On July 26th, 2024, Rona Therapeutics announced that its Investigational New Drug (IND) application for its potentially Best-in-Class siRNA pipeline product, RN0361, has been approved in Australia. The drug candidate, as a leading product in RONA’s siRNA pipeline, targets severe hypertriglyceridemia (sHTG), mixed dyslipidemia, and familial chylomicronemia.

About RN0361

RN0361 is a double-stranded small interfering RNA (siRNA) drug targeting the apolipoprotein C3 (APOC3) gene, derived from Rona Therapeutics' proprietary GAIA liver-targeting platform. The GAIA platform features superior GalNAc (N-acetylgalactosamin) conjugation, finely optimized full-sequence chemical modifications, and chemically modified frameworks that enhanced activity and specificity, achieving deeper and more sustained gene silencing in the liver. RN0361 works by efficiently, significantly, and persistently silencing APOC3 mRNA levels, reducing the expression of APOC3 protein. It is poised to become a Best-in-Class siRNA drug molecule for this target.
This trial is a randomized, single-blind, placebo-controlled, single-center Phase I clinical study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RN0361 in adult subjects with elevated triglyceride levels.

About Hypertriglyceridemia and Familial Chylomicronemia Syndrome

Hypertriglyceridemia (HTG) is a common type of lipid abnormality. HTG is a risk factor for atherosclerotic cardiovascular disease (ASCVD) and acute pancreatitis, and is associated with obesity, insulin resistance/type 2 diabetes, non-alcoholic fatty liver disease (NAFLD), and chronic kidney disease (CKD).
Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive genetic disorder primarily caused by mutations in the lipoprotein lipase gene, leading to significantly elevated plasma chylomicron concentrations and triglyceride levels.

About APOC3

The apolipoprotein C (APOC) family includes APOC1, APOC2, APOC3, and APOC4 subtypes. APOC3 is the most abundant member of the APOC family, mainly expressed in hepatocytes and to a lesser extent in intestinal epithelial cells. In circulation, APOC3 associates with triglyceride-rich lipoproteins (TRLs) – consisting of chylomicrons (CM) from the intestine and very-low-density lipoproteins (VLDL) from the liver – and high-density lipoprotein (HDL). APOC3 increases plasma triglyceride levels by inhibiting lipoprotein lipase (LPL) activity, increasing plasma triglyceride levels, and interferes with the clearance of TRLs and their remnants via LDL receptors (LDLR) and LDL receptor-related protein 1 (LRP1), making it a promising therapeutic target for reducing dyslipidemia and cardiovascular disease (CVD) risk.

About Rona Therapeutics

Rona Therapeutics is a pioneering RNA therapeutics platform company dedicated to discovery and development of modular and programmable RNA medicines to deliver to patients around the world.
Backed by blue-chip investors, Rona has developed proprietary RAZOR™ platform to discover differentiated siRNA therapeutics from next-generation chemical modification and novel biological insights. Together with partners, Rona is dedicated to advancing transformative RNA therapeutics into clinic in metabolic syndrome, glomerulonephritis.