13 Sep, 2024 Design of Phase 1 Study of ALN-HTT02 in Adult Patients with Huntington’s Disease Presented at the EHDN & Enroll-HD 2024 Congress
Posted at 08:50h
in Capella
The design of a Phase 1 study of ALN-HTT02—an investigational RNAi therapeutic targeting huntingtin (HTT) for Huntington’s disease—was presented at the EHDN & Enroll-HD 2024 Congress in Strasbourg, France. 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, a Novel C16-siRNA Conjugate for HTT-lowering in the CNS”
Cantley, et al. “Tolerability of HTT-Lowering: Lessons Learned from Nonhuman Primates”
