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ALN-ANGsc
- Development stage
- Unknown
- 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-ANGsc is an investigational, subcutaneously administered small interfering RNA (siRNA) therapeutic designed to inhibit the synthesis of Angiopoietin-like 3 (ANGPTL3) in the liver. Developed by Alnylam Pharmaceuticals in collaboration with Regeneron Pharmaceuticals, the drug utilizes Alnylam's GalNAc-conjugate delivery platform (Enhanced Stabilization Chemistry) to specifically target hepatocytes. By silencing the ANGPTL3 gene, ALN-ANGsc aims to lower circulating levels of triglycerides and LDL cholesterol, addressing unmet needs in patients with mixed hyperlipidemia and other forms of dyslipidemia. Preclinical studies in rodent and non-human primate models have demonstrated significant and durable reductions in ANGPTL3 protein and lipid levels following a single dose, supporting its potential for infrequent dosing in clinical settings.
02
Targets
ANGPTL3 (Angiopoietin-like protein 3)
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