A SIRT6 gene therapy trial in senior beagles hit its primary endpoint, lowering biological age and offering a test bed for human medicine. #longevity# #geroscience# #SIRT6# #genetherapy# #biologicalage#

$QURE is down ~70% after its Huntington’s gene therapy showed slower disease progression at 48 months but failed to reach statistical significance. Another function test still looked better but missing data and serious brain inflammation in 17% of high dose patients made the update much harder to trust.

📢 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.