Bayer Backs AskBio Gene Therapy Push in Pompe Disease: Early Trial Update and Investor Takeaways

Bayer AG (BAYRY) announced an update on their ongoing clinical study.

Study Overview

The PROGRESS-GT LOPD trial is an early-stage clinical study sponsored by AskBio, with Bayer as a collaborator, to test a new gene therapy called AB-1009 in adults with late-onset Pompe disease. The official title describes it as a single-arm, open-label, dose-escalation study assessing the safety, tolerability, and early effectiveness of a one-time intravenous infusion of AB-1009. This program is important because it targets a rare, chronic muscle disease with high treatment burden, and it fits Bayer’s broader push into genetic medicines that could drive long-term growth if successful.

Intervention/Treatment

The treatment being tested is AB-1009, a gene therapy given as a single intravenous infusion. It is designed to deliver a working copy of the GAA gene to help the body produce an enzyme that people with late-onset Pompe disease lack. The aim is to reduce the need for ongoing enzyme replacement and to improve muscle function and quality of life with a one-time treatment.

Study Design

This is an interventional Phase 1/2 study with no control group. All enrolled patients receive AB-1009, but at different dose levels in sequence, starting with a lower dose and then moving to a higher one once safety is checked. The study is non-randomized and not blinded, meaning doctors and patients know the treatment and dosage being given. The main goal at this stage is to understand safety and side effects, while also collecting early signals of benefit.

Study Timeline

The study was first submitted in late November 2025, signaling that the program is at an early clinical stage and not yet recruiting participants. The most recent update was filed on December 17, 2025, which shows active preparation and regulatory engagement. Primary completion and final completion dates have not yet been specified, reflecting the exploratory nature of a first-in-human gene therapy trial where timelines often adjust as safety and dose decisions evolve.

Market Implications

For investors in Bayer, this study update highlights continued investment in high-risk, high-reward gene therapy platforms through AskBio. While the trial is only in Phase 1/2 and years away from commercialization, a successful path could broaden Bayer’s rare disease footprint and support a higher long-term growth narrative. In the near term, the impact on the share price is likely modest, as early-stage gene therapy data are uncertain and will take time. However, visible progress may strengthen sentiment around Bayer’s innovation pipeline, especially against competitors in Pompe disease who rely on chronic enzyme replacement or more traditional approaches. The update also reinforces Bayer’s positioning in a competitive gene therapy field, where partnerships and platform depth are seen as strategic value drivers by the market.

The study is ongoing in set-up and has been recently updated, with further details available on the ClinicalTrials.gov portal.

To learn more about BAYRY’s potential, visit the Bayer AG drug pipeline page.