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AB-1009

Late-Onset Pompe Disease (LOPD)

Phase 1/Phase 2Active

Key Facts

Indication
Late-Onset Pompe Disease (LOPD)
Phase
Phase 1/Phase 2
Status
Active
Company

About 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.

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Therapeutic Areas