MYO-301

MYO-301 is an **adeno-associated virus (AAV)-based gene therapy** designed to treat **Limb-Girdle Muscular Dystrophy type 2L (LGMD2L)**, which is caused by mutations in the ANO5 (anoctamin 5) gene. The therapy works by delivering a functional copy of the ANO5 gene to muscle cells, thereby enabling these cells to produce the critical anoctamin 5 protein that patients with LGMD2L lack. This has the potential to improve muscle function and slow or halt disease progression. The therapy is administered by intravenous injection, and includes a muscle-specific promoter to ensure the gene is activated predominantly in muscle cells. MYO-301 was originally developed by Nationwide Children's Hospital and further developed by Myonexus Therapeutics. Sarepta Therapeutics acquired Myonexus and continues its development. This therapy is in preclinical or early clinical stages and has not yet been approved for use.