Design of Phase 1 Study of ALN-HTT02 in Adult Patients with Huntington’s Disease Presented at the CHDI Foundation’s Annual Huntington’s Disease Therapeutics Conference

27 Feb, 2025 Design of Phase 1 Study of ALN-HTT02 in Adult Patients with Huntington’s Disease Presented at the CHDI Foundation’s Annual Huntington’s Disease Therapeutics Conference

The design of an ongoing Phase 1 study of ALN-HTT02—an investigational RNAi therapeutic targeting exon 1 of huntingtin (HTT) for Huntington’s disease—was presented at the CHDI Foundation’s 20th Annual Huntington’s Disease Therapeutics Conference in Palm Springs, CA. Nonclinical data supporting the tolerability of deep and sustained HTT-lowering in wild-type nonhuman primates after single and repeated intrathecal administration of ALN-HTT02—which employs Alnylam’s C16-siRNA CNS delivery platform—were also shared.

Sloan et al. “ALN-HTT02, an Investigational RNAi Therapeutic Targeting Exon 1 of HTT in Phase 1 Development for Huntington’s Disease”