JPWO2021125311A5 - - Google Patents

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JPWO2021125311A5
JPWO2021125311A5 JP2021565671A JP2021565671A JPWO2021125311A5 JP WO2021125311 A5 JPWO2021125311 A5 JP WO2021125311A5 JP 2021565671 A JP2021565671 A JP 2021565671A JP 2021565671 A JP2021565671 A JP 2021565671A JP WO2021125311 A5 JPWO2021125311 A5 JP WO2021125311A5
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JP
Japan
Prior art keywords
base sequence
region
bases
exon
antisense oligomer
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JP2021565671A
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English (en)
Japanese (ja)
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JP7842395B2 (ja
JPWO2021125311A1 (https=
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Priority claimed from PCT/JP2020/047340 external-priority patent/WO2021125311A1/ja
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Publication of JPWO2021125311A5 publication Critical patent/JPWO2021125311A5/ja
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JP2021565671A 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 Active JP7842395B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019229763 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

Publications (3)

Publication Number Publication Date
JPWO2021125311A1 JPWO2021125311A1 (https=) 2021-06-24
JPWO2021125311A5 true JPWO2021125311A5 (https=) 2023-12-18
JP7842395B2 JP7842395B2 (ja) 2026-04-08

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JP2021565671A Active JP7842395B2 (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

Country Status (14)

Country Link
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

Family Cites Families (26)

* 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
EP2392660A3 (en) 2002-11-25 2012-03-28 Masafumi Matsuo ENA Nucleic Acid Drugs Modifying Splicing in mRNA Precursor
AU2003225410A1 (en) * 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
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
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
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
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
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

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