RN3271

RN3271 is a novel GalNAc-conjugated small interfering RNA (siRNA) therapeutic developed by Shanghai Rona Therapeutics for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). Developed using the company's proprietary GAIA platform, RN3271 is designed to specifically target and silence the expression of the transthyretin (TTR) protein in the liver. By reducing the production of both wild-type and mutant TTR, the drug aims to prevent the formation of amyloid deposits that lead to heart failure. Preclinical data presented at the Heart Failure Society of America (HFSA) 2025 meeting demonstrated that RN3271 achieves deep and durable TTR silencing with a favorable safety profile, potentially offering improved efficacy and less frequent dosing compared to current standards of care.