Drug intelligence / Profile preview

ALN-ANG

Development stage
Preclinical
Lead developer
Alnylam 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
Administration
Subcutaneous
01

Overview

**ALN-ANG** is an investigational subcutaneously administered RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals targeting **angiopoietin-like 3 (ANGPTL3)**, a protein that inhibits lipoprotein lipase and endothelial lipase to regulate triglyceride and LDL cholesterol metabolism. By silencing ANGPTL3 mRNA in the liver via GalNAc-conjugated siRNA technology, it reduces circulating triglycerides, LDL-C, and total cholesterol, offering potential for treating genetic forms of **mixed hyperlipidemia** and **severe hypertriglyceridemia**, conditions linked to elevated cardiovascular and pancreatitis risks. Preclinical data in ob/ob mice showed >80% triglyceride reduction and up to 68% total cholesterol lowering after a single dose, supporting advancement toward clinical development.[10]

02

Targets

ANGPTL3 (Angiopoietin-like protein 3)

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