TW202132567A - 亨汀頓蛋白（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法 - Google Patents

亨汀頓蛋白（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法 Download PDF

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TW202132567A
TW202132567A TW109137670A TW109137670A TW202132567A TW 202132567 A TW202132567 A TW 202132567A TW 109137670 A TW109137670 A TW 109137670A TW 109137670 A TW109137670 A TW 109137670A TW 202132567 A TW202132567 A TW 202132567A
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nucleotides
dsrna agent
strand
antisense strand
agent according
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TW109137670A
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曼葛拉 蜜納柯斯西 索拉帕迪恩
詹姆斯 Ｄ 麥克尼奇
布萊特 李 博斯特威克
馬克Ｋ 史奇蓋爾
艾登 卡斯特瑞諾
杰弗里 祖柏
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美商阿尼拉製藥公司
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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TW109137670A 2019-11-01 2020-10-29 亨汀頓蛋白（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法 TW202132567A (zh)

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US201962929174P 2019-11-01 2019-11-01
US62/929,174 2019-11-01

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EP (1) EP4051795A1 (enExample)
JP (2) JP7830319B2 (enExample)
KR (1) KR20220094213A (enExample)
CN (2) CN114728018B (enExample)
AR (1) AR120341A1 (enExample)
AU (1) AU2020376840A1 (enExample)
CA (1) CA3155921A1 (enExample)
IL (1) IL292460A (enExample)
MX (1) MX2022004388A (enExample)
TW (1) TW202132567A (enExample)
WO (1) WO2021087036A1 (enExample)

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BR112023019981A2 (pt) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
JP2024522068A (ja) * 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ， インコーポレイテッド ナトリウム－グルコース共輸送体２（ｓｇｌｔ２）ｉｒｎａ組成物およびその使用方法
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
EP4423272A2 (en) * 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2024156083A1 (en) * 2023-01-27 2024-08-02 Beijing Jitai Pharmaceutical Technology Co., Ltd. Ai-assisted design of lnps for effective cns delivery of rna
CN118853663A (zh) * 2023-04-28 2024-10-29 北京福元医药股份有限公司 用于抑制marc1基因表达的双链核糖核酸及其修饰物、缀合物和用途
TW202532088A (zh) * 2024-02-07 2025-08-16 大陸商上海舶望製藥有限公司 抑制亨廷頓蛋白（htt）表達的組合物和方法
WO2025170027A1 (ja) 2024-02-08 2025-08-14 テルモ株式会社 オリゴヌクレオチド
WO2025265013A2 (en) * 2024-06-20 2025-12-26 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of huntingtin (htt), compositions thereof, and methods of use

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