CN115087452A - 用于治疗病况和疾病的反义寡聚体 - Google Patents

用于治疗病况和疾病的反义寡聚体 Download PDF

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Publication number
CN115087452A
CN115087452A CN202080095947.9A CN202080095947A CN115087452A CN 115087452 A CN115087452 A CN 115087452A CN 202080095947 A CN202080095947 A CN 202080095947A CN 115087452 A CN115087452 A CN 115087452A
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diluent
dose
diluted
dissolved
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Inventor
伊莎贝尔·阿兹纳雷兹
韩舟
安妮·克里斯蒂安森
米纳·米纳
巴鲁克·蒂乔
吉恩·里奥
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Stoke Therapeutics Inc
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Stoke Therapeutics Inc
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
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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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CN202080095947.9A 2019-12-06 2020-12-03 用于治疗病况和疾病的反义寡聚体 Pending CN115087452A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962945048P 2019-12-06 2019-12-06
US62/945,048 2019-12-06
US202062993971P 2020-03-24 2020-03-24
US62/993,971 2020-03-24
PCT/US2020/063157 WO2021113541A1 (en) 2019-12-06 2020-12-03 Antisense oligomers for treatment of conditions and diseases

Publications (1)

Publication Number Publication Date
CN115087452A true CN115087452A (zh) 2022-09-20

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CN202080095947.9A Pending CN115087452A (zh) 2019-12-06 2020-12-03 用于治疗病况和疾病的反义寡聚体

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Country Link
US (1) US20230116704A1 (https=)
EP (1) EP4069256A4 (https=)
JP (2) JP2023504195A (https=)
KR (1) KR20220113743A (https=)
CN (1) CN115087452A (https=)
AU (1) AU2020395835A1 (https=)
BR (1) BR112022010882A2 (https=)
CA (1) CA3159162A1 (https=)
IL (1) IL293531A (https=)
MX (1) MX2022006858A (https=)
WO (1) WO2021113541A1 (https=)

Families Citing this family (10)

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Publication number Priority date Publication date Assignee Title
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
WO2021174036A1 (en) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating scn1a expression
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
EP4392562A4 (en) * 2021-08-27 2025-09-24 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
EP4562020A2 (en) * 2022-07-29 2025-06-04 Stoke Therapeutics, Inc. Compounds for treatment of conditions and diseases
JP2025526712A (ja) * 2022-08-11 2025-08-15 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその方法
EP4665407A2 (en) * 2023-02-14 2025-12-24 Stoke Therapeutics, Inc. Antisense oligomer formulations
MX2025013084A (es) * 2023-05-03 2026-02-03 Stoke Therapeutics Inc Compuestos y métodos para tratar sujetos humanos
WO2026036032A1 (en) * 2024-08-09 2026-02-12 Amylyx Pharmaceuticals, Inc. Formulations containing antisense oligonucleotides targeting calpain-2
WO2026052819A1 (en) * 2024-09-06 2026-03-12 Ospedale San Raffaele S.R.L. Gene therapy

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CN109312343A (zh) * 2015-12-14 2019-02-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
WO2019040923A1 (en) * 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASE CONDITIONS AND OTHER DISEASES
CN111936163A (zh) * 2017-10-23 2020-11-13 斯托克制药公司 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体
CN113748209A (zh) * 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体

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WO2011163499A2 (en) * 2010-06-23 2011-12-29 Opko Curna, Llc Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna
WO2019224864A1 (ja) * 2018-05-21 2019-11-28 国立研究開発法人理化学研究所 Ｓｃｎ１ａ遺伝子の発現増強法とそれによるドラベ症候群の治療法

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WO2019040923A1 (en) * 2017-08-25 2019-02-28 Stoke Therapeutics, Inc. ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASE CONDITIONS AND OTHER DISEASES
CN111936163A (zh) * 2017-10-23 2020-11-13 斯托克制药公司 用于治疗基于无义介导的rna衰变的病况和疾病的反义寡聚体
CN113748209A (zh) * 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体

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KIAN HUAT LIM等: "Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression", NATURE COMMUNICATIONS, vol. 11, 9 July 2020 (2020-07-09), pages 1 - 13 *
SOPHIE F. HILL等: "Antisense Oligonucleotide Therapy for Neurodevelopmental Disorders", DEV NEUROSCI, vol. 43, 5 August 2021 (2021-08-05), pages 247 - 252, XP093123448, DOI: 10.1159/000517686 *
STOKE THERAPEUTICS: "Stoke Therapeutics Presents Data from the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2021 Annual Meeting", AMERICAN EPILEPSY SOCIETY (AES) 2021 ANNUAL MEETING, 3 December 2021 (2021-12-03), pages 1 - 3 *
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WO2021113541A1 (en) 2021-06-10
IL293531A (en) 2022-08-01
KR20220113743A (ko) 2022-08-16
AU2020395835A1 (en) 2022-06-23
JP2026048892A (ja) 2026-03-17
EP4069256A1 (en) 2022-10-12
BR112022010882A2 (pt) 2022-10-04
EP4069256A4 (en) 2023-10-18
JP2023504195A (ja) 2023-02-01
MX2022006858A (es) 2022-07-11
US20230116704A1 (en) 2023-04-13
CA3159162A1 (en) 2021-06-10

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