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ARO-PNPLA3
- Development stage
- Phase 1
- 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
ARO-PNPLA3 (formerly JNJ-75220795) is an investigational, GalNAc-conjugated small interfering RNA (siRNA) therapeutic designed to treat non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). Developed using Arrowhead Pharmaceuticals' Targeted RNAi Molecule (TRiM) platform, the drug specifically targets the mRNA of the patatin-like phospholipase domain containing 3 (PNPLA3) gene to reduce its expression in the liver. It is particularly aimed at patients carrying the I148M genetic mutation in PNPLA3, which is strongly associated with increased liver fat and progression to advanced liver disease. By silencing the expression of this variant, ARO-PNPLA3 aims to reduce hepatic steatosis and inflammation. Originally developed in collaboration with Janssen Pharmaceuticals, the rights were returned to Arrowhead in early 2023 following a strategic portfolio review by Janssen.
- Other names
- JNJ-75220795JNJ75220795JNJ 75220795ARO-PNPLA3ARO-PNPLA-3ARO-PNPLA 3JNJ 0795JNJ0795JNJ-0795
02
Targets
PNPLA3 (Patatin-like phospholipase domain-containing protein 3)
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