C-GEEHsiRNA, RNA therapeutics, delivery platforms
0-100 chain-logic scale · 15 dimensions · scored on public evidence
Enable extrahepatic delivery for Rona Therapeutics' siRNA medicines.
Extrahepatic RNA therapeutic delivery platform.
Described on the company site as a delivery platform.
Support development of bi-valent or multi-valent siRNA therapeutic approaches.
Plug-and-play platform approach for bi-valent or multi-valent RNA therapeutics.
Described on the company site as part of Rona Therapeutics' platform approaches.
Evaluate liver-specific inhibition of CIDEB as a therapeutic approach for MASH and metabolic disease.
GalNAc-conjugated Cideb surrogate siRNAs tested in diet-induced obesity and CDAA-HFD MASH animal models, with humanized CIDEB knock-in mice generated to support human therapeutic siRNA discovery.
Preclinical pharmacological proof-of-concept study published in 2025; liver-targeting siRNA candidates are under development for therapeutic hypothesis testing in humans.
Cideb knockdown reduced serum total cholesterol and triglycerides, reduced hepatic steatosis, produced notable weight loss in diet-induced obesity mice, reduced liver cholesterol and triglycerides in MASH models, improved NAS score, and ameliorated liver fibrosis.
Enable liver delivery for Rona Therapeutics' siRNA medicines.
Liver-targeted RNA therapeutic delivery platform.
Described on the company site as a delivery platform.
Develop an siRNA therapeutic candidate within Rona Therapeutics' metabolic pipeline, which targets unmet needs in cardiovascular diseases, obesity, MASH, hypertension, and related metabolic indications.
siRNA medicine developed using Rona Therapeutics' RNA therapeutics and delivery platform approaches.
Listed on the company pipeline.
Develop an siRNA therapeutic candidate within Rona Therapeutics' metabolic pipeline, which targets unmet needs in cardiovascular diseases, obesity, MASH, hypertension, and related metabolic indications.
siRNA medicine developed using Rona Therapeutics' RNA therapeutics and delivery platform approaches.
Listed on the company pipeline.
Develop an siRNA therapeutic candidate within Rona Therapeutics' metabolic pipeline, which targets unmet needs in cardiovascular diseases, obesity, MASH, hypertension, and related metabolic indications.
siRNA medicine developed using Rona Therapeutics' RNA therapeutics and delivery platform approaches.
Listed on the company pipeline.