Drugs / AMT-191
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT06270316 | Jun 2024 → Dec 2027 expected | Fabry disease | UniQure Biopharma B.V. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2026-02-06 | UniQure Biopharma B.V. | Results uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease gcs-web.com ↗
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated preliminary safety and exploratory efficacy data from 11 patients in its Phase I/IIa trial of AMT-191, an investigational AAV gene therapy for the treatment of Fabry disease. |
| 2025-09-05 | UniQure Biopharma B.V. | Results uniQure Announces Initial AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease gcs-web.com ↗ |
| 2025-02-03 | UniQure Biopharma B.V. | Results uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease gcs-web.com ↗
The IDMC’s review identified no significant safety concerns and recommended proceeding with enrollment in the second cohort. |
| 2024-09-23 | UniQure Biopharma B.V. | Regulatory uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease gcs-web.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. |
| 2023-11-29 | UniQure Biopharma B.V. | Regulatory uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-191 Gene Therapy for Fabry Disease gcs-web.com ↗ |
| 2021-10-19 | UniQure Biopharma B.V. | Results uniQure Announces Presentations at the Annual Meeting of The European Society of Gene and Cell Therapy (ESGCT) gcs-web.com ↗
New preclinical data confirms high efficiency and cross correction of AMT-191 in a Fabry mouse model, with increased GLA-activity in the liver, kidney, heart, and brain and normalized (lyso-)Gb3 levels in main target organs. |
All press releases naming this drug 7 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AMT-191 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06270316 ↗ |
| Action | Restore | “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...” NCT06270316 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “a recombinant serotype 5 based adeno-associated viral vector (AMT-191)” NCT06270316 ↗ |
| Route | Intravenous | “AMT-191 is delivered via a single (one-time) intravenous (IV) infusion.” NCT06270316 ↗ |
| Target | GLA | “This recombinant AAV5-based vector contains a coding deoxyribonucleic acid (DNA) sequence for human α-galactosidase A.” NCT06270316 ↗ |