BR112021024764A2 - Oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4 - Google Patents

Oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4

Info

Publication number
BR112021024764A2
BR112021024764A2 BR112021024764A BR112021024764A BR112021024764A2 BR 112021024764 A2 BR112021024764 A2 BR 112021024764A2 BR 112021024764 A BR112021024764 A BR 112021024764A BR 112021024764 A BR112021024764 A BR 112021024764A BR 112021024764 A2 BR112021024764 A2 BR 112021024764A2
Authority
BR
Brazil
Prior art keywords
dux4
antisense oligonucleotide
mrna splicing
oligonucleotide capable
altering
Prior art date
Application number
BR112021024764A
Other languages
English (en)
Portuguese (pt)
Inventor
Akifumi Nakamura
Hiroaki Mitsuhashi
Makoto Koizumi
Takahiro Katagiri
Original Assignee
Daiichi Sankyo Co Ltd
Tokai Univ 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 Daiichi Sankyo Co Ltd, Tokai Univ Educational System filed Critical Daiichi Sankyo Co Ltd
Publication of BR112021024764A2 publication Critical patent/BR112021024764A2/pt

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-R Modification
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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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • 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

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  • 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)
BR112021024764A 2019-07-12 2020-07-10 Oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4 BR112021024764A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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 (1)

Publication Number Publication Date
BR112021024764A2 true BR112021024764A2 (pt) 2022-04-19

Family

ID=74210830

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021024764A BR112021024764A2 (pt) 2019-07-12 2020-07-10 Oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4

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

Family Cites Families (14)

* 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
JP6966463B2 (ja) * 2016-02-26 2021-11-17 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 組換えウイルス産物及びｄｕｘ４エクソンスキッピングを誘導するための方法
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

Also Published As

Publication number Publication date
CN114026234A (zh) 2022-02-08
JP7627495B2 (ja) 2025-02-06
JPWO2021010301A1 (https=) 2021-01-21
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

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