AskBio

AskBio is a pioneering gene therapy company with a core focus on AAV vector technology and a clinical pipeline targeting rare genetic disorders. A key recent milestone is the FDA acceptance of its IND for AB-1009 for late-onset Pompe disease, advancing it to Phase 1/2 trials with Fast Track and Orphan Drug designations. As a subsidiary of Bayer, it operates with strategic independence while leveraging the resources of a pharmaceutical giant to progress its pipeline from research to clinic.

Proprietary AAV (adeno-associated virus) vector design, engineering, and manufacturing platform, including the Pro10™ cell line for scalable production.

AskBio competes in the rapidly advancing gene therapy space, facing competition from other biotechs and large pharma companies developing AAV therapies for neuromuscular and metabolic diseases. In Pompe disease, it competes against established enzyme replacement therapies (Sanofi, Amicus) and other investigational gene therapies.