actus-101

ACTUS-101 is an investigational gene therapy designed to treat Pompe disease, specifically late-onset Pompe disease (LOPD). It uses a recombinant adeno-associated virus vector (AAV2/8) to deliver a functional copy of the GAA gene to hepatocytes in the liver. This enables continuous production of acid alpha-glucosidase (GAA), the enzyme deficient in Pompe disease, thereby addressing the underlying cause by reducing glycogen accumulation in affected tissues. The therapy is administered as a single intravenous infusion and aims to provide a long-term alternative to bi-weekly enzyme replacement therapy (ERT). Early clinical data suggest that ACTUS-101 increases GAA levels and may allow patients to discontinue ERT[1][3][4][5][6][7][8].

Brand names

ACTUS-101ACTUS101ACTUS 101

Other names

Pompe Disease rAAV Gene Therapy - BayerACT-101ACT101ACT 101