uniQure AMT-130 shows dose-dependent clinical benefit in Huntington's; NfL below baseline at 30 months

uniQure N.V.

AMT-130 showed favorable cUHDRS difference of 0.39 (low-dose at 30 months) and 1.24 (high-dose at 18 months) vs natural history.
Mean CSF NfL low-dose cohort 6.6% below baseline at 30 months; high-dose cohort near baseline at 18 months.
AMT-130 generally well-tolerated; 4 AMT-130-related SAEs (CNS inflammation) in high-dose cohort managed with steroids.
Company plans regulatory interactions in Q1 2024 and next clinical update mid-2024.

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