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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    • 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
    • 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
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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)
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  • 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 (OSRAM) 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 (OSRAM)
EP (1) EP3341480A1 (OSRAM)
JP (2) JP6987041B2 (OSRAM)
AU (1) AU2016317667A1 (OSRAM)
CA (1) CA2996164A1 (OSRAM)
HK (1) HK1257498A1 (OSRAM)
MA (1) MA42695A (OSRAM)
WO (1) WO2017040271A1 (OSRAM)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2017040271A1 (en) 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
JP7394753B2 (ja) * 2017-10-18 2023-12-08 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマー化合物
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular 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
DK1910395T3 (da) * 2005-06-23 2013-02-04 Isis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US7943762B2 (en) 2006-05-10 2011-05-17 Avi Biopharma, Inc. Oligonucleotide analogs having cationic intersubunit linkages
ES2373246T3 (es) 2006-08-11 2012-02-01 Prosensa Technologies B.V. Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn.
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2010120820A1 (en) * 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of smn2 splicing
SI3305302T1 (sl) * 2009-06-17 2018-12-31 Biogen Ma Inc. Sestave in metode za modulacijo združevanja smn2 pri subjektu
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
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
CA2799501C (en) 2010-05-28 2022-02-15 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
EP2582397A4 (en) 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
KR102229650B1 (ko) * 2011-05-05 2021-03-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
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JP6317675B2 (ja) 2011-11-30 2018-04-25 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
US9725716B2 (en) * 2011-12-06 2017-08-08 Ohio State Innovation Foundation and 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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Publication number Publication date
AU2016317667A1 (en) 2018-03-22
US12121532B2 (en) 2024-10-22
EP3341480A1 (en) 2018-07-04
MA42695A (fr) 2018-07-04
HK1257498A1 (zh) 2019-10-25
JP2021048877A (ja) 2021-04-01
WO2017040271A1 (en) 2017-03-09
US20190015440A1 (en) 2019-01-17
CA2996164A1 (en) 2017-03-09
US10905709B2 (en) 2021-02-02
JP2018525015A (ja) 2018-09-06
US20250241940A1 (en) 2025-07-31
US20210169918A1 (en) 2021-06-10

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