Rona Therapeutics Advances First-in-Class Dual-Targeting PCSK9-LPA siRNA RN5681 into Clinical Development
Rona Therapeutics
Nucleic Acid Drug Developer
December 17,Rona Therapeutics（Rona Therapeutics）Announced that the company has successfully submitted to the Australian Human Research Ethics Committee (HREC)RN5681The clinical trial application advances the company's first dual-target siRNA into the clinical development stage. This Phase 1 clinical trial is expected to begin dosing in the first quarter of 2026.
RN5681 is a GalNAc-conjugated dual-targeting siRNA designed toSimultaneous Silencing of PCSK9 and LPAThese two genetically validated and complementary drivers of atherosclerotic cardiovascular disease.By simultaneously lowering low-density lipoprotein cholesterol and lipoprotein(a) through a single molecule, RN5681 aims to achieve deep and lasting improvements in blood lipid levels.
Alex DePaoli, M.D., Chief Medical Officer (CMO) of Rona TherapeuticsRN5681 is expected to expand the existing treatment paradigm by simultaneously intervening in the two most important cardiovascular risk factors through a single-molecule drug that may require dosing only once every 6 to 12 months.
References:
[1] Rona Therapeutics' World's First Dual-Target PCSK9-LPA siRNA Enters Clinical Development, Marking a New Phase in Cardiovascular Risk Treatment. From https://www.prnasia.com/story/516413-1.shtml
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