nucresiran

ALN-TTRsc04 (nucresiran) is an investigational small interfering RNA (siRNA) therapeutic developed for the treatment of transthyretin amyloidosis (ATTR), a progressive and fatal disease caused by the deposition of misfolded transthyretin (TTR) amyloid fibrils in organs and tissues, leading to manifestations such as polyneuropathy and cardiomyopathy. The drug specifically targets hepatic TTR mRNA, resulting in a rapid and sustained reduction of circulating TTR protein levels. In Phase 1 studies, single subcutaneous doses of 300 mg or higher led to mean serum TTR reductions greater than 90% by Day 15, with effects sustained for at least six months. The safety profile has been encouraging, with most adverse events being mild and not considered related to the drug. Nucresiran utilizes Alnylam’s IKARIA platform technology and is designed for infrequent dosing—potentially biannual or annual administration[1][2][3][5][7].

Other names

nucresiran ALN-TTRsc04