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Also known as AB-1009 (GAA Gene)
AB-1009 · 1 trial · 3 indications
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | 1.0E13 vg/kg |
| Cohort 2 | EXPERIMENTAL | 1.5E13 vg/kg |
| Name | Type | Description |
|---|---|---|
| AB-1009 (GAA Gene) | GENETIC | A single intravenous infusion of AB-1009 |
Inclusion Criteria: 1. Participant must be ≥18 to ≤65 years of age at the time of signing the informed consent form. 2. Confirmed GAA enzyme deficiency from any tissue source and/or confirmed biallelic GAA gene mutations. 3. Undergone enzyme replacement treatment (ERT) (either alglucosidase alfa (L...
AB-1009 is an investigational gene therapy being developed for adults with late-onset Pompe disease, a rare inherited disorder. It is designed as a one-time treatment approach and is currently being studied in a Phase 1 clinical trial in the United States. It is not approved for any use.
AB-1009 is being developed by Bayer AG, which trades on the OTC market under the ticker BAYRY. Bayer is the sponsor of the ongoing Phase 1 clinical program evaluating the therapy in adult patients with late-onset Pompe disease.
AB-1009 is in Phase 1 clinical development. The single ongoing trial is currently recruiting adult participants with late-onset Pompe disease. No Phase 2 or Phase 3 studies have been initiated, and the therapy remains investigational.
AB-1009 is being evaluated in the PROGRESS-GT LOPD trial, registered as NCT07282847. The Phase 1 study is recruiting 12 adult participants with late-onset Pompe disease at sites in the United States. It is a controlled trial without randomization or double blinding.
Yes, AB-1009 (GAA Gene) refers to the same investigational therapy as AB-1009. The parenthetical describes the gene delivered by the therapy. Both names refer to the Bayer AG gene therapy candidate being studied in late-onset Pompe disease.
AB-1009 is a gene therapy that delivers the GAA gene, which encodes acid alpha-glucosidase. In late-onset Pompe disease, mutations in this gene lead to enzyme deficiency. The therapy aims to provide a functional copy of GAA to address the underlying cause of the disease.