CN117795069A - 用于将治疗性寡核苷酸递送至中枢神经系统的组合物和方法 - Google Patents

用于将治疗性寡核苷酸递送至中枢神经系统的组合物和方法 Download PDF

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Publication number
CN117795069A
CN117795069A CN202280052900.3A CN202280052900A CN117795069A CN 117795069 A CN117795069 A CN 117795069A CN 202280052900 A CN202280052900 A CN 202280052900A CN 117795069 A CN117795069 A CN 117795069A
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formula
ribonucleoside
internucleoside linkage
sirna molecule
divalent cations
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Chinese (zh)
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M·哈斯勒
G·A·基恩贝格尔
B·M·D·C·戈丁霍
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Atalanta Treatment Co
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Atalanta Treatment Co
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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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CN202280052900.3A 2021-06-02 2022-06-02 用于将治疗性寡核苷酸递送至中枢神经系统的组合物和方法 Pending CN117795069A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163195993P 2021-06-02 2021-06-02
US63/195,993 2021-06-02
PCT/US2022/032014 WO2022256565A2 (en) 2021-06-02 2022-06-02 Compositions and methods for delivering therapeutic oligonucleotides to the central nervous system

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Publication Number Publication Date
CN117795069A true CN117795069A (zh) 2024-03-29

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CN202280052900.3A Pending CN117795069A (zh) 2021-06-02 2022-06-02 用于将治疗性寡核苷酸递送至中枢神经系统的组合物和方法

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US (1) US20240287526A1 (enExample)
EP (1) EP4346845A4 (enExample)
JP (1) JP2024521897A (enExample)
KR (1) KR20240038930A (enExample)
CN (1) CN117795069A (enExample)
AU (1) AU2022286420A1 (enExample)
CA (1) CA3222212A1 (enExample)
IL (1) IL308994A (enExample)
WO (1) WO2022256565A2 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024073595A2 (en) * 2022-09-28 2024-04-04 Atalanta Therapeutics, Inc. Compositions and methods for treatment of huntington's disease
CN118202055A (zh) * 2023-02-17 2024-06-14 苏州时安生物技术有限公司 一种抑制SCN9A基因表达的siRNA、其药物组合物及用途
CN121241143A (zh) * 2023-03-23 2025-12-30 阿塔兰塔治疗公司 用于改善的基因沉默的组合物和方法
WO2025111497A1 (en) * 2023-11-21 2025-05-30 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating acute activation
WO2025193786A1 (en) * 2024-03-12 2025-09-18 University Of Massachusetts Oligonucleotides for prnp modulation

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004042024A2 (en) * 2002-11-01 2004-05-21 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF HIF-1 ALPHA
WO2011017297A2 (en) * 2009-08-03 2011-02-10 The University Of North Carolina At Chapel Hill Biodegradable delivery system complexes for the delivery of bioactive compounds
JP5775581B2 (ja) * 2010-08-20 2015-09-09 レプリコール インコーポレーティッド オリゴヌクレオチドキレート錯体
CN102659495B (zh) * 2012-04-25 2014-02-26 天津大学 利用寡核苷酸拆分手性药物的方法
RU2668136C2 (ru) * 2012-05-10 2018-09-26 Эйдинкс, Инк. Композиции для доставки активных ингредиентов
AR091065A1 (es) * 2012-05-18 2014-12-30 Replicor Inc Una formulacion farmaceutica que comprende un quelato de oligonucleotidos antiviral para el tratamiento de una infeccion antiviral
WO2015125147A1 (en) * 2014-02-20 2015-08-27 B. G. Negev Technologies And Applications Ltd Anionic polyplexes for use in the delivery of nucleic acids
WO2017132669A1 (en) * 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
US20200339982A1 (en) * 2017-12-22 2020-10-29 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage

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AU2022286420A1 (en) 2024-01-04
IL308994A (en) 2024-01-01
EP4346845A2 (en) 2024-04-10
KR20240038930A (ko) 2024-03-26
WO2022256565A3 (en) 2023-01-05
JP2024521897A (ja) 2024-06-04
WO2022256565A2 (en) 2022-12-08
EP4346845A4 (en) 2026-07-15
US20240287526A1 (en) 2024-08-29
CA3222212A1 (en) 2022-12-08

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