uniQure Reports Trial Success for Experimental Huntington’s Disease Gene Therapy
uniQure has announced positive topline results from its Phase I/II study of AMT-130, an experimental gene therapy for Huntington’s disease. Patients receiving the high dose showed a 75% slowing of disease progression after 36 months compared to a matched external control group, as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), meeting the study’s primary endpoint. A key secondary endpoint was also achieved, with a 60% slowing of decline in Total Functional Capacity (TFC). The therapy showed favorable trends across additional motor and cognitive measures and was generally well tolerated with a manageable safety profile.
The study included 29 treated patients, with outcomes compared against data from the Enroll-HD natural history database. High-dose AMT-130 patients maintained cerebrospinal fluid neurofilament light protein (NfL) levels below baseline, a biomarker linked to neurodegeneration. Safety was described as manageable, with no new treatment-related serious adverse events observed since late 2022.
Following the readout, uniQure’s share price rose from $13.66 at Tuesday’s close to around $40 when markets opened on Wednesday, according to Fierce Biotech.
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Based on these results, uniQure plans to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration in the first quarter of 2026, aiming for a potential U.S. launch later that year if approved. AMT-130 already holds Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA.
Headquartered in the Netherlands and the U.S., uniQure is a gene therapy company that reportedly previously secured the first regulatory approval of a gene therapy for hemophilia B, HEMGENIX (etranacogene dezaparvovec). Its pipeline, enabled by the company’s technology platform, includes programs in Huntington’s disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe conditions.
Image credit: uniQure PipelineThe uniQure Platform
The company has built a modular adeno-associated virus (AAV) gene therapy platform designed for central nervous system and liver disorders. By reusing standard components like capsids and promoters, only the disease-specific genetic payload needs to be adapted for each new program, reducing development time and cost.
uniQure has also developed a set of enabling technologies:
- Smart AAV capsids designed to improve delivery to the brain and liver, including the ability to cross the blood–brain barrier.
- miQURE for silencing disease-causing genes through targeted degradation.
- LinQURE for delivering multiple microRNAs in a single vector, allowing modulation of several pathways at once.
- GoQURE for combined gene knockdown and replacement in the same vector.
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