Rona Therapeutics, a global pioneer in RNAi-based therapies, revealed key Phase 1 findings for RN0361, a long-acting siRNA that targets Apolipoprotein C3 (ApoC3). The data, presented at the American Heart Association 2025 Scientific Sessions by Dr. Alex DePaoli, MD, Chief Medical Officer, showed that a single dose produced strong and lasting reductions in ApoC3 and triglycerides for at least six months. These results suggest RN0361 may offer a best-in-class option to prevent pancreatitis in patients with severe hypertriglyceridemia and reduce cardiovascular risk from atherogenic lipoproteins.
Health Technology Insights: Verily Accelerates Precision Health AI With NVIDIA
The first-in-human, randomized, placebo-controlled Single Ascending Dose study enrolled participants with baseline triglycerides above 80 mg/dl. RN0361 was well tolerated, with no serious adverse events. Observed side effects included mild, self-limiting injection-site reactions and temporary elevations in liver enzymes, consistent with GalNAc-conjugated siRNAs. Dose-dependent reductions were observed, with ApoC3 falling as much as 93 percent and triglycerides decreasing by up to 69 percent. The therapy also lowered non-HDL cholesterol, VLDL cholesterol, and remnant cholesterol without affecting fasting glucose or HbA1c compared to placebo.
Health Technology Insights: Innovaccer Brings Multimodal AI to the Frontlines of Care with NVIDIA
ApoC3 contributes to the buildup of triglyceride-rich lipoproteins, which increases the risk of acute pancreatitis and cardiovascular disease, especially in individuals with diabetes. Current treatments often do not provide sustained control in severe cases. RN0361’s single-dose, six-month efficacy addresses this gap and represents a significant advance in lipid management.
“These results highlight RN0361’s strong, durable effect and favorable safety profile, supporting its progression to Phase 2 trials in hypertriglyceridemia patients,” said Stella Shi, CEO. “By effectively lowering ApoC3, triglycerides, and atherogenic lipids for at least six months with a single dose, RN0361 could become a critical therapy for severe hypertriglyceridemia and associated cardiovascular risk.”
Rona Therapeutics continues to develop RN0361 with the goal of providing patients with a long-lasting, effective treatment for lipid disorders and cardiovascular risk reduction.
Health Technology Insights: Health Technology Insights: Latest News And Innovations – Roundup 29 October 2025
To participate in our interviews, please write to our HealthTech Media Room at info@intentamplify.com
