Drug intelligence / Profile preview

ANLN siRNA

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

ANLN siRNA (also known as siAnln) is a research-stage RNA interference (RNAi) therapeutic designed to silence the expression of the ANLN gene, which encodes the actin-binding scaffolding protein anillin. Developed in partnership with Alnylam Pharmaceuticals and researchers at the University of Texas Southwestern (UTSW) Medical Center, the therapeutic is formulated as a GalNAc (N-acetylgalactosamine)-conjugated siRNA to enable selective, receptor-mediated uptake by hepatocytes via the asialoglycoprotein receptor. Knockdown of ANLN in hepatocytes inhibits cytokinesis and promotes hepatocyte polyploidization, a natural physiological mechanism that protects the liver from malignant transformation. Preclinical studies in multiple autochthonous mouse models of hepatocellular carcinoma (HCC) and metabolic dysfunction-associated steatohepatitis (MASH) have demonstrated that ANLN siRNA is safe, prevents cancer development, and improves overall survival. It is being evaluated in an investigator-initiated Phase Ia dose-escalation adjuvant clinical trial to reduce the risk of de novo recurrence in patients with HCC undergoing locoregional therapy.

Other names
ANLN-siRNAGalNAc-siANLNanillin siRNAsiANLN
02

Targets

Cytoskeleton (Cytoskeletal regulators)

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