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UniQure Biopharma clinical trials

Source: ClinicalTrials.gov, U.S. National Library of Medicine — current trials in our USA and UK registers with UniQure Biopharma as lead sponsor. Synced 26 Sep 2026
5Current trials
2Recruiting now
0Drugs in these trials
2Trial registers
Maintained by Sreepriya Prasannan
MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist
Compiled from ClinicalTrials.gov and CTIS registry records · Last verified · Editorial standards
At a glance

UniQure Biopharma is the lead sponsor of 5 current clinical trials in our USA and UK registers. 2 are recruiting patients now. All registered phases are Phase 1/2. The most-studied condition is Huntington Disease.

ClinicalTrials.gov trials by status

  • Recruiting240%
  • Active, closed360%

Trials by phase

  • Phase 1/25100%
01

Clinical trials sponsored by UniQure Biopharma

5 on ClinicalTrials.gov
StudyPhaseStatusStartRegisters
AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
NCT06063850 · Mesial Temporal Lobe Epilepsy
Phase 1/2 Recruiting 2024-06 USA
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
NCT06270316 · ClinicalTrials.gov ↗ · Fabry Disease
Phase 1/2 Recruiting 2024-06 USA
Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease
NCT05243017 · ClinicalTrials.gov ↗ · Huntington Disease
Phase 1/2 Active, closed 2021-10 UK
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
NCT04120493 · ClinicalTrials.gov ↗ · Huntington's Disease
Phase 1/2 Active, closed 2019-09 USA
Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)
NCT06100276 · ClinicalTrials.gov ↗ · Amyotrophic Lateral Sclerosis
Phase 1/2 Active, closed 2024-08 USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

02

Conditions in UniQure Biopharma trials

5 conditions
Huntington Disease1Mesial Temporal Lobe Epilepsy1Huntington's Disease1Amyotrophic Lateral Sclerosis1Fabry Disease1
03

By trial register

RegisterTrialsRecruiting
Clinical trials in USA42
Clinical trials in UK10

A trial with sites in more than one country appears in each of those registers.

04

Other sponsors in Huntington Disease

SponsorTrials
BioSensics1

Sponsors with the most current trials registered for this condition.

05

Questions about UniQure Biopharma trials

How many clinical trials does UniQure Biopharma sponsor?

UniQure Biopharma is the lead sponsor of 5 current trials on ClinicalTrials.gov in our USA and UK registers, as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are UniQure Biopharma trials recruiting?

Yes. 2 of its 5 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

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How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.