AskBio, a wholly owned and independently operated subsidiary of Bayer AG, has announced that the first participant has been dosed with investigational gene therapy AB-1009 in the Phase 1/2 PROGRESS-GT LOPD clinical trial for late-onset Pompe disease (LOPD). The study is being conducted across the United States and will evaluate the safety, tolerability and efficacy of the therapy in adult participants living with the rare genetic disorder.

Late-onset Pompe disease is a progressive and debilitating inherited disorder caused by a deficiency of the enzyme acid alpha-glucosidase, leading to glycogen accumulation in cells and resulting in severe muscle weakness and respiratory complications. The disease is estimated to affect between 5,000 and 10,000 people worldwide. While enzyme replacement therapies are currently available, many patients continue to face unmet medical needs due to declining treatment response over time.

The PROGRESS-GT LOPD study is a non-randomized, single-arm, open-label, dose-escalation trial expected to enroll around 12 participants across the US. The trial is investigating AB-1009, an Adeno-Associated Virus (AAV)-based gene therapy designed to address the underlying genetic cause of Pompe disease by enabling sustained production of the deficient GAA enzyme.

Tahseen Mozaffar, Director of the UCI Health ALS and Neuromuscular Center and Principal Investigator for the AB-1009 clinical trial programme, said current treatment options may not fully address the long-term needs of patients and highlighted the importance of continued research into new therapeutic approaches for the Pompe disease community.

AB-1009 has already received Fast Track and Orphan Drug designations from the US Food and Drug Administration (FDA). The Fast Track designation is intended to accelerate the development and review of therapies targeting serious conditions with unmet medical needs, while Orphan Drug designation provides incentives for therapies developed for rare diseases.

Canwen Jiang, Chief Development Officer and Chief Medical Officer at AskBio, said the initiation of the PROGRESS-GT LOPD study marks an important milestone for the company’s gene therapy platform and reflects its commitment to advancing potential treatment options for people living with rare diseases.

The investigational therapy has not yet been approved by any regulatory authority, and its safety and efficacy continue to be evaluated through ongoing clinical research. AskBio also acknowledged early contributions from Genethon, Belief BioMed and Duke University to its Pompe disease programme.