VGM-R02b

VGM-R02b is an investigational gene replacement therapy being developed for the treatment of glutaric acidemia type I (GA-I), a rare inherited metabolic disorder. The therapy works by delivering a functional copy of the GCDH gene to patients' cells using an adeno-associated virus serotype 9 (AAV9) vector, thereby restoring production of the glutaryl-CoA dehydrogenase enzyme. This restoration enables proper metabolism of lysine, hydroxylysine, and tryptophan, preventing accumulation of toxic metabolites and potentially improving clinical outcomes in affected individuals. The drug is currently in Phase 1 clinical trials in pediatric patients with genetically confirmed GA-I[2][4][5][6].