DURHAM, N.C., May 11, 2026
AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has announced that the first participant has been dosed in the company’s Phase 1/Phase 2 PROGRESS-GT LOPD clinical trial evaluating investigational gene therapy AB-1009 for the treatment of late-onset Pompe disease (LOPD). The milestone marks an important advancement in the development of next-generation gene therapies targeting rare neuromuscular disorders and further strengthens AskBio’s growing clinical pipeline focused on genetic and metabolic diseases. The trial initiation also highlights increasing industry momentum behind adeno-associated virus (AAV)-based gene therapy technologies designed to address the underlying causes of inherited diseases rather than only managing symptoms.
AB-1009 Targets Underlying Cause of Pompe Disease
Pompe disease is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA), leading to harmful glycogen accumulation in muscle tissues and progressive muscle weakness. Late-onset Pompe disease can significantly impair respiratory and skeletal muscle function over time, creating substantial long-term disability for affected patients. While enzyme replacement therapies currently exist, many patients continue to experience declining clinical response and increasing treatment burden over time, leaving significant unmet medical need within the Pompe disease community.
AskBio’s investigational therapy AB-1009 is designed as an AAV-based gene therapy intended to address the disease’s underlying genetic defect by enabling sustained production of the deficient GAA enzyme in affected individuals. Researchers believe the therapy may potentially provide a longer-lasting therapeutic effect compared with conventional enzyme replacement approaches. The company stated that AB-1009 is being evaluated for its ability to improve enzyme production while targeting the root biological mechanism responsible for disease progression.
The PROGRESS-GT LOPD trial is a non-randomized, open-label, dose-escalation Phase 1/Phase 2 study evaluating the safety, tolerability, and efficacy of AB-1009 in adult patients with late-onset Pompe disease. The study is expected to enroll approximately 12 participants across the United States as recruitment continues throughout 2026.
Advertisement
FDA Designations Accelerate Gene Therapy Development
The clinical advancement of AB-1009 has already received important regulatory support from the U.S. Food and Drug Administration (FDA). The investigational therapy has been granted both Fast Track Designation and Orphan Drug Designation, reflecting the serious nature of Pompe disease and the urgent need for new therapeutic options. The FDA previously accepted AskBio’s investigational new drug (IND) application for AB-1009 earlier this year, enabling the clinical program to move forward into human studies.
Fast Track Designation is intended to accelerate the development and regulatory review of therapies targeting serious diseases with unmet medical need. Therapies receiving this designation may benefit from increased FDA interaction, accelerated approval opportunities, and priority review pathways. Meanwhile, Orphan Drug Designation provides incentives for rare disease therapies, including market exclusivity and development support benefits.
Clinical investigators involved in the study emphasized that ongoing innovation remains essential because currently available Pompe disease treatments may not adequately address long-term disease progression in all patients. Researchers noted that continued advancement in gene therapy technology could potentially reshape future treatment standards for inherited neuromuscular diseases.
AskBio Strengthens Rare Disease Gene Therapy Pipeline
The initiation of PROGRESS-GT LOPD further expands AskBio’s growing presence within the global gene therapy sector, where pharmaceutical and biotechnology companies continue investing heavily in genetic medicine platforms. AskBio’s broader pipeline includes investigational programs targeting cardiovascular diseases, Parkinson’s disease, muscular dystrophies, and neurological disorders, positioning the company as an important player in next-generation therapeutic development.
Industry analysts continue to view AAV-based gene therapies as one of the most promising areas in biotechnology because of their potential to provide durable therapeutic benefit through a single administration. As regulatory agencies increasingly support rare disease innovation, successful clinical progress in Pompe disease could significantly strengthen the future commercial and scientific value of AskBio’s gene therapy platform.
Source: AskBio press release
