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

被引：11

作者：

Fabbrini, Elisa ^{[1
]}

Rady, Brian ^{[2
]}

Koshkina, Anastasiya ^{[1
]}

Jeon, Jae Yoon ^{[1
]}

Ayyar, Vivaswath S. ^{[2
]}

Gargano, Cynthia ^{[2
]}

DiProspero, Nicholas ^{[1
]}

Wendel, Susan ^{[1
]}

Hegge, Julia ^{[3
]}

Hamilton, Holly ^{[3
]}

Ding, Zhi-Ming ^{[3
]}

Afrazi, Maria ^{[3
]}

Nicholas, Anthony ^{[3
]}

Pei, Tao ^{[3
]}

Nakano, Masayoshi ^{[4
]}

Ouchi, Shohei ^{[4
]}

Saito, Yuki ^{[4
]}

Yamashita, Aimi ^{[4
]}

Tamamura, Ryo ^{[4
]}

Salazar, Hernan ^{[5
]}

Shapiro, Craig ^{[6
]}

Yoshihara, Tatsuya ^{[7
]}

Yonemura, Takuma ^{[8
]}

Inoue, Satoshi ^{[9
]}

Matsuoka, Osamu ^{[10
]}

Erion, Mark ^{[2
]}

Pocai, Alessandro ^{[2
]}

Makimura, Hideo ^{[1
]}

机构：

[1] Janssen Res & Dev, Raritan, NJ 08869 USA

[2] Janssen Res & Dev, Spring House, PA USA

[3] Arrowhead Pharmaceut, Pasadena, CA USA

[4] Janssen Pharmaceut, Tokyo, Japan

[5] Endeavor Clin Trials, San Antonio, TX USA

[6] CenExel RCA, Hollywood, FL USA

[7] Souseikai Fukuoka Mirai Hosp, Fukuoka, Japan

[8] Sumida Hosp, Tokyo, Japan

[9] OCROM Clin, Osaka, Japan

[10] Med Corp Heishinkai, Tokyo, Japan

来源：

NEW ENGLAND JOURNAL OF MEDICINE | 2024年 / 391卷 / 05期

关键词：

D O I：

10.1056/NEJMc2402341

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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.

引用

页数：3

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