JWK007

JWK007 is an investigational gene therapy designed for the treatment of Duchenne Muscular Dystrophy (DMD). It utilizes an adeno-associated virus serotype rh74 (AAVrh74) as a vector to deliver a micro-dystrophin (μDystrophin) gene construct. The μDystrophin protein encoded by this construct includes essential domains for promoting neuronal nitric oxide synthase (nNOS) activity and membrane binding, aiming to restore some of the lost function caused by mutations in the DMD gene. The therapy is administered as a single intravenous infusion and is currently being evaluated in pediatric patients with confirmed DMD due to frameshift or premature stop codon mutations between exons 18 and 58. The primary goal is to assess safety, tolerability, and preliminary efficacy[1][2][3][4][5][6].