Fantastic update from uniQure about its Huntington's disease (HD) program. I'm impressed by the safety and durability of the treatment. These data seem to demonstrate that reducing expression of the huntingtin protein in neurons can slow down disease progression. ~ Pivotal study met primary endpoint; high-dose AMT-130 demonstrated statistically significant 75% disease slowing at 36 months as measured by cUHDRS compared to a propensity score-matched external control ~ ~ High-dose AMT-130 also demonstrated statistically significant slowing of disease progression as measured by TFC, a key secondary endpoint, and favorable trends across additional clinical measures ~ ~ Mean cerebrospinal fluid NfL levels were below baseline at 36 months ~ ~ AMT-130 continued to be generally well-tolerated with a manageable safety profile ~ ~ uniQure plans to submit a BLA in the first quarter of 2026, with anticipated U.S. launch later that year, pending approval ~ https://lnkd.in/eHpAfk-8
Thanks a lot Arno for sharing. New hope for HD patients, and great to see gene therapy delivering on its efficacy and safety promises. Kuddos to Uniqure!
Fantastic news for patients. Great data.
Such a wonderful news and a great validation of the platform.
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