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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CN202280052900.3A
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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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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 (https=)
EP (1) EP4346845A2 (https=)
JP (1) JP2024521897A (https=)
KR (1) KR20240038930A (https=)
CN (1) CN117795069A (https=)
AU (1) AU2022286420A1 (https=)
CA (1) CA3222212A1 (https=)
IL (1) IL308994A (https=)
WO (1) WO2022256565A2 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL319764A (en) * 2022-09-28 2025-05-01 Atalanta Therapeutics Inc Compositions and methods for treating Huntington's disease
WO2024169770A1 (zh) * 2023-02-17 2024-08-22 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
JP2026509929A (ja) * 2023-03-23 2026-03-25 アタランタ セラピューティクス，インコーポレーテッド 改善された遺伝子サイレンシングのための組成物及び方法
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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2019122279A1 (en) * 2017-12-22 2019-06-27 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage

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

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