Post by Spencer Knight

𝗕𝗿𝗲𝗮𝗸𝘁𝗵𝗿𝗼𝘂𝗴𝗵 𝗶𝗻 𝗡𝗲𝘂𝗿𝗼𝗹𝗼𝗴𝘆: Gene Therapy Slows Huntington’s Disease👇 Jack May-Davis, 30, always knew he carried the gene for Huntington’s disease, a devastating inherited neurodegenerative condition that claimed the lives of both his father and grandmother. Determined to change the course of his future, Jack enrolled in a pioneering clinical trial led by Professors Sarah Tabrizi and Ed Wild at UCL. The treatment, UniQure's AMT-130, uses gene therapy delivered directly into the brain to reduce production of the toxic huntingtin protein believed to drive disease progression. NOW, in a major development for the Huntington’s community, the FDA has reversed its previous position and cleared UniQure to submit AMT-130 for BLA in Q3 2026 accelerated approval in the US. Just months ago, the agency indicated the available data would not support a filing. Following further discussions, the FDA has now agreed that three-year Phase 1/2 data can form the primary basis of an accelerated approval application. For patients, families, clinicians, and researchers who have spent decades searching for a disease-modifying treatment, this is an important step forward. Congratulations to Jack, the trial investigators, UniQure, and the wider Huntington’s disease community on reaching this milestone. #genetherapy #neurology #neuroscience #CGTweekly