JP6987041B2 - 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー - Google Patents

脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー Download PDF

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JP6987041B2
JP6987041B2 JP2018510718A JP2018510718A JP6987041B2 JP 6987041 B2 JP6987041 B2 JP 6987041B2 JP 2018510718 A JP2018510718 A JP 2018510718A JP 2018510718 A JP2018510718 A JP 2018510718A JP 6987041 B2 JP6987041 B2 JP 6987041B2
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modified antisense
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マルコ エー． パッシーニ，
ガンナー ジェイ． ハンソン，
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サレプタ セラピューティクス，インコーポレイテッド
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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
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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
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2018510718A 2015-08-28 2016-08-26 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー Active JP6987041B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562211678P 2015-08-28 2015-08-28
US62/211,678 2015-08-28
US201662379696P 2016-08-25 2016-08-25
US62/379,696 2016-08-25
PCT/US2016/048965 WO2017040271A1 (en) 2015-08-28 2016-08-26 Modified antisense oligomers for exon inclusion in spinal muscular atrophy

Related Child Applications (1)

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JP2020216610A Division JP2021048877A (ja) 2015-08-28 2020-12-25 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー

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JP2018525015A JP2018525015A (ja) 2018-09-06
JP2018525015A5 JP2018525015A5 (cg-RX-API-DMAC7.html) 2019-10-03
JP6987041B2 true JP6987041B2 (ja) 2021-12-22

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JP2018510718A Active JP6987041B2 (ja) 2015-08-28 2016-08-26 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー
JP2020216610A Withdrawn JP2021048877A (ja) 2015-08-28 2020-12-25 脊髄性筋萎縮症におけるエクソン包含のための修飾アンチセンスオリゴマー

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US (3) US10905709B2 (cg-RX-API-DMAC7.html)
EP (1) EP3341480A1 (cg-RX-API-DMAC7.html)
JP (2) JP6987041B2 (cg-RX-API-DMAC7.html)
AU (1) AU2016317667A1 (cg-RX-API-DMAC7.html)
CA (1) CA2996164A1 (cg-RX-API-DMAC7.html)
HK (1) HK1257498A1 (cg-RX-API-DMAC7.html)
MA (1) MA42695A (cg-RX-API-DMAC7.html)
WO (1) WO2017040271A1 (cg-RX-API-DMAC7.html)

Families Citing this family (6)

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WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
MA42695A (fr) 2015-08-28 2018-07-04 Sarepta Therapeutics Inc Oligomères antisens modifiés pour l'inclusion d'exons dans l'atrophie musculaire spinale
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
WO2021077000A1 (en) 2019-10-16 2021-04-22 The Broad Institute, Inc. Engineered muscle targeting compositions
AU2021225957A1 (en) 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2

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FR2802206B1 (fr) 1999-12-14 2005-04-22 Sod Conseils Rech Applic Derives de 4-aminopiperidine et leur utilisation en tant que medicament
KR20020097241A (ko) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
PT3308788T (pt) 2005-06-23 2019-01-30 Cold Spring Harbor Laboratory Composições e métodos para a modulação do splicing de smn2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
EP2024499B1 (en) 2006-05-10 2017-10-25 Sarepta Therapeutics, Inc. Oligonucleotide analogs having cationic intersubunit linkages
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AU2010262862C1 (en) * 2009-06-17 2020-04-30 Biogen Ma Inc. Compositions and methods for modulation of SMN2 splicing in a subject
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JP5585822B2 (ja) 2010-05-11 2014-09-10 東レ・ファインケミカル株式会社 光学活性ニペコチン酸誘導体の製造方法
KR101829644B1 (ko) 2010-05-28 2018-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US9518259B2 (en) * 2010-06-15 2016-12-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
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CA2858576A1 (en) * 2011-12-06 2013-06-13 Research Institute At Nationwide Children's Hospital Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
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KR20150030205A (ko) * 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
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EP3341480A1 (en) 2018-07-04
US12121532B2 (en) 2024-10-22
US20250241940A1 (en) 2025-07-31
AU2016317667A1 (en) 2018-03-22
JP2021048877A (ja) 2021-04-01
CA2996164A1 (en) 2017-03-09
US20190015440A1 (en) 2019-01-17
HK1257498A1 (zh) 2019-10-25
MA42695A (fr) 2018-07-04
US10905709B2 (en) 2021-02-02
JP2018525015A (ja) 2018-09-06
US20210169918A1 (en) 2021-06-10
WO2017040271A1 (en) 2017-03-09

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