Drugs / AMT-130
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | UK (MHRA) | — | Huntington disease | 2026-09-02 | “The Company also announced that its Marketing Authorisation Application (MAA) for ifezuntirgene inilparvovec has been submitted to the United Kingdom’s ( U.K .) Medicines and...” gcs-web.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05243017 | Oct 2021 → Mar 2029 expected | Huntington disease | UniQure Biopharma B.V. | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT04120493 | Sep 2019 → Jun 2029 expected | Huntington disease | UniQure Biopharma B.V. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 34
| Date | Issuer | Release |
|---|---|---|
| 2026-09-02 | UniQure Biopharma B.V. | Regulatory uniQure Announces Submission of Biologics License Application for Ifezuntirgene Inilparvovec (AMT-130) in Huntington’s Disease gcs-web.com ↗ |
| 2026-06-17 | UniQure Biopharma B.V. | Regulatory uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease gcs-web.com ↗
FDA communicated that the 3-year analysis from the Phase I/II study would be acceptable as the primary basis of a Biologics License Application (BLA) for the accelerated approval of AMT-130 in Huntington’s disease. |
| 2026-03-31 | Hopstem Biotechnology Inc. | Regulatory 2026【三月精选】领域最新进展 hopstem.com ↗
FDA明确该疗法 I/II 期研究与外部对照的数据不足以作为上市申请的有效性主要证据 |
| 2026-03-02 | UniQure Biopharma B.V. | Regulatory uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease gcs-web.com ↗
The FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. |
| 2025-12-04 | UniQure Biopharma B.V. | Regulatory uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease gcs-web.com ↗
In the final meeting minutes, and consistent with uniQure’s November 3, 2025 press release, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission. |
| 2025-11-03 | UniQure Biopharma B.V. | Regulatory uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease gcs-web.com ↗ |
| 2025-09-24 | UniQure Biopharma B.V. | Results uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease gcs-web.com ↗ |
| 2025-04-17 | UniQure Biopharma B.V. | Regulatory uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington’s Disease gcs-web.com ↗ |
| 2024-12-10 | UniQure Biopharma B.V. | Regulatory uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington’s 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 that the company reached agreement with the U.S. Food and Drug Administration (FDA) on key elements of an Accelerated Approval pathway for AMT-130. |
| 2024-07-09 | UniQure Biopharma B.V. | Results uniQure Announces Positive Interim Data Update Demonstrating Slowing of Disease Progression in Phase I/II Trials of AMT-130 for Huntington’s 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 interim data including up to 24 months of follow-up data from 29 treated patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington’s disease. |
| 2024-06-03 | UniQure Biopharma B.V. | Regulatory uniQure Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for Investigational Gene Therapy AMT-130 in Huntington’s Disease gcs-web.com ↗ |
| 2023-12-19 | UniQure Biopharma B.V. | Results uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington’s 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 interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington’s disease. |
| 2023-06-21 | UniQure Biopharma B.V. | Results uniQure Announces Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s 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 promising interim data, including up to 24 months of follow-up, from 26 patients enrolled in the ongoing U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. |
| 2022-08-08 | UniQure Biopharma B.V. | Results uniQure Announces Second Quarter 2022 Financial Results and Highlights Recent Company Progress gcs-web.com ↗
Announced 12-month data on the lower-dose cohort of AMT-130 in Huntington’s disease showed the investigative gene therapy was generally well tolerated at this dose with a mean reduction of 53.8% of mutant Huntingtin protein (mHTT) observed in cerebral spinal fluid (CSF) |
| 2022-06-23 | UniQure Biopharma B.V. | Results uniQure Announces Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s 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 safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease. |
| 2021-12-16 | UniQure Biopharma B.V. | Results uniQure Announces Clinical Update on First Patients in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease gcs-web.com ↗
Neurofilament light chain, a key biomarker of injury in the brain, trended downward and returned to baseline levels in the treated patients, and structural MRI data are consistent with the safety profile of AMT-130. |
| 2021-11-02 | UniQure Biopharma B.V. | Results uniQure Announces Latest Positive Recommendation from Data Safety Monitoring Board in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease gcs-web.com ↗
With the positive recommendation, the final 12 patients in this second cohort are now cleared for enrollment. |
| 2021-05-27 | UniQure Biopharma B.V. | Results uniQure Announces Positive Recommendation to Advance Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease gcs-web.com ↗
The DSMB recommended continued dosing in the study, and uniQure will now begin to enroll patients in the higher-dose cohort of the trial. |
| 2021-04-08 | UniQure Biopharma B.V. | Results uniQure Announces Publications of Preclinical Data for AMT-130 in Huntington’s Disease Showing Safety of Administration in NHPs and Widespread Long-Term HTT-Lowering in the Brain gcs-web.com ↗
The publications show the safety and efficacy of AMT-130 in the deep brain structures of a large animal model and outline a promising novel efficacy biomarker for AMT-130. |
| 2021-02-08 | UniQure Biopharma B.V. | Results uniQure Announces Positive Recommendation from Data Safety Monitoring Board of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease gcs-web.com ↗
No significant safety concerns were noted to prevent further dosing, and the final six patients in the first cohort are now cleared for enrollment. |
| 2020-09-25 | UniQure Biopharma B.V. | Results uniQure Announces Recommendations from Data Safety Monitoring Board of Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease gcs-web.com ↗
No significant safety concerns were noted to prevent further dosing, and the next two patients are now cleared for enrollment in the study. |
| 2020-02-27 | UniQure Biopharma B.V. | Results uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI’s 15th Annual Huntington’s Disease Therapeutics Conference gcs-web.com ↗
Collectively, these findings represent a robust package of new preclinical data to better inform how researchers and clinicians pursue a much-needed treatment for this devastating disease. |
| 2019-12-19 | UniQure Biopharma B.V. | Results uniQure Announces Publication of Preclinical Data for AMT-130 in Huntington’s Disease Supporting Non-Selective HTT-Lowering Approach gcs-web.com ↗
A dose-dependent suppression of full length HTT (both wild-type and mutant) was observed 7 months after a single injection of AAV5-miHTT, with reductions of 92% in the striatum and 64% in the frontal cortex. |
| 2019-05-07 | UniQure Biopharma B.V. | Results uniQure Announces Featured Presentations of New Data on Spinocerebellar Ataxia Type 3 at the 2019 American Academy of Neurology Annual Meeting gcs-web.com ↗
One presentation included findings from magnetic resonance imaging volumetric analysis in twenty early manifest Huntington’s disease patients to determine the safety of delivering gene therapy to the striatum, or deep structures of the brain. |
| 2019-04-08 | UniQure Biopharma B.V. | Regulatory uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease gcs-web.com ↗ |
| 2019-02-27 | UniQure Biopharma B.V. | Results uniQure Presents New Preclinical Data on AMT-130 at the CHDI’s 14th Annual Huntington’s Disease Therapeutics Conference gcs-web.com ↗
At 3 months after administration, significant levels of vector were present in the treated areas resulting in robust expression of microRNA and a significant knock down of the mutant huntingtin protein (mHTT). |
| 2019-01-22 | UniQure Biopharma B.V. | Regulatory uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-130 in Huntington’s Disease gcs-web.com ↗ |
| 2018-04-25 | UniQure Biopharma B.V. | Results uniQure Delivers Oral Presentation on Broad Set of Preclinical Data on AMT-130 in Huntington's Disease at the 2018 American Academy of Neurology Annual Meeting gcs-web.com ↗ |
| 2018-01-22 | UniQure Biopharma B.V. | Regulatory uniQure Receives Orphan Medicinal Product Designation in Europe for AMT-130 in Huntington's disease gcs-web.com ↗
AMT-130, its proprietary gene therapy candidate for Huntington's disease, has received an Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency. |
| 2017-10-18 | UniQure Biopharma B.V. | Results uniQure Presents New Preclinical Data on AMT-130 in Huntington's Disease at the ESGCT 25th Anniversary Congress in Berlin gcs-web.com ↗
Data from the study demonstrate that following administration of AMT-130 in Huntington's disease mouse models, significant improvements in both motor-coordination and survival were observed, as well as a dose-dependent, sustained reduction in huntingtin protein. |
| 2017-10-06 | UniQure Biopharma B.V. | Regulatory uniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntington's disease gcs-web.com ↗ |
| 2017-04-26 | UniQure Biopharma B.V. | Results uniQure Presents New Preclinical Data on AMT-130 in Huntington's Disease at CHDI's 12th Annual Huntington's Disease Therapeutics Conference gcs-web.com ↗
Data from the study demonstrate widespread and effective AAV5 vector distribution and extensive silencing of the human mutant huntingtin gene (HTT) in minipigs, among the largest HD animal models available for testing. |
| 2017-04-04 | UniQure Biopharma B.V. | Results uniQure Publishes Data Demonstrating Wide Distribution of AAV5 in the Central Nervous System gcs-web.com ↗
Varying doses of AAV5 achieved predictable transduction of connected areas of the brain. |
| 2016-03-22 | UniQure Biopharma B.V. | Results uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington's Disease gcs-web.com ↗
uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced the publication of preclinical data supporting its proprietary Huntington's disease gene therapy program, AMT-130. |
All press releases naming this drug 44 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | AMT 130 | “A Phase Ib/II Randomized, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin...” NCT05243017 ↗2“AMT-130 is an investigational, single administration gene therapy intended to modify the disease course for HD.” NCT04120493 ↗ “Cohort 3 participants will receive either high or low dose AMT-130.” NCT05243017 ↗ |
| Known as | rAAV5-miHTT | “Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington's Disease” NCT04120493 ↗ |
| Action | Inhibit | “AMT-130 lowers huntingtin protein” NCT04120493 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy” NCT05243017 ↗ |
| Route | Other | “One time MRI-guided stereotaxic infusion of rAAV5-miHTT into the brain” NCT04120493 ↗ |
| Target | HTT | “Total Huntingtin Gene (HTT) Lowering Therapy” NCT05243017 ↗ |