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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • 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
    • C12N2310/32—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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base

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  • Physical Education & Sports Medicine (AREA)
  • 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

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

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HK1257498A1 (zh) 2015-08-28 2019-10-25 Sarepta Therapeutics, Inc. 在脊髓性肌肉萎缩症中用於内显子纳入的改良的反义寡聚体
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
AR121446A1 (es) 2020-02-28 2022-06-08 Ionis Pharmaceuticals Inc Compuestos y 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
PL1910395T3 (pl) * 2005-06-23 2013-03-29 Cold Spring Harbor Laboratory Kompozycja i sposób modulacji splicingu SMN2
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
PL2735568T3 (pl) 2006-05-10 2018-02-28 Sarepta Therapeutics, Inc. Analogi oligonukleotydowe mające kationowe połączenia pomiędzy podjednostkami
EP2049664B1 (en) 2006-08-11 2011-09-14 Prosensa Technologies B.V. Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders
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KR102095478B1 (ko) 2010-05-28 2020-04-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
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ES2832531T3 (es) 2011-11-30 2021-06-10 Sarepta Therapeutics Inc Oligonucleótidos para el tratamiento de enfermedades por expansión de repeticiones
AU2012345638C1 (en) * 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
EP2788087A4 (en) * 2011-12-06 2015-08-26 Ohio State Innovation Foundation Non-ionic, low-osmolar contrast agents for the administration of antisense oligonucleotides and the treatment of diseases
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CA2996164A1 (en) 2017-03-09
US12121532B2 (en) 2024-10-22
AU2016317667A1 (en) 2018-03-22
WO2017040271A1 (en) 2017-03-09
EP3341480A1 (en) 2018-07-04
US20250241940A1 (en) 2025-07-31
HK1257498A1 (zh) 2019-10-25
US20190015440A1 (en) 2019-01-17
MA42695A (fr) 2018-07-04
JP2021048877A (ja) 2021-04-01
JP2018525015A (ja) 2018-09-06
US10905709B2 (en) 2021-02-02
US20210169918A1 (en) 2021-06-10

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