ALN-TTR01 + ALN-TTR02

ALN-TTR01 + ALN-TTR02 is a combination concept referring to two successive intravenously administered small interfering RNA (siRNA) lipid nanoparticle (LNP) formulations developed by Alnylam Pharmaceuticals to silence hepatic production of transthyretin (TTR) for the treatment of transthyretin-mediated amyloidosis (ATTR), including familial amyloid polyneuropathy and cardiomyopathy.[1][2][5][9] Both agents contain the same TTR-targeted siRNA, with ALN-TTR01 using a first-generation LNP delivery system and ALN-TTR02 (later named patisiran) using a proprietary second-generation MC3-containing LNP with markedly enhanced potency and improved tolerability, achieving rapid, dose-dependent, and durable reductions of both wild-type and mutant serum TTR levels after intravenous infusion.[2][3][4][5][7] ALN-TTR01 served primarily as an early proof-of-concept clinical tool, while ALN-TTR02/patisiran advanced into later-stage development and ultimately became the go-to-market RNAi therapeutic for ATTR.[1][2][3][7]