📢 𝗝𝗨𝗦𝗧 𝗜𝗡: $QURE uniQure Reports Updated Huntington’s Gene Therapy Data Through 48 Months 👉 𝗞𝗲𝘆 𝗛𝗶𝗴𝗵𝗹𝗶𝗴𝗵𝘁𝘀: ➤ 𝗨𝗻𝗶𝗤𝘂𝗿𝗲 reports updated Phase I/II data for 𝗶𝗳𝗲𝘇𝘂𝗻𝘁𝗶𝗿𝗴𝗲𝗻𝗲 𝗶𝗻𝗶𝗹𝗽𝗮𝗿𝘃𝗼𝘃𝗲𝗰. ➤ At 36 months, high-dose patients showed 𝟴𝟬% slower cUHDRS progression. ➤ 36-month TFC progression slowed 𝟲𝟳% versus matched external controls. ➤ At 48 months, cUHDRS slowing was 𝟰𝟰% and not statistically significant. ➤ 48-month TFC showed 𝟲𝟭% slowing with nominal 𝗽=𝟬.𝟬𝟬𝟴. ➤ Sensitivity analysis showed 𝟱𝟰% cUHDRS and 𝟲𝟴% TFC slowing. ➤ Missing external-control data reached 𝟱𝟯% at the 48-month timepoint. ➤ Five high-dose patients, or 𝟭𝟳%, experienced treatment-related serious CNS inflammation. ➤ 𝗨𝗻𝗶𝗤𝘂𝗿𝗲 submitted an accelerated-approval 𝗕𝗟𝗔 to the FDA. ➤ FDA previously accepted 36-month data as the submission's primary basis. 👉 𝗪𝗵𝘆 𝗧𝗵𝗶𝘀 𝗠𝗮𝘁𝘁𝗲𝗿𝘀: ➤ Longer-term data provide additional evidence on potential durability in 𝗛𝘂𝗻𝘁𝗶𝗻𝗴𝘁𝗼𝗻’𝘀 disease. ➤ Significant 𝗧𝗙𝗖 slowing at 48 months supports continued functional-effect evaluation. ➤ However, 48-month 𝗰𝗨𝗛𝗗𝗥𝗦 results introduce uncertainty by missing statistical significance. ➤ High missing-control data complicates interpretation of the 𝟰𝟴-𝗺𝗼𝗻𝘁𝗵 comparison. ➤ FDA review of the 𝗕𝗟𝗔 is the key regulatory catalyst ahead.