JP2023504195A - 状態および疾患の処置のためのアンチセンスオリゴマー - Google Patents

状態および疾患の処置のためのアンチセンスオリゴマー Download PDF

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JP2023504195A
JP2023504195A JP2022534221A JP2022534221A JP2023504195A JP 2023504195 A JP2023504195 A JP 2023504195A JP 2022534221 A JP2022534221 A JP 2022534221A JP 2022534221 A JP2022534221 A JP 2022534221A JP 2023504195 A JP2023504195 A JP 2023504195A
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dose
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JP2023504195A5 (https=
JPWO2021113541A5 (https=
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アズナレズ，イザベル
ハン，チョウ
クリスティアンセン，アン
ミーナ，ミーナ
ティホ，バルク
リャオ，ジーン
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ストーク セラピューティクス，インク．
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Publication of JP2023504195A5 publication Critical patent/JP2023504195A5/ja
Publication of JPWO2021113541A5 publication Critical patent/JPWO2021113541A5/ja
Priority to JP2025264280A priority Critical patent/JP2026048892A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • A—HUMAN NECESSITIES
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2022534221A 2019-12-06 2020-12-03 状態および疾患の処置のためのアンチセンスオリゴマー Pending JP2023504195A (ja)

Priority Applications (1)

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JP2025264280A JP2026048892A (ja) 2019-12-06 2025-12-18 状態および疾患の処置のためのアンチセンスオリゴマー

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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

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JP2025264280A Division JP2026048892A (ja) 2019-12-06 2025-12-18 状態および疾患の処置のためのアンチセンスオリゴマー

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JP2023504195A true JP2023504195A (ja) 2023-02-01
JP2023504195A5 JP2023504195A5 (https=) 2023-12-07
JPWO2021113541A5 JPWO2021113541A5 (https=) 2023-12-07

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JP2025264280A Pending JP2026048892A (ja) 2019-12-06 2025-12-18 状態および疾患の処置のためのアンチセンスオリゴマー

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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=)

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JP2023515974A (ja) * 2020-02-28 2023-04-17 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｓｃｎ１ａ発現を調節するための化合物及び方法

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KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
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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GB2610100B (en) * 2017-10-23 2023-08-16 Stoke Therapeutics Inc Antisense oligomers for treatment of non-sense mediated RNA decay based conditions and diseases
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Publication number Priority date Publication date Assignee Title
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CN115087452A (zh) 2022-09-20
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
MX2022006858A (es) 2022-07-11
US20230116704A1 (en) 2023-04-13
CA3159162A1 (en) 2021-06-10

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