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JNJ-75220795
- Development stage
- Unknown
- Lead developer
- Arrowhead 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
JNJ-75220795 (also known as ARO-HSD) is an investigational RNA interference (RNAi) therapeutic designed to reduce the expression of hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) in the liver. Originally developed by Arrowhead Pharmaceuticals and licensed to Janssen Research & Development, the drug utilizes Arrowhead's Targeted RNAi Molecule (TRiM) platform to deliver small interfering RNA (siRNA) specifically to hepatocytes via GalNAc conjugation. HSD17B13 is a member of the hydroxysteroid dehydrogenase family, and genetic loss-of-function variants of this enzyme have been shown to provide protection against the progression of non-alcoholic fatty liver disease (NAFLD) to non-alcoholic steatohepatitis (NASH), fibrosis, and cirrhosis. By silencing the HSD17B13 transcript, JNJ-75220795 aims to replicate this protective genetic effect to treat patients with chronic liver diseases.
- Other names
- ARO-HSD17B13ARO-HSD-17B13ARO-HSD 17B13
02
Targets
PNPLA3 (Patatin-like phospholipase domain-containing protein 3)
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