Drug intelligence / Profile preview

BW-41

Development stage
Preclinical
Lead developer
Shanghai Argo Biopharmaceutical
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

BW-41 is an investigational small interfering RNA (siRNA) therapy developed by Shanghai Argo Biopharmaceutical for the treatment of Primary Hyperoxaluria Type 1 (PH1). It is designed to silence the *HAO1* gene in the liver, which encodes the enzyme hydroxyacid oxidase 1 (also known as glycolate oxidase). By reducing the levels of this enzyme, BW-41 aims to decrease the production of oxalate, the toxic metabolite that causes kidney damage and systemic oxalosis in PH1 patients. The drug utilizes Argo's proprietary RNAi platform, which typically employs GalNAc conjugation for targeted delivery to hepatocytes via the asialoglycoprotein receptor (ASGPR). BW-41 is part of a broader pipeline of liver-targeted RNAi therapeutics developed by Argo Biopharma for rare and metabolic diseases.

02

Targets

HAO1 (Glycolate Oxidase)

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