KR20220115995A - 인간 염색체 ９ 개방 판독 프레임 72 (C9orf72) iRNA 제제 조성물 및 이의 사용 방법 - Google Patents

인간 염색체 ９ 개방 판독 프레임 72 (C9orf72) iRNA 제제 조성물 및 이의 사용 방법 Download PDF

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KR20220115995A
KR20220115995A KR1020227023988A KR20227023988A KR20220115995A KR 20220115995 A KR20220115995 A KR 20220115995A KR 1020227023988 A KR1020227023988 A KR 1020227023988A KR 20227023988 A KR20227023988 A KR 20227023988A KR 20220115995 A KR20220115995 A KR 20220115995A
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nucleotides
c9orf72
dsrna agent
strand
antisense strand
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엘레인 피쉴레비치
스튜어트 밀스타인
커크 브라운
트레이시 짐머만
제임스 디. 맥시닌치
데이빗 프렌더웨이
에릭 치아오
아르티 샤르마-칸닝
안토니 가글리아르디
구스타보 드로게트
브리타니 두보스
브라이언 잠브로비치
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알닐람 파마슈티칼스 인코포레이티드
리제너론 파아마슈티컬스, 인크.
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KR1020227023988A 2019-12-13 2020-12-10 인간 염색체 ９ 개방 판독 프레임 72 (C9orf72) iRNA 제제 조성물 및 이의 사용 방법 Pending KR20220115995A (ko)

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US201962947768P 2019-12-13 2019-12-13
US201962947605P 2019-12-13 2019-12-13
US62/947,605 2019-12-13
US62/947,768 2019-12-13
PCT/US2020/064159 WO2021119226A1 (en) 2019-12-13 2020-12-10 Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof

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KR20220115995A true KR20220115995A (ko) 2022-08-19

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CN (1) CN115151641A (https=)
AU (1) AU2020402885A1 (https=)
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JP2022528487A (ja) * 2019-03-29 2022-06-13 ユニバーシティ・オブ・マサチューセッツ Ｃ９ｏｒｆ７２のオリゴヌクレオチドベースの調節
US11629347B2 (en) 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
WO2021195510A1 (en) 2020-03-27 2021-09-30 University Of Massachusetts Dual-acting sirna based modulation of c9orf72
EP4347822A2 (en) 2021-06-04 2024-04-10 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
WO2022271972A1 (en) * 2021-06-23 2022-12-29 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing filamin a (flna)
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法
WO2024036343A2 (en) * 2022-08-12 2024-02-15 University Of Miami Synergistic nucleic acid based therapeutics and methods of use for treating genetic disorders
CN120035669A (zh) * 2022-09-28 2025-05-23 阿塔兰塔治疗公司 用于治疗神经系统病症的组合物和方法
KR20250116795A (ko) 2022-11-14 2025-08-01 리제너론 파마슈티칼스 인코포레이티드 별아교세포로의 섬유아세포 성장 인자 수용체 3-매개된 전달을 위한 조성물 및 방법

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