A one-time gene therapy delivered via a surgical procedure directly into the brain, using an AAV5 viral vector to carry a microRNA construct that silences the huntingtin gene, reducing mutant huntingtin production at the source. Positive 36-month Phase 1/2 follow-up data showed slowed disease progression and reduced cerebrospinal fluid neurofilament light (NfL), a biomarker of nerve damage. uniQure met with the FDA to discuss a Biologics License Application (BLA) filing.
Each finding below is linked to its primary source. Confidence levels reflect the quality and quantity of available evidence — not CelluTarget's endorsement of any treatment.
High: Supported by multiple robust studies or regulatory approval
Moderate: Supported by limited controlled studies or consistent case series
Low: Based on case reports, expert opinion, or early-phase data only
Moderate ConfidenceSupported by limited controlled studies or consistent case series
Positive 36-month follow-up data from the Phase 1/2 trial showed that roughly 80% of patients who received AMT-130 had slowed disease progression, along with reductions in cerebrospinal fluid neurofilament light (NfL), a biomarker of nerve damage. Results are based on fewer than 30 participants with comparisons to external controls, so some caution is warranted given the small, non-randomized-controlled nature of the readout. uniQure met with the FDA in 2025 and aims to file for approval in early 2026.
Verified Jul 2026
Institutional Research
The First Domino Falls: AMT-130 Gene Therapy Slows Huntington's in Landmark Trial — HDBuzz