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Recently, Rona Therapeutics, a global biopharmaceutical company focused on the next generation of RNAi therapies, had its Chief Medical Officer, Dr. Alex M. DePaoli, deliver an oral presentation at the American Heart Association Annual Meeting.Phase I Clinical Study Results of RN0361, a Long-Acting siRNA Drug Targeting ApoC3.
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This study was a randomized, placebo-controlled, single ascending dose trial enrolling patients with baseline triglyceride levels exceeding 80 mg/dL. The results showed that RN0361 was well-tolerated with no serious adverse events; injection site reactions were mild and self-limiting; transient increases in ALT and AST were consistent with the typical characteristics of GalNAc-conjugated siRNA drugs. In terms of efficacy, RN0361 demonstrated dose-dependent, significant, and sustained suppression of ApoC3 and triglycerides.Sustained until Day 180 post-dosing, achieving a maximum reduction of 93% in ApoC3 and 69% in triglycerides., while simultaneously reducing non-high-density lipoprotein cholesterol, very low-density lipoprotein cholesterol, and remnant cholesterol. No significant differences were observed in fasting blood glucose or glycated hemoglobin.RN0361 achieves long-lasting efficacy of up to six months with a single dose.With potentially best-in-class characteristics, it is expected to fill the unmet clinical needs of patients with severe hypertriglyceridemia-related pancreatitis and cardiovascular risks, marking a significant advancement in the field of lipid management.Rona Therapeutics is a globally leading RNAi drug platform company dedicated to leveraging next-generation RNA technology platforms to develop therapeutics for cardiometabolic diseases, obesity, and neurodegenerative disorders. Since its establishment in 2021, the company has advanced four programs into clinical stages, driven by its proprietary advantages in delivery systems, multivalent structural design, and oligonucleotide chemistry platforms. To date, Rona Therapeutics has raised approximately $200 million from several top-tier global healthcare investment firms and strategic partners.References:
Mastering CAR-T Therapy, Stem Cell Therapy, Gene
Therapy,Comprehensive Information on Oncolytic Viruses and Nucleic Acid Drugs
