Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
AMT-191 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Dose Ranging Cohort 1 | EXPERIMENTAL | - |
| Dose Ranging Cohort 2 | EXPERIMENTAL | - |
| Dose Ranging Cohort 3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| AMT-191 | DRUG | A recombinant serotype 5 based adeno-associated viral vector (AMT-191) for one-time intravenous (IV) administration will be investigated in this study. This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A. Delivery of AMT-191 to the systemic circulation is expected to result in a therapeutic effect by promoting the liver expression of the lysosomal enzyme GLA in plasma levels in patients with Fabry disease. |
Key Inclusion Criteria: * Male of age ≥ 18 years and ≤65 years * Confirmed clinical diagnosis of classic Fabry disease (FD) defined as: 1. Absent or minimal αGAL A enzyme activity \< 1% of mean normal measured in plasma regardless of variant status; OR 2. α-galactosidase A (GLA) pathogenic or ...
AMT-191 is an investigational gene therapy being developed for the treatment of classic Fabry disease, a rare inherited lysosomal storage disorder. It is designed to deliver a functional copy of the GLA gene to enable patients' cells to produce the missing enzyme on their own, addressing the root cause of the disease rather than just managing symptoms.
AMT-191 is designed to deliver a functional copy of the GLA gene, which encodes the enzyme alpha-galactosidase A (α-Gal A). In Fabry disease, mutations in GLA lead to deficient or absent α-Gal A activity, causing harmful substrate accumulation. By restoring enzyme production, AMT-191 aims to address the underlying cause of the disease.
AMT-191 is being developed by uniQure N.V., a gene therapy company traded on the Nasdaq under the ticker symbol QURE. uniQure specializes in adeno-associated virus (AAV)-based gene therapies for rare diseases, and AMT-191 is part of its pipeline for Fabry disease.
AMT-191 is currently in Phase 1 clinical development. The only trial to date is a Phase 1 study evaluating the safety, pharmacokinetics, pharmacodynamics, and exploratory efficacy of AMT-191 in patients with classic Fabry disease. The trial is actively recruiting participants.
AMT-191 is being evaluated in a single Phase 1 clinical trial, NCT06270316, titled "Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease." The study is currently recruiting up to 20 adult male participants in the United States. It is a controlled trial without randomization or blinding.
AMT-191 has received Orphan Drug designation from the FDA for the treatment of Fabry disease. This designation is granted to therapies intended for rare diseases affecting fewer than 200,000 people in the United States and provides incentives to support development.