RN596

RN596 is a preclinical-stage small interfering RNA (siRNA) therapeutic being developed by Shanghai Rona Therapeutics for the treatment of Alzheimer's disease and other tauopathies. It is a lead candidate emerging from the company's proprietary APOLLO platform, which is engineered to enable the delivery of oligonucleotide therapeutics to extra-hepatic tissues, specifically the central nervous system (CNS). RN596 functions by targeting and silencing the expression of the Microtubule-associated protein tau (MAPT) gene through the RNA interference (RNAi) pathway. By reducing the production of tau protein, the drug aims to prevent the formation of neurofibrillary tangles, a hallmark of neurodegeneration. As of 2024, the program is in the IND-enabling or preclinical stage, representing Rona's strategic expansion into neurodegenerative and neuromuscular indications beyond liver-targeted therapies.