uniQure gene therapy gets FDA breakthrough status for Huntington’s disease

The U.S. FDA has granted breakthrough therapy designation to Amsterdam-based uniQure’s gene therapy candidate, AMT-130, for the treatment of Huntington’s disease.

The designation is supported by clinical data from ongoing phase 1/2 trials. In July 2024, uniQure presented interim data at 24 months that showed dose-dependent slowing of Huntington’s disease progression of treated patients compared to a propensity-weighted natural history. To date, a total of 45 patients have received AMT-130.

AMT-130 consists of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging the company’s proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein.

Huntington’s disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. 

AMT-130 has also been granted FDA regenerative medicine advanced therapy (RMAT), orphan drug and fast track designations.