AskBio has announced that the first participant has been dosed in a phase 1/2 clinical trial evaluating AB-1009, an investigational gene therapy for adults with late-onset Pompe disease (LOPD). AB-1009 is designed to address the underlying cause of Pompe disease by enabling the body to continuously produce acid alpha-glucosidase (GAA).
Pompe disease is caused by deficiency of GAA, resulting in glycogen accumulation within cells—particularly in skeletal and respiratory muscles. While enzyme replacement therapies (ERTs) have improved outcomes for many individuals living with Pompe disease, treatment burden remains significant and some patients continue to experience disease progression despite therapy.
AB-1009 is an investigational adeno-associated virus (AAV)-based gene therapy intended to provide a one-time treatment that enables sustained production of GAA. Unlike ERT, which requires ongoing infusions of recombinant enzyme, gene therapy aims to provide long-term enzyme production after a single administration. This approach has the potential to reduce treatment burden while addressing the underlying enzyme deficiency that drives disease progression.
The ongoing phase 1/2 study, known as PROGRESS-GT LOPD, is an open-label clinical trial evaluating the safety, tolerability, and preliminary efficacy of AB-1009 in adults with LOPD. Approximately 12 participants are expected to be enrolled across sites in the United States. As additional participants are enrolled and data emerge, the study may provide important insight into whether gene therapy can become a viable long-term treatment strategy for people living with LOPD.
Source
Clinical trial: https://clinicaltrials.gov/study/NCT07282847