CO2023003810A2 - Oligonucleótidos antisentido que se dirigen al exón 51 del gen de la distrofina - Google Patents

Oligonucleótidos antisentido que se dirigen al exón 51 del gen de la distrofina

Info

Publication number
CO2023003810A2
CO2023003810A2 CONC2023/0003810A CO2023003810A CO2023003810A2 CO 2023003810 A2 CO2023003810 A2 CO 2023003810A2 CO 2023003810 A CO2023003810 A CO 2023003810A CO 2023003810 A2 CO2023003810 A2 CO 2023003810A2
Authority
CO
Colombia
Prior art keywords
antisense oligonucleotides
dystrophin gene
oligonucleotides targeting
targeting exon
exon
Prior art date
Application number
CONC2023/0003810A
Other languages
English (en)
Inventor
Deutekom Judith Christina Theodora Van
Visser Peter Christian De
Original Assignee
Biomarin Tech Bv
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 Biomarin Tech Bv filed Critical Biomarin Tech Bv
Publication of CO2023003810A2 publication Critical patent/CO2023003810A2/es

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    • 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
    • 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
    • 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
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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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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    • 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
    • C12N2310/32—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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
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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
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    • C12N2310/335—Modified T or U
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se proporcionan oligonucleótidos antisentido hidroxialcoxilados de cambio de empalme para prevenir, tratar y/o retrasar trastornos neuromusculares, más específicamente la distrofia muscular de Duchenne.
CONC2023/0003810A 2020-09-30 2023-03-27 Oligonucleótidos antisentido que se dirigen al exón 51 del gen de la distrofina CO2023003810A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063085668P 2020-09-30 2020-09-30
PCT/EP2021/076738 WO2022069511A1 (en) 2020-09-30 2021-09-29 Antisense oligonucleotides targeting the exon 51 of dystrophin gene

Publications (1)

Publication Number Publication Date
CO2023003810A2 true CO2023003810A2 (es) 2023-07-10

Family

ID=78049251

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2023/0003810A CO2023003810A2 (es) 2020-09-30 2023-03-27 Oligonucleótidos antisentido que se dirigen al exón 51 del gen de la distrofina

Country Status (15)

Country Link
US (1) US20220098586A1 (es)
EP (1) EP4222262A1 (es)
JP (1) JP2023543495A (es)
KR (1) KR20230079183A (es)
CN (1) CN117015604A (es)
AR (1) AR123640A1 (es)
AU (1) AU2021354760A1 (es)
CA (1) CA3193919A1 (es)
CL (1) CL2023000949A1 (es)
CO (1) CO2023003810A2 (es)
IL (1) IL301781A (es)
MX (1) MX2023003609A (es)
TW (1) TW202229550A (es)
UY (1) UY39444A (es)
WO (1) WO2022069511A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023265665A1 (en) 2022-05-05 2024-10-10 Biomarin Pharmaceutical Inc. Method of treating duchenne muscular dystrophy

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013518603A (ja) 2010-02-08 2013-05-23 アイシス ファーマシューティカルズ， インコーポレーテッド 反復伸張に関連する疾患または病態の治療に有用な方法および組成物
WO2014112463A1 (ja) 2013-01-15 2014-07-24 国立大学法人大阪大学 スルホンアミド構造を有するヌクレオシドおよびヌクレオチド
WO2014126229A1 (ja) 2013-02-18 2014-08-21 塩野義製薬株式会社 含窒素複素環構造を有するヌクレオシド及びヌクレオチド
CA2906209A1 (en) * 2013-03-14 2014-09-25 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy targeting the annealing site h44a (-07+15)
CN105189751B (zh) 2013-03-15 2019-04-23 米拉根医疗股份有限公司 桥接双环核苷
EP3119789B1 (en) 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
WO2016017422A1 (ja) 2014-07-31 2016-02-04 国立大学法人大阪大学 架橋型ヌクレオシドおよびヌクレオチド
EP3359668A4 (en) * 2015-10-09 2019-06-05 Sarepta Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods thereof
MA45290A (fr) * 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
AU2017291960B2 (en) 2016-07-05 2024-03-14 Biomarin Technologies B.V. Pre-mRNA splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide

Also Published As

Publication number Publication date
TW202229550A (zh) 2022-08-01
MX2023003609A (es) 2023-04-05
CL2023000949A1 (es) 2023-11-10
CA3193919A1 (en) 2022-04-07
WO2022069511A1 (en) 2022-04-07
AR123640A1 (es) 2022-12-28
CN117015604A (zh) 2023-11-07
IL301781A (en) 2023-05-01
KR20230079183A (ko) 2023-06-05
AU2021354760A1 (en) 2023-05-11
UY39444A (es) 2022-04-29
US20220098586A1 (en) 2022-03-31
JP2023543495A (ja) 2023-10-16
AU2021354760A9 (en) 2023-07-06
EP4222262A1 (en) 2023-08-09

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