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RN5681
- Development stage
- Phase 1
- Lead developer
- Rona Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long 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, dual-targeting small interfering RNA designed by Rona Therapeutics to silence hepatic expression of **proprotein convertase subtilisin/kexin type 9** and **lipoprotein(a)**. The single molecular entity is intended to concurrently lower low-density lipoprotein cholesterol through PCSK9 suppression and lower lipoprotein(a) through LPA silencing, thereby addressing complementary drivers of residual atherosclerotic cardiovascular risk. RN5681 entered Phase 1 clinical evaluation in a randomized, placebo-controlled single-ascending-dose study in healthy adults with elevated lipid parameters. ([ronatherapeutics.com](https://www.ronatherapeutics.com/news/43))
- Other names
- PCSK9/LPA bispecific siRNAPCSK9-LPA bispecific siRNAPCSK-9-LPA bispecific siRNAPCSK 9-LPA bispecific siRNAPCSK9-LPA dual-targeting siRNAPCSK-9-LPA dual-targeting siRNAPCSK 9-LPA dual-targeting siRNA
02
Targets
PCSK9 (Proprotein convertase subtilisin/kexin type 9)LPA (Lysophosphatidic acid)
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