MX2021004822A - Oligonucleotidos antisentido biespecificos para la omision del exon de la distrofina. - Google Patents

Oligonucleotidos antisentido biespecificos para la omision del exon de la distrofina.

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Publication number
MX2021004822A
MX2021004822A MX2021004822A MX2021004822A MX2021004822A MX 2021004822 A MX2021004822 A MX 2021004822A MX 2021004822 A MX2021004822 A MX 2021004822A MX 2021004822 A MX2021004822 A MX 2021004822A MX 2021004822 A MX2021004822 A MX 2021004822A
Authority
MX
Mexico
Prior art keywords
antisense oligonucleotides
exon skipping
dystrophin exon
bispecific antisense
bispecific
Prior art date
Application number
MX2021004822A
Other languages
English (en)
Inventor
Deutekom Judith Christina Theodora Van
Nicole Ann Datson
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 MX2021004822A publication Critical patent/MX2021004822A/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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • C12N2310/30—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
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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
    • 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
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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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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

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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)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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

La presente invención proporciona compuestos que modifican el empalme con características mejoradas que potencian la aplicabilidad clínica preferentemente para el tratamiento, mejora, prevención y/o retraso de los trastornos neuromusculares, más específicamente DMD.
MX2021004822A 2018-11-02 2019-10-30 Oligonucleotidos antisentido biespecificos para la omision del exon de la distrofina. MX2021004822A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18204170 2018-11-02
PCT/EP2019/079714 WO2020089325A1 (en) 2018-11-02 2019-10-30 Bispecific antisense oligonucleotides for dystrophin exon skipping

Publications (1)

Publication Number Publication Date
MX2021004822A true MX2021004822A (es) 2021-07-06

Family

ID=64172289

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021004822A MX2021004822A (es) 2018-11-02 2019-10-30 Oligonucleotidos antisentido biespecificos para la omision del exon de la distrofina.

Country Status (12)

Country Link
US (1) US20220025368A1 (es)
EP (1) EP3874044A1 (es)
JP (1) JP2022506219A (es)
KR (1) KR20210091180A (es)
CN (1) CN113286887A (es)
AU (1) AU2019370937A1 (es)
BR (1) BR112021008069A2 (es)
CA (1) CA3118167A1 (es)
IL (1) IL282818A (es)
MX (1) MX2021004822A (es)
SG (1) SG11202103938UA (es)
WO (1) WO2020089325A1 (es)

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WO2022232478A1 (en) 2021-04-30 2022-11-03 Sarepta Therapeutics, Inc. Treatment methods for muscular dystrophy
CN114540461A (zh) * 2022-01-26 2022-05-27 深圳市儿童医院 一种plga微球-crispr免疫共沉淀法检测杜氏肌萎缩症的试剂盒
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2023215781A1 (en) * 2022-05-05 2023-11-09 Biomarin Pharmaceutical Inc. Method of treating duchenne muscular dystrophy

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DK1766010T3 (da) * 2004-06-28 2011-06-06 Univ Western Australia Antisense-oligonukleotider til induktion af exon-skipping og fremgangsmåder til anvendelse deraf
EP1857548A1 (en) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
KR102366851B1 (ko) * 2009-11-12 2022-02-23 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
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EP2699269A1 (en) * 2011-04-22 2014-02-26 Prosensa Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1)
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PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
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WO2014043544A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
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JP6292631B2 (ja) 2013-02-18 2018-03-14 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
WO2014145356A1 (en) 2013-03-15 2014-09-18 MiRagen Therapeutics, Inc. Bridged bicyclic nucleosides
RU2702424C2 (ru) * 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
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Also Published As

Publication number Publication date
JP2022506219A (ja) 2022-01-17
US20220025368A1 (en) 2022-01-27
AU2019370937A1 (en) 2021-05-27
WO2020089325A1 (en) 2020-05-07
EP3874044A1 (en) 2021-09-08
CN113286887A (zh) 2021-08-20
KR20210091180A (ko) 2021-07-21
BR112021008069A2 (pt) 2021-11-03
CA3118167A1 (en) 2020-05-07
IL282818A (en) 2021-06-30
SG11202103938UA (en) 2021-05-28

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