brezosiran

Brezosiran (ALN-HTT02) is an investigational, intrathecally administered small interfering RNA (siRNA) therapeutic in development for the treatment of Huntington's disease. Developed by Alnylam in collaboration with Regeneron, brezosiran utilizes Alnylam's proprietary C16 conjugate platform for targeted delivery to the central nervous system. It is designed to target exon 1 of the huntingtin (HTT) mRNA transcript, thereby reducing the production of all forms of the huntingtin protein, including full-length mutant HTT and the shorter mutant HTT1a fragment. In Phase 1b clinical trials, brezosiran demonstrated robust, dose-dependent reductions of mutant huntingtin in the cerebrospinal fluid, supporting a potential twice-yearly dosing regimen.