Phase 1 Trials of PNPLA3 siRNA in I148M Homozygous Patients with MAFLD

Homozygosity for the PNPLA3 risk allele is linked to liver fat accumulation. In phase 1 trials, JNJ-75220795, a hepatocyte-targeted GalNAc-conjugated PNPLA3 siRNA, reduced liver fat in PNPLA3 I148M homozygous patients with metabolic dysfunction–associated fatty liver disease.

​   The New England Journal of Medicine: Search Results in Gastroenterology