AR123640A1 - Oligonucleótido - Google Patents

Oligonucleótido

Info

Publication number
AR123640A1
AR123640A1 ARP210102703A ARP210102703A AR123640A1 AR 123640 A1 AR123640 A1 AR 123640A1 AR P210102703 A ARP210102703 A AR P210102703A AR P210102703 A ARP210102703 A AR P210102703A AR 123640 A1 AR123640 A1 AR 123640A1
Authority
AR
Argentina
Prior art keywords
seq
ethylene glycol
hydroxyalkoxylated
antisense oligonucleotide
sequence nucleotide
Prior art date
Application number
ARP210102703A
Other languages
English (en)
Inventor
Visser Peter Christian De
Deutekom Judith Christina Theodora Van
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 AR123640A1 publication Critical patent/AR123640A1/es

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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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • 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
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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
    • 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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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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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    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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

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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)
  • 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 proveen oligonucleótidos antisentido hidroxialcoxilados interruptores de corte y empalme, para prevenir, tratar y/o demorar trastornos neuromusculares, más específicamente Distrofia Muscular de Duchene. Reivindicación 1: Un ONA hidroxialcoxilado que consiste en un oligonucleótido antisentido y uno o dos grupos hidroxialcoxi, en donde dicho grupo hidroxialcoxi comprende o consiste en un monómero etilenglicol, oligómero de etilenglicol o polímero de etilenglicol, en donde el oligonucleótido antisentido está representado por una secuencia nucleotídica que comprende o consiste en: i) una cualquiera de las SEQ ID Nº 9 - 404, o ii) un fragmento de una cualquiera de las SEQ ID Nº 9 - 404, o iii) una cualquiera de las SEQ ID Nº 9 - 404 con 1, 2, 3, 4, ó 5 nucleótidos adicionales, o iv) una cualquiera de las SEQ ID Nº 9 - 404 con 1, 2, 3, 4, ó 5 nucleótidos faltantes de dichas SEQ ID Nº, o v) una secuencia nucleotídica que tiene al menos 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98% o 99%, o al menos 95%, o al menos 97% de identidad con una cualquiera de las SEQ ID Nº 9 - 404.
ARP210102703A 2020-09-30 2021-09-29 Oligonucleótido AR123640A1 (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
AR123640A1 true AR123640A1 (es) 2022-12-28

Family

ID=78049251

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210102703A AR123640A1 (es) 2020-09-30 2021-09-29 Oligonucleótido

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
CL2023000949A1 (es) 2023-11-10
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
JP2023543495A (ja) 2023-10-16
TW202229550A (zh) 2022-08-01

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