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patidistrogene bexoparvovec
- Development stage
- Unknown
- Lead developer
- Sarepta Therapeutics
- Modality
- Gene Therapies
- Administration
- Intravenous, Intra-arterial, Intramuscular
01
Overview
Patidistrogene bexoparvovec is an investigational gene therapy designed to treat certain forms of limb-girdle muscular dystrophy (LGMD), specifically sarcoglycanopathies, which are caused by mutations in the SGCA gene encoding alpha-sarcoglycan. The therapy uses an adeno-associated virus (AAV) vector to deliver a functional copy of the SGCA gene to muscle cells, aiming to restore production of alpha-sarcoglycan protein and improve muscle function. It is being developed for neuromuscular and congenital disorders affecting skeletal muscle integrity[6]. The primary developer is Nationwide Children's Hospital, with further development by Myonexus Therapeutics and Sarepta Therapeutics[6].
- Other names
- MYO-101MYO101MYO 101
02
Targets
SGCA (Sarcoglycan Alpha Subunit)
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