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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JP2018525015A (ja
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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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • 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
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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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  • 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 (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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US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
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
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法

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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
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
EP3470072A1 (en) * 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
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
SI2049664T1 (sl) 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
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
ES2699827T3 (es) * 2009-06-17 2019-02-13 Biogen Ma Inc Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto
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
KR20200133284A (ko) * 2010-05-28 2020-11-26 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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US10066228B2 (en) 2011-11-30 2018-09-04 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
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CN104411831B (zh) 2012-03-20 2020-08-11 萨勒普塔医疗公司 寡核苷酸类似物的硼酸缀合物
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
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Publication number Publication date
JP2018525015A (ja) 2018-09-06
EP3341480A1 (en) 2018-07-04
AU2016317667A1 (en) 2018-03-22
WO2017040271A1 (en) 2017-03-09
US20250241940A1 (en) 2025-07-31
US10905709B2 (en) 2021-02-02
US12121532B2 (en) 2024-10-22
HK1257498A1 (zh) 2019-10-25
CA2996164A1 (en) 2017-03-09
US20210169918A1 (en) 2021-06-10
MA42695A (fr) 2018-07-04
US20190015440A1 (en) 2019-01-17
JP2021048877A (ja) 2021-04-01

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