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RN5681 (Rona Therapeutics)
- Development stage
- Unknown
- Lead developer
- Rona Therapeutics
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
**RN5681** is an investigational GalNAc-conjugated, bi-valent small interfering RNA designed by Rona Therapeutics to concurrently silence **PCSK9** and **LPA** expression through RNA interference. The single-molecule RNA therapy is intended to reduce both LDL cholesterol and lipoprotein(a), addressing complementary lipid-mediated drivers of atherosclerotic cardiovascular risk. RN5681 is being evaluated in a randomized, placebo-controlled Phase 1a single-ascending-dose study in healthy adults with elevated LDL-C and lipoprotein(a); the study is sponsored by Ikaria Bioscience. ([ronatherapeutics.com](https://www.ronatherapeutics.com/news/43))
- Other names
- RN-5681RN5681RN 5681
02
Targets
ASGPR (Asialoglycoprotein Receptor 1)LPA (Lysophosphatidic acid)PCSK9 (Proprotein convertase subtilisin/kexin type 9)
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