May 11, 2026

Durham, N.C. – May 11, 2026 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first participant has been dosed with investigational gene therapy AB-1009 as part of PROGRESS-GT LOPD, a Phase 1/Phase 2 clinical trial in participants with late-onset Pompe disease (LOPD). LOPD is a rare, progressive, debilitating genetic disorder that is estimated to affect at least 5,000 to 10,000 people worldwide.1 While there are multiple approved enzyme replacement therapies (ERTs) with recombinant human acid alpha-glucosidase (rhGAA), there remains a strong level of unmet medical need, as some individuals receiving ERT may experience a decrease in clinical response over time, which can contribute to overall increased treatment demands.2-5 PROGRESS-GT LOPD (NCT07282847) is evaluating the safety, tolerability, and efficacy of AB-1009 in adult participants with LOPD.6 PROGRESS-GT LOPD is estimated to enroll 12 participants across the United States.6 “Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical,” said Tahseen Mozaffar, MD, Director of the UCI Health ALS & Neuromuscular Center, and Principal Investigator, AB-1009 Clinical Trial Program. “We look forward to advancing the PROGRESS-GT LOPD trial and generating data with the potential to help inform the future of care in the Pompe community.” AB-1009 was granted United States Food and Drug Administration (FDA) Fast Track and Orphan Drug designations.7 The FDA Fast Track process is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill unmet medical needs.8 The purpose of the process is to get important new therapeutics to patients earlier.8 Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the clinical development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review. Orphan Drug Designation provides orphan status to drugs and biologics for rare diseases that meet certain criteria and potentially gives a company exclusive marketing rights for a seven-year period, along with other benefits.9 Additionally, in January, AskBio announced that the FDA accepted the investigational new drug application for AB-1009.7 “Reaching this milestone reflects the momentum behind our gene therapy platform and our commitment to working toward improving quality of life for people with rare diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “The initiation of PROGRESS-GT LOPD marks an important step in the development of AB-1009 and demonstrates our commitment to moving our clinical program forward with determination, to investigate a potential new treatment approach for people living with Pompe disease.” AB-1009 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated.  About Pompe Disease Pompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA).2 Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which ...read the news