- 专利标题： HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
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申请号： PCT/US2022/031519申请日： 2022-05-31
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公开(公告)号： WO2022256290A2公开(公告)日： 2022-12-08
- 发明人: FRENDEWEY, David , SHARMA-KANNING, Aarti
- 申请人： ALNYLAM PHARMACEUTICALS, INC. , REGENERON PHARMACEUTICALS, INC. , DANG, Lan, Thi Hoang , MCININCH, James, D. , NGUYEN, Tuyen, M. , DUBOSE, Brittany
- 申请人地址： 675 West Kendall Street; 777 Old Saw Mill River Road; 675 West Kendall Street; 675 West Kendall Street; 675 West Kendall Street; c/o Regeneron Pharmaceuticals, Inc.
- 专利权人： ALNYLAM PHARMACEUTICALS, INC.,REGENERON PHARMACEUTICALS, INC.,DANG, Lan, Thi Hoang,MCININCH, James, D.,NGUYEN, Tuyen, M.,DUBOSE, Brittany
- 当前专利权人： ALNYLAM PHARMACEUTICALS, INC.,REGENERON PHARMACEUTICALS, INC.,DANG, Lan, Thi Hoang,MCININCH, James, D.,NGUYEN, Tuyen, M.,DUBOSE, Brittany
- 当前专利权人地址： 675 West Kendall Street; 777 Old Saw Mill River Road; 675 West Kendall Street; 675 West Kendall Street; 675 West Kendall Street; c/o Regeneron Pharmaceuticals, Inc.
- 代理机构： ZACHARAKIS, Maria, Laccotripe et al.
- 优先权： US63/196,791 2021-06-04
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; A61K31/713 ; A61P25/28 ; C12N2310/14 ; C12N2310/3183 ; C12N2310/321 ; C12N2310/322 ; C12N2310/351 ; C12N2310/3515
摘要：
The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disorder, e.g., C9orf72 amyotrophic lateral sclerosis, frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.
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