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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 late-onset Pompe disease (LOPD), a rare inherited disorder. It is designed to deliver the GAA gene to address the underlying enzyme deficiency. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
AB-1009 is a gene therapy that delivers the GAA gene, which encodes the enzyme acid alpha-glucosidase. In late-onset Pompe disease, mutations in this gene lead to enzyme deficiency and glycogen accumulation. By introducing a functional GAA gene, AB-1009 aims to restore enzyme activity and reduce disease pathology.
AB-1009 is being developed by Bayer AG, a multinational pharmaceutical company. Bayer's stock is traded over-the-counter under the ticker BAYRY. The company is conducting clinical trials to evaluate the safety, tolerability, and efficacy of this gene therapy in patients with late-onset Pompe disease.
AB-1009 is in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory agencies. The ongoing Phase 1 trial is recruiting participants to assess the safety, tolerability, and efficacy of the therapy in adults with late-onset Pompe disease.
AB-1009 is being studied in a Phase 1 clinical trial registered as NCT07282847, titled "A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)." The trial is recruiting 12 participants in the United States and is currently active.
Yes, AB-1009 is also referred to as AB-1009 (GAA Gene). The alternative name highlights that the therapy involves the GAA gene, which is central to its mechanism of action. Both names refer to the same investigational gene therapy for late-onset Pompe disease.