uniQure is developing AMT-130 gene therapy to treat Huntington's disease, which delivers microRNAs to the patient's brain to silence the mutated HTT gene and reduce production of the defective huntingtin protein.[1][5] The therapy is being tested at two dose levels in phase 1/2 trials in the US (NCT04120493) and Europe (NCT05243017).[1] Interim data show that AMT-130 slows disease progression and reduces levels of mutated huntingtin in adults with early-stage Huntington's disease.[1] The study involved 29 patients, and those on the higher dose had up to a 75 percent reduction in symptoms over three years.[2][4][5] The primary goal of the studies is to compare three-year changes on the Unified Huntington's Disease Rating Scale between high-dose patients and controls.[1] UniQure plans to file for AMT-130 approval with the FDA in early 2026 and has held a Type A meeting with the agency to discuss the data.[1][3][6] The FDA has acknowledged a significant unmet need for a treatment modifying Huntington's disease.[3][6]