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Alnylam has reported initial P-Ib trial data of brezosiran (ALN-HTT02; intrathecal RNAi therapeutic) in 48 adults with HD-ISS Stage 2 or early Stage 3 Huntington’s disease across 5 single-dose cohorts, presented at EHDN 2026
The highest dose showed a mean CSF mHTT reduction of 60-64% at M3 (maximum individual reduction: 83%), with durability supporting potential twice-yearly dosing & no dose-dependent rise in CSF inflammatory markers, NfL or ventricular volume
A placebo-controlled pivotal study is planned for 2027; brezosiran targets exon 1 of HTT mRNA & is being developed with Regeneron
Ref: Business Wire | Image: Alnylam | Press Release
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