LY3954068

LY3954068 is an investigational small interfering RNA (siRNA) therapeutic developed by Eli Lilly and Company for the treatment of Alzheimer's disease. It is designed to reduce levels of tau protein in the brain by targeting its messenger RNA through RNA interference, thereby inhibiting tau aggregation—a key pathological hallmark of Alzheimer’s disease. The drug is administered via intrathecal infusion (into the fluid surrounding the spinal cord) to facilitate distribution to the brain. Its primary mechanism aims to slow clinical progression of Alzheimer’s disease by reducing neurofibrillary tangles formed from tau protein clumping. As of early 2025, LY3954068 is in Phase 1 clinical trials evaluating safety, pharmacokinetics, and effects on biomarkers in patients with early symptomatic Alzheimer’s disease[1][2][3][4][5][7].