Overview
- Researchers at UCL working with uniQure followed 29 patients for 36 months in a phase 1/2 study, comparing outcomes with untreated individuals.
- AMT‑130 is a one‑time gene therapy delivered by a viral vector into the brain’s striatum through a complex neurosurgical procedure lasting 12 to 18 hours.
- Investigators describe the approach as silencing production of toxic huntingtin protein, aiming to preserve daily function for people with Huntington’s disease.
- No serious adverse events have been reported so far, though clinicians urge caution over durability, long‑term safety, cost, and access.
- uniQure says it plans to seek accelerated FDA approval in early 2026, with subsequent filings expected in the UK and Europe, after prior approaches in the field faced setbacks such as a halted 2021 spinal injection trial.