RNAi Therapeutic Shows Positive Phase 1b Results for Huntington's Disease
1 report, 1 independent
Updated Oct 23
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What happened
Initial Phase 1b results for an investigational RNAi therapeutic for Huntington's disease were presented at the EHDN Clinical Research Congress 2026 in Krakow, Poland. The study showed robust, dose-dependent reductions of mutant huntingtin protein in cerebrospinal fluid, with a mean reduction of 60 to 64% at month 3 in the highest dose group evaluated to date. A placebo-controlled pivotal study is planned for 2027, pending regulatory alignment.
From finanznachrichten.de
Why it matters
The positive initial findings for the RNAi therapeutic could influence investment and research funding within the gene therapy and pharmaceutical industry. Capella participated in the congress where the results were shared.
From finanznachrichten.de