uniQure is developing AMT-130 gene therapy to treat Huntington's disease.[1] It initially planned to submit an application for approval (BLA) in the first quarter of 2026 based on data from Phase 1/2.[1][3] Data from September showed that the treatment slowed disease progression by 75% after three years in 12 of 17 high-dose patients compared to an external control group.[1][5] Experts called the results groundbreaking, and uniQure shares jumped 300%.[1] However, the FDA has changed its mind and no longer agrees that these externally reviewed data are sufficient for approval.[1][3][5] The timing of the filing of the BLA is therefore unclear.[1] UniQure has scheduled a Type A meeting with the FDA on January 9, 2026 to seek a path to expedited approval.[2][4] The Society highlights the great unmet need of Huntington's disease patients.[2]