HG-001

HG-001 is an investigational gene replacement therapy being developed by HaliGene for the treatment of congenital megaconial muscular dystrophy (MDCMC), an ultra-rare, progressive neuromuscular disorder. MDCMC is caused by loss-of-function mutations in the choline kinase beta (CHKB) gene, which leads to impaired mitochondrial function, severe cellular energy disruption, and muscle-cell integrity loss. HG-001 is designed to deliver a functional copy of the CHKB gene to restore choline kinase beta enzyme activity, thereby addressing the underlying genetic cause of the disease and improving mitochondrial and muscle function. The program is currently in preclinical development.