HaliGene Therapeutics

HaliGene Therapeutics is a private gene therapy biotechnology company focused on ultra-rare neuromuscular and metabolic diseases. The company is developing adeno-associated virus gene replacement therapies designed for disorders affecting skeletal muscle, cardiac function, and the central nervous system. Its lead program, HG-001, targets CHKB-associated congenital megaconial muscular dystrophy, a severe inherited disorder caused by loss-of-function variants in CHKB. HaliGene presents itself as a collaborative, patient-centered rare-disease company that combines vector engineering, translational biology, natural-history research, and partnership with clinicians and patient advocates to advance development.