JP7627495B2 - ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド - Google Patents

ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド Download PDF

Info

Publication number
JP7627495B2
JP7627495B2 JP2021533025A JP2021533025A JP7627495B2 JP 7627495 B2 JP7627495 B2 JP 7627495B2 JP 2021533025 A JP2021533025 A JP 2021533025A JP 2021533025 A JP2021533025 A JP 2021533025A JP 7627495 B2 JP7627495 B2 JP 7627495B2
Authority
JP
Japan
Prior art keywords
dux4
oligonucleotide
pharma
acceptable salt
gene
Prior art date
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.)
Active
Application number
JP2021533025A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2021010301A5 (https=
JPWO2021010301A1 (https=
Inventor
誠 小泉
晃史 中村
隆廣 片桐
弘明 三橋
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Tokai University Educational System
Original Assignee
Tokai University Educational System
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 by Tokai University Educational System filed Critical Tokai University Educational System
Publication of JPWO2021010301A1 publication Critical patent/JPWO2021010301A1/ja
Publication of JPWO2021010301A5 publication Critical patent/JPWO2021010301A5/ja
Application granted granted Critical
Publication of JP7627495B2 publication Critical patent/JP7627495B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K31/712—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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • 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
    • C12N2510/00—Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Cell Biology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021533025A 2019-07-12 2020-07-10 ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド Active JP7627495B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019129735 2019-07-12
JP2019129735 2019-07-12
PCT/JP2020/026950 WO2021010301A1 (ja) 2019-07-12 2020-07-10 ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド

Publications (3)

Publication Number Publication Date
JPWO2021010301A1 JPWO2021010301A1 (https=) 2021-01-21
JPWO2021010301A5 JPWO2021010301A5 (https=) 2023-07-25
JP7627495B2 true JP7627495B2 (ja) 2025-02-06

Family

ID=74210830

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021533025A Active JP7627495B2 (ja) 2019-07-12 2020-07-10 ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド

Country Status (11)

Country Link
US (1) US20220364086A1 (https=)
EP (1) EP3998108A4 (https=)
JP (1) JP7627495B2 (https=)
KR (1) KR20220032004A (https=)
CN (1) CN114026234A (https=)
AU (1) AU2020313255A1 (https=)
BR (1) BR112021024764A2 (https=)
CA (1) CA3142925A1 (https=)
IL (1) IL288596A (https=)
TW (1) TW202117015A (https=)
WO (1) WO2021010301A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230023612A (ko) 2020-04-02 2023-02-17 마이레큘, 인크. 조작된 올리고뉴클레오티드를 사용한 표적화된 억제

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017147467A1 (en) 2016-02-26 2017-08-31 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inducing dux4 exon skipping

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0787982A (ja) 1993-01-29 1995-04-04 Sankyo Co Ltd 修飾オリゴデオキシリボヌクレオチド
US6096881A (en) 1997-05-30 2000-08-01 Hybridon, Inc. Sulfur transfer reagents for oligonucleotide synthesis
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
ES2596977T3 (es) * 2010-09-02 2017-01-13 Université de Mons Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral
EP2736539B1 (en) * 2011-07-25 2017-08-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013120038A2 (en) * 2012-02-10 2013-08-15 Regents Of The University Of Minnesota Morpholino targeting dux4 for treating fshd
WO2014109384A1 (ja) 2013-01-10 2014-07-17 塩野義製薬株式会社 架橋型核酸誘導体の製造方法
US20180237775A1 (en) * 2015-09-21 2018-08-23 Association Institut De Myologie Antisense oligonucleotides and uses thereof
CN109462995A (zh) 2016-05-04 2019-03-12 波涛生命科学有限公司 生物活性剂的方法和组合物
WO2019060432A2 (en) * 2017-09-19 2019-03-28 Children's National Medical Center GAPMERS AND METHODS OF USING THE SAME FOR THE TREATMENT OF MUSCLE DYSTROPHY
JP2019129735A (ja) 2018-01-30 2019-08-08 株式会社ＧＩＳｕｐｐｌｙ 有害動物の捕獲支援システム

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017147467A1 (en) 2016-02-26 2017-08-31 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inducing dux4 exon skipping

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ANSSEAU, E. et al.，Antisense Oligonucleotides Used to Target the DUX4 mRNA as Therapeutic Approaches in FaciosScapuloHumeral Muscular Dystrophy(FSHD)，Genes，2017年，8,93，1-21，doi:10.3390/genes8030093
VANDERPLANCK, C. et al.，KEYNOTE 5: SUPPRESSION OF DUX4 OR DUX4C EXPRESSION BY ANTISENSE STRATEGIES IN A THERAPEUTIC APPROACH FOR FSHD，THE JOURNAL OF GENE MEDICINE，2011年，13，p.414，DOI: 10.1002/jgm.1582
VANDERPLANCK, C. et al.，The FSHD Atrophic Myotube Phenotype Is Caused by DUX4 Expression，PLoS ONE，2011年10月28日，Volume 6, Issue 10，e26820，doi:10.1371/journal.pone.0026820

Also Published As

Publication number Publication date
CN114026234A (zh) 2022-02-08
JPWO2021010301A1 (https=) 2021-01-21
BR112021024764A2 (pt) 2022-04-19
WO2021010301A1 (ja) 2021-01-21
IL288596A (en) 2022-02-01
KR20220032004A (ko) 2022-03-15
AU2020313255A1 (en) 2022-01-06
US20220364086A1 (en) 2022-11-17
EP3998108A4 (en) 2024-10-30
TW202117015A (zh) 2021-05-01
EP3998108A1 (en) 2022-05-18
CA3142925A1 (en) 2021-01-21

Similar Documents

Publication Publication Date Title
EP2135948B1 (en) ENA nucleic acid drugs modifying splicing in mRNA precursor
JP2022036182A (ja) Ｂ型肝炎感染症治療用のＰＡＰＤ５又はＰＡＰＤ７のｍＲＮＡ減少用核酸分子
JP7416852B2 (ja) Ｈｔｒａ１の発現を調節するためのアンチセンスオリゴヌクレオチド
JP7627495B2 (ja) ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド
JP6934695B2 (ja) 核酸医薬とその使用
JPWO2019009299A1 (ja) α−シヌクレイン発現抑制するＥＮＡアンチセンスオリゴヌクレオチド
HK40065915A (en) Antisense oligonucleotide capable of altering splicing of dux4 pre-mrna
EP3816286A1 (en) Myocardial dysfunction therapeutic agent
JP2025114590A (ja) 色素性乾皮症ｆ群治療薬
JP7072748B2 (ja) アルポート症候群治療薬
TW202600822A (zh) 抑制冠狀病毒之增殖的反義寡核苷酸
JP6536911B2 (ja) ＣＤ４４遺伝子のバリアントエクソンのスキッピングを誘導し、正常型ＣＤ４４ｍＲＮＡの発現を増加させる核酸医薬
HK40040396A (en) Myocardial dysfunction therapeutic agent
WO2019111791A1 (ja) ジストロフィン遺伝子のイントロンリテンションを解消するアンチセンスオリゴヌクレオチド
HK40015131A (en) Therapeutic drug for alport's syndrome

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20210913

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20210913

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230619

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20230619

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230626

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230626

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20230619

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240529

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240724

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240911

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250108

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250120

R150 Certificate of patent or registration of utility model

Ref document number: 7627495

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150