23 Oct, 2026 Initial Phase 1b Data for Brezosiran (ALN-HTT02) in Huntington’s Disease Presented at the European Huntington’s Disease Network (EHDN) Clinical Research Congress 2026

Initial Phase 1b data for brezosiran, an investigational RNAi therapeutic in development for Huntington’s disease, were presented at the European Huntington’s Disease Network (EHDN) Clinical Research Congress 2026.

Oral Presentation: Tabrizi, et al. "Interim Data From a Phase 1b Study of ALN-HTT02, an Investigational RNA Interference Therapeutic Targeting Exon 1 of Huntingtin for HTT-lowering in People with Huntington’s Disease"

Poster: Tabrizi, et al. "Interim Data From a Phase 1b Study of ALN-HTT02, an Investigational RNA Interference Therapeutic Targeting Exon 1 of Huntingtin for HTT-lowering in People with Huntington’s Disease"

Participants on single ascending intrathecal doses of brezosiran experienced robust, dose-dependent reductions of mutant huntingtin protein in cerebrospinal fluid, with an encouraging safety and tolerability profile observed to date. An oral presentation and poster highlighted initial results from the ongoing Phase 1b study and brezosiran’s differentiated approach of targeting exon 1 of huntingtin.