UniQure moved a major regulatory step forward in Huntington’s disease by submitting applications to the FDA and the UK’s MHRA for AMT-130, a gene therapy the company says could become the first to treat the disease’s underlying cause. UniQure is requesting priority review from FDA, targeting a decision in roughly eight months, and it also completed a UK submission. The filing is based on previously reported three-year data indicating AMT-130 could significantly slow disease progression. The program follows a year of regulatory and ethical scrutiny over UniQure’s control strategy, including FDA requests for a new double-blind trial with a sham surgery control group. The submission underscores how gene-therapy approvals can hinge not only on efficacy readouts, but also on trial design details for surgical therapies where appropriate controls are difficult to implement.