KR20220118459A - 엑손 스키핑을 가능하게 하는 안티센스 핵산 - Google Patents

엑손 스키핑을 가능하게 하는 안티센스 핵산 Download PDF

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KR20220118459A
KR20220118459A KR1020227023507A KR20227023507A KR20220118459A KR 20220118459 A KR20220118459 A KR 20220118459A KR 1020227023507 A KR1020227023507 A KR 1020227023507A KR 20227023507 A KR20227023507 A KR 20227023507A KR 20220118459 A KR20220118459 A KR 20220118459A
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나오키 와타나베
유이치로 도네
신이치 다케다
요시츠구 아오키
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니뽄 신야쿠 가부시키가이샤
고쿠리쯔겡뀨가이하쯔호징 고꾸리쯔 세이신ㆍ신께이 이료겡뀨센따
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    • C12N15/09—Recombinant DNA-technology
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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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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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
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    • C12N2320/33—Alteration of splicing

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  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020227023507A 2019-12-19 2020-12-18 엑손 스키핑을 가능하게 하는 안티센스 핵산 Pending KR20220118459A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JPJP-P-2019-229763 2019-12-19
JP2019229763 2019-12-19
PCT/JP2020/047340 WO2021125311A1 (ja) 2019-12-19 2020-12-18 エクソンスキッピングを可能にするアンチセンス核酸

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KR20220118459A true KR20220118459A (ko) 2022-08-25

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KR1020227023507A Pending KR20220118459A (ko) 2019-12-19 2020-12-18 엑손 스키핑을 가능하게 하는 안티센스 핵산

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US (1) US20230045557A1 (https=)
EP (1) EP4079329A4 (https=)
JP (1) JP7842395B2 (https=)
KR (1) KR20220118459A (https=)
CN (1) CN115151642A (https=)
AU (1) AU2020408773A1 (https=)
BR (1) BR112022012114A2 (https=)
CA (1) CA3165316A1 (https=)
CO (1) CO2022009983A2 (https=)
IL (1) IL293997A (https=)
MX (1) MX2022007491A (https=)
PH (1) PH12022551438A1 (https=)
TW (1) TW202136511A (https=)
WO (1) WO2021125311A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4363578A1 (en) * 2021-07-02 2024-05-08 Aarhus Universitet Methods of treating alzheimer's disease
JP2025079347A (ja) * 2022-03-11 2025-05-22 日本新薬株式会社 キャリアペプチドが連結された核酸
EP4680250A1 (en) * 2023-03-17 2026-01-21 The Regents of the University of California Factor viii splice-modulating antisense oligonucleotides and methods of use

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004048570A1 (ja) 2002-11-25 2004-06-10 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
WO2004083446A2 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
WO2006000057A1 (en) 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007135105A1 (en) 2006-05-19 2007-11-29 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2009139630A2 (en) 2008-05-14 2009-11-19 Prosensa Technologies B.V. Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
US20100168212A1 (en) 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU655164B2 (en) 1989-12-20 1994-12-08 Antivirals Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
JP2924179B2 (ja) 1993-02-19 1999-07-26 日本新薬株式会社 グリセロール誘導体，デバイス及び医薬組成物
ES2639852T3 (es) * 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
RU2606627C2 (ru) 2007-11-15 2017-01-10 Серепта Терапьютикс,Инк. Способ синтеза морфолиновых олигомеров
AU2009310557B2 (en) * 2008-10-27 2014-09-11 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in Duchenne Muscular Dystrophy pre-mRNA
TWI541024B (zh) * 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CA2861247C (en) 2011-12-28 2021-11-16 Nippon Shinyaku Co., Ltd. Antisense nucleic acids
NZ627896A (en) * 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
CN110218727A (zh) * 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
DK3118311T3 (en) * 2014-03-12 2019-03-11 Nippon Shinyaku Co Ltd Antisense nucleic acid
HUE050061T2 (hu) * 2015-09-15 2020-12-28 Nippon Shinyaku Co Ltd Antiszensz nukleinsav
MX2019008199A (es) * 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
MX2020003130A (es) * 2017-09-22 2020-09-25 Avidity Biosciences Inc Composiciones y metodos de induccion de salto de exon en acidos nucleicos-polipeptidos.
EP3735462A1 (en) * 2018-01-05 2020-11-11 The Board of Regents of The University of Texas System Therapeutic crispr/cas9 compositions and methods of use
SG11202009877XA (en) * 2018-04-12 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
CA3108282A1 (en) * 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN114901823A (zh) * 2019-12-26 2022-08-12 日本新药株式会社 诱导外显子50的跳读的反义核酸
IL295967A (en) * 2020-02-28 2022-10-01 Nippon Shinyaku Co Ltd Antisense nucleic acids for splicing on exon 51

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004048570A1 (ja) 2002-11-25 2004-06-10 Nonprofit Organization Translational Research Organization Of Duchenne Muscular Dystrophy ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
WO2004083446A2 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
WO2006000057A1 (en) 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007135105A1 (en) 2006-05-19 2007-11-29 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2009139630A2 (en) 2008-05-14 2009-11-19 Prosensa Technologies B.V. Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
US20100168212A1 (en) 2008-09-11 2010-07-01 Royal Holloway, University Of London Oligomers
WO2010048586A1 (en) 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
WO2011057350A1 (en) 2009-11-12 2011-05-19 The University Of Western Australia Antisense molecules and methods for treating pathologies

Non-Patent Citations (10)

* Cited by examiner, † Cited by third party
Title
Annemieke Aartsma-Rus et al, Am. J. Hum. Genet. 74(1), 83-92, 2004
Annemieke Aartsma-Rus et al., (2002) Neuromuscular Disorders 12：S71-S77
Christophe Beroud et al., Human Mutation, 28(2), 2007, 196－202
Joshua Lee et al., Methods in Molecular Biology, 1828, 141-150, 2018
Joshua Lee et al., PLoS ONE, 13(5), e0197084, 2018
Laura van Vliet et al., BMC Medical Genetics, 9, 105, 2008
Wilton S. D., e t al., Molecular Therapy 2007：15：p. 1288-96
Yoshitsugu Aoki et al., PNAS, 109(34), 2012, 13763-13768
Yusuke Echigoya et al., Molecular Therapy, 27(11), 1-13, 2019
Yusuke Echigoya et al., Molecular Therapy-Nucleic Acids, 4(2), 2015, e225

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Publication number Publication date
CN115151642A (zh) 2022-10-04
PH12022551438A1 (en) 2023-11-20
JP7842395B2 (ja) 2026-04-08
CA3165316A1 (en) 2021-06-24
AU2020408773A1 (en) 2022-07-14
MX2022007491A (es) 2022-08-15
JPWO2021125311A1 (https=) 2021-06-24
IL293997A (en) 2022-08-01
WO2021125311A1 (ja) 2021-06-24
CO2022009983A2 (es) 2022-07-19
EP4079329A4 (en) 2024-04-17
TW202136511A (zh) 2021-10-01
US20230045557A1 (en) 2023-02-09
EP4079329A1 (en) 2022-10-26
BR112022012114A2 (pt) 2022-09-06

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