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

Elisa Fabbrini; Brian Rady; Anastasiya Koshkina; Jae Yoon Jeon; Vivaswath S. Ayyar; Cynthia Gargano; Nicholas A. DiProspero; Susan Wendel; Julia Hegge; Holly Hamilton; Zhiming Ding; Maria Afrazi; Anthony L. Nicholas; Tao Pei; Masayoshi Nakano; Shohei Ouchi; Yuki Saito; Aimi Yamashita; Ryo Tamamura; Hernan Salazar; Craig Shapiro; Tatsuya Yoshihara; Takuma Yonemura; Satoshi Inoue; Osamu Matsuoka; Mark D. Erion; Alessandro Pocai; Hideo Makimura

doi:10.1056/nejmc2402341

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Abstract

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.

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Fabbrini et al. (2024) studied this question.

synapsesocial.com/papers/6a6b6c2e47a1bfb288f31fb3 https://doi.org/10.1056/nejmc2402341

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

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

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