VGN-R08b

VGN-R08b is an investigational gene therapy developed for the treatment of type II Gaucher disease, a severe and rapidly progressive neurovisceral lysosomal storage disorder caused by mutations in the GBA1 gene. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human GBA1 gene directly into the central nervous system via intracerebroventricular injection. This approach aims to restore beta-glucocerebrosidase enzyme activity in affected tissues, thereby reducing substrate accumulation and ameliorating disease symptoms. The drug is currently being evaluated in early phase 1 clinical trials for safety and tolerability in infants with type II Gaucher disease.