MX2023006467A - Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos. - Google Patents

Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos.

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Publication number
MX2023006467A
MX2023006467A MX2023006467A MX2023006467A MX2023006467A MX 2023006467 A MX2023006467 A MX 2023006467A MX 2023006467 A MX2023006467 A MX 2023006467A MX 2023006467 A MX2023006467 A MX 2023006467A MX 2023006467 A MX2023006467 A MX 2023006467A
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Mexico
Prior art keywords
treatment
improved characteristics
genetic disorders
mrna splice
splice switching
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MX2023006467A
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English (en)
Inventor
Deutekom Judith Christina Theodora Van
Visser Peter Christian De
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Biomarin Tech Bv
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Publication date
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Publication of MX2023006467A publication Critical patent/MX2023006467A/es

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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/34—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
    • 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
    • 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
    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-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
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/336—Modified G
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Plant Pathology (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La presente invención proporciona oligonucleótidos antisentido de cambio de empalme con características mejoradas que mejoran la aplicabilidad clínica para tratar, mejorar, prevenir y/o retrasar los trastornos neuromusculares, tal como la Distrofia Muscular de Duchenne.
MX2023006467A 2016-07-05 2018-12-19 Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos. MX2023006467A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP16177973 2016-07-05

Publications (1)

Publication Number Publication Date
MX2023006467A true MX2023006467A (es) 2023-06-16

Family

ID=56360269

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018016253A MX2018016253A (es) 2016-07-05 2017-07-05 Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos.
MX2023006467A MX2023006467A (es) 2016-07-05 2018-12-19 Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos.

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MX2018016253A MX2018016253A (es) 2016-07-05 2017-07-05 Oligonucleotidos de conmutacion o modulacion de empalme de pre-arnm que comprenden radicales de andamio biciclicos, con caracteristicas mejoradas para el tratamiento de trastornos geneticos.

Country Status (12)

Country Link
US (2) US20180028554A1 (es)
EP (2) EP4252845A3 (es)
JP (2) JP2019522972A (es)
KR (3) KR20230175313A (es)
CN (1) CN109757108A (es)
AU (2) AU2017291960B2 (es)
BR (1) BR112019000061A2 (es)
CA (1) CA3029772A1 (es)
IL (2) IL301091A (es)
MX (2) MX2018016253A (es)
RU (2) RU2022107926A (es)
WO (1) WO2018007475A1 (es)

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AU2017291960B2 (en) 2024-03-14
CA3029772A1 (en) 2018-01-11
EP3481955A1 (en) 2019-05-15
RU2018144875A (ru) 2020-08-05
EP4252845A2 (en) 2023-10-04
IL264084B1 (en) 2023-04-01
JP2023024968A (ja) 2023-02-21
CN109757108A (zh) 2019-05-14
RU2769249C2 (ru) 2022-03-29
KR20230175313A (ko) 2023-12-29
US20230330128A1 (en) 2023-10-19
JP2019522972A (ja) 2019-08-22
IL264084B2 (en) 2023-08-01
AU2017291960A1 (en) 2019-01-17
KR20230175312A (ko) 2023-12-29
RU2022107926A (ru) 2022-04-27
BR112019000061A2 (pt) 2019-11-26
AU2023204080A1 (en) 2023-08-31
WO2018007475A1 (en) 2018-01-11
EP4252845A3 (en) 2024-05-22
KR20190027373A (ko) 2019-03-14
US20180028554A1 (en) 2018-02-01
RU2018144875A3 (es) 2020-11-24
MX2018016253A (es) 2019-09-09
IL301091A (en) 2023-05-01
IL264084A (en) 2019-01-31

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