CL2023000949A1 - 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

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Publication number
CL2023000949A1
CL2023000949A1 CL2023000949A CL2023000949A CL2023000949A1 CL 2023000949 A1 CL2023000949 A1 CL 2023000949A1 CL 2023000949 A CL2023000949 A CL 2023000949A CL 2023000949 A CL2023000949 A CL 2023000949A CL 2023000949 A1 CL2023000949 A1 CL 2023000949A1
Authority
CL
Chile
Prior art keywords
antisense oligonucleotides
dystrophin gene
target exon
exon
target
Prior art date
Application number
CL2023000949A
Other languages
English (en)
Inventor
Christina Theodora Van Deutekom Judith
Christian De Visser Peter
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 CL2023000949A1 publication Critical patent/CL2023000949A1/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
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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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    • C12N2310/33—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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    • C12N2310/335—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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    • C12N2310/30—Chemical structure
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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)
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  • 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)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (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.
CL2023000949A 2020-09-30 2023-03-30 Oligonucleótidos antisentido que se dirigen al exón 51 del gen de la distrofina CL2023000949A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063085668P 2020-09-30 2020-09-30

Publications (1)

Publication Number Publication Date
CL2023000949A1 true CL2023000949A1 (es) 2023-11-10

Family

ID=78049251

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2023000949A CL2023000949A1 (es) 2020-09-30 2023-03-30 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
US20230416741A1 (en) 2022-05-05 2023-12-28 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
US20130059902A1 (en) 2010-02-08 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
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
US9315535B2 (en) 2013-02-18 2016-04-19 Shionogi & Co., Ltd. Nucleoside and nucleotide having nitrogen-containing heterocycle structure
BR122020016865B1 (pt) * 2013-03-14 2022-12-27 Sarepta Therapeutics, Inc. Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
MX364814B (es) 2013-03-15 2019-05-08 Miragen Therapeutics Inc Nucleosidos biciclicos unidos en puente.
WO2015142910A1 (en) 2014-03-17 2015-09-24 Isis 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
TW201722439A (zh) 2015-10-09 2017-07-01 波濤生命科學有限公司 寡核苷酸組合物及其方法
BR112018007066A2 (pt) * 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
MA45290A (fr) * 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
WO2018007475A1 (en) 2016-07-05 2018-01-11 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
UY39444A (es) 2022-04-29
IL301781A (en) 2023-05-01
MX2023003609A (es) 2023-04-05
CN117015604A (zh) 2023-11-07
AU2021354760A9 (en) 2023-07-06
EP4222262A1 (en) 2023-08-09
AU2021354760A1 (en) 2023-05-11
CO2023003810A2 (es) 2023-07-10
KR20230079183A (ko) 2023-06-05
US20220098586A1 (en) 2022-03-31
WO2022069511A1 (en) 2022-04-07
CA3193919A1 (en) 2022-04-07
AR123640A1 (es) 2022-12-28
JP2023543495A (ja) 2023-10-16
TW202229550A (zh) 2022-08-01

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