglycanopathy. It consists of a recombinant adeno-associated virus serotype rh74 (rAAVrh74) vector containing a codon-optimized human SGCG transgene, which encodes the gamma-sarcoglycan protein. The therapy is designed to deliver a healthy copy of the SGCG gene to muscle cells, including cardiac muscle, aiming to restore functional gamma-sarcoglycan protein expression and improve muscle function. Preclinical studies in mouse models have shown successful delivery and expression of the transgene in targeted muscles, reduction in disease biomarkers such as CPK levels, restoration of muscle histopathology, and improved ambulation. Clinical development is ongoing with first-in-human trials initiated in 2025[1][2][3][4][5][6].
- Other names
- rAAVrh74.MHCK7.SGCGrAAVrh-74.MHCK7.SGCGrAAVrh 74.MHCK7.SGCG
02
Targets
SGCG (Gamma-sarcoglycan)
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