zocaglusagene nuzaparvovec

Zocaglusagene nuzaparvovec (AT845) is an investigational adeno-associated virus (AAV) gene therapy designed to treat Pompe disease, a rare lysosomal storage disorder caused by mutations in the GAA gene. The therapy utilizes an AAV8 vector to deliver a functional copy of the human acid alpha-glucosidase (GAA) gene directly to muscle cells, utilizing a muscle-specific promoter to drive expression. By restoring GAA enzyme activity, the drug aims to clear accumulated glycogen and prevent further muscle damage, potentially offering a long-term alternative to lifelong enzyme replacement therapy. It is currently being evaluated in the Phase 1/2 FORTIS clinical trial.

Other names

zocaglusagene nuzaparvovec-ylasAAV8-hGAAAAV-8-hGAAAAV 8-hGAA