TTR siRNA

TTR siRNA (Argo Biopharma) is an investigational small interfering RNA (siRNA) therapeutic developed by Shanghai Argo Biopharmaceutical for the treatment of transthyretin-mediated (ATTR) amyloidosis and Stargardt disease. Utilizing the company's proprietary RADS (RNA molecules with superior Activity, Durability, and Safety) platform, the drug is designed to target and degrade transthyretin (TTR) messenger RNA (mRNA) in the liver. By silencing the TTR gene, the therapy reduces the synthesis of both wild-type and mutant TTR proteins, thereby preventing the formation of amyloid deposits that lead to progressive organ dysfunction in conditions such as ATTR cardiomyopathy (ATTR-CM) and ATTR polyneuropathy (ATTR-PN). Additionally, by reducing circulating TTR—the primary carrier of retinol-binding protein 4 (RBP4)—the therapy is being explored for Stargardt disease to limit the accumulation of toxic vitamin A byproducts in the retina. As of early 2026, the program is in the preclinical and IND-enabling stages of development.

Other names

TTR siRNA