JPWO2021172498A1 - - Google Patents

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Publication number
JPWO2021172498A1
JPWO2021172498A1 JP2022503739A JP2022503739A JPWO2021172498A1 JP WO2021172498 A1 JPWO2021172498 A1 JP WO2021172498A1 JP 2022503739 A JP2022503739 A JP 2022503739A JP 2022503739 A JP2022503739 A JP 2022503739A JP WO2021172498 A1 JPWO2021172498 A1 JP WO2021172498A1
Authority
JP
Japan
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2022503739A
Other versions
JPWO2021172498A5 (ja
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed filed Critical
Publication of JPWO2021172498A1 publication Critical patent/JPWO2021172498A1/ja
Priority to JP2022141656A priority Critical patent/JP7292636B2/ja
Publication of JPWO2021172498A5 publication Critical patent/JPWO2021172498A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/32—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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
JP2022503739A 2020-02-28 2021-02-26 Pending JPWO2021172498A1 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022141656A JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン５１のスキッピングを誘導するアンチセンス核酸

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2020033483 2020-02-28
PCT/JP2021/007286 WO2021172498A1 (ja) 2020-02-28 2021-02-26 エクソン５１のスキッピングを誘導するアンチセンス核酸

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022141656A Division JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン５１のスキッピングを誘導するアンチセンス核酸

Publications (2)

Publication Number Publication Date
JPWO2021172498A1 true JPWO2021172498A1 (ja) 2021-09-02
JPWO2021172498A5 JPWO2021172498A5 (ja) 2024-03-04

Family

ID=77491251

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022503739A Pending JPWO2021172498A1 (ja) 2020-02-28 2021-02-26
JP2022141656A Active JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン５１のスキッピングを誘導するアンチセンス核酸

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022141656A Active JP7292636B2 (ja) 2020-02-28 2022-09-06 エクソン５１のスキッピングを誘導するアンチセンス核酸

Country Status (16)

Country Link
US (2) US20230140736A1 (ja)
EP (1) EP4112083A1 (ja)
JP (2) JPWO2021172498A1 (ja)
KR (1) KR20220145865A (ja)
CN (1) CN115210376A (ja)
AU (1) AU2021226089A1 (ja)
BR (1) BR112022017066A2 (ja)
CA (1) CA3173049A1 (ja)
CL (1) CL2022002279A1 (ja)
CO (1) CO2022013685A2 (ja)
EC (1) ECSP22074446A (ja)
IL (1) IL295967A (ja)
MX (1) MX2022010545A (ja)
PE (1) PE20230237A1 (ja)
TW (1) TW202200162A (ja)
WO (1) WO2021172498A1 (ja)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
DE69033986T2 (de) 1989-12-20 2003-03-13 Antivirals Inc Ungeladene, auf Morpholin basierende Polymere mit Chiralen, Phosphor enthaltenden Brücken zwischen den Untereinheiten
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
IL115849A0 (en) 1994-11-03 1996-01-31 Merz & Co Gmbh & Co Tangential filtration preparation of liposomal drugs and liposome product thereof
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
CA2507125C (en) 2002-11-25 2014-04-22 Masafumi Matsuo Ena nucleic acid pharmaceuticals capable of modifying splicing of mrna precursors
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP4272748A3 (en) 2004-06-28 2024-03-27 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
JP2010505741A (ja) 2006-05-10 2010-02-25 エイブイアイ バイオファーマ， インコーポレイテッド カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ
ES2694726T3 (es) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
EP2203173B1 (en) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2009064471A1 (en) 2007-11-15 2009-05-22 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
JP5864257B2 (ja) 2008-10-24 2016-02-17 サレプタ セラピューティクス， インコーポレイテッド Ｄｍｄのための複数のエキソンスキッピング組成物
ES2532634T5 (es) 2008-10-27 2018-04-30 Biomarin Technologies B.V. Procedimientos y medios para el salto eficiente del exón 45 en el pre-ARNm de la distrofia muscular de Duchenne
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
KR102339196B1 (ko) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
CN104203289B (zh) 2012-01-27 2020-11-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
JP6519842B2 (ja) 2013-10-04 2019-05-29 国立大学法人神戸大学 福山型筋ジストロフィー治療用アンチセンス核酸
RU2730681C2 (ru) 2014-03-12 2020-08-24 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
WO2016187425A1 (en) 2015-05-19 2016-11-24 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
HUE057593T2 (hu) 2015-08-05 2022-05-28 Eisai R&D Man Co Ltd Eljárás lényegében diasztereomeresen tiszta foszfor-diamidát oligomer elõállítására, az eljárással készített foszfor-diamidát oligomer és a foszfor-diamidát oligomert tartalmazó gyógyszerészeti készítmény
CR20180233A (es) 2015-10-09 2018-05-25 Wave Life Sciences Ltd Composiciones oligonucleotídicas y sus métodos
EP3485015A4 (en) * 2016-07-15 2020-07-29 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS OF MODULATION OF DYSTROPHINE TRANSCRIPT
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WO2018118627A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
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WO2019241385A2 (en) 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy

Also Published As

Publication number Publication date
MX2022010545A (es) 2022-09-21
CO2022013685A2 (es) 2022-10-11
US11781140B2 (en) 2023-10-10
US20230097387A1 (en) 2023-03-30
BR112022017066A2 (pt) 2022-11-16
PE20230237A1 (es) 2023-02-07
WO2021172498A1 (ja) 2021-09-02
KR20220145865A (ko) 2022-10-31
CN115210376A (zh) 2022-10-18
US20230140736A1 (en) 2023-05-04
IL295967A (en) 2022-10-01
ECSP22074446A (es) 2022-10-31
CA3173049A1 (en) 2021-09-02
JP2022180420A (ja) 2022-12-06
EP4112083A1 (en) 2023-01-04
AU2021226089A1 (en) 2022-09-15
JP7292636B2 (ja) 2023-06-19
CL2022002279A1 (es) 2023-02-10
TW202200162A (zh) 2022-01-01

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