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RN0361
- Development stage
- Phase 2
- 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
RN0361 is an investigational small interfering RNA (siRNA) therapeutic targeting apolipoprotein C-III (APOC3), developed for the treatment of severe hypertriglyceridemia, mixed dyslipidemia, and familial chylomicronemia syndrome. It utilizes a proprietary siRNA conjugate and oligo chemistry platform to enable robust and durable hepatocyte-specific silencing of APOC3 via subcutaneous administration. The drug achieves its effect by significantly reducing APOC3 mRNA and protein expression in the liver, leading to sustained reductions in triglyceride levels. Clinical development has advanced through phase 1 with favorable safety data and is entering phase 2 trials[1][4][6].
- Other names
- RONA-APOC3RONA-APOC-3RONA-APOC 3
02
Targets
APOC3 (Apolipoprotein C-III)
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