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ALN-APOC3
- Development stage
- Phase 1
- Lead developer
- Regeneron Pharmaceuticals
- 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, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Subcutaneous
01
Overview
ALN-APOC3 is an investigational RNA interference (RNAi) therapeutic targeting apolipoprotein C-III (APOC3), a key regulator of triglyceride metabolism. APOC3 inhibits the hydrolysis and clearance of triglyceride-rich lipoproteins by interfering with lipoprotein lipase and hepatic uptake, leading to elevated plasma triglycerides. By reducing APOC3 expression, ALN-APOC3 aims to lower triglyceride levels in patients with dyslipidemia. The drug is being developed for the treatment of dyslipidemias, including hypertriglyceridemia, and is currently in Phase 1/2 clinical trials. The developer and sponsor is Regeneron Pharmaceuticals[1][2][3][4].
02
Targets
APOC3 (Apolipoprotein C-III)
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