MX2018016052A - Oligomeros de omision de exon para distrofia muscular. - Google Patents

Oligomeros de omision de exon para distrofia muscular.

Info

Publication number
MX2018016052A
MX2018016052A MX2018016052A MX2018016052A MX2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A MX 2018016052 A MX2018016052 A MX 2018016052A
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MX
Mexico
Prior art keywords
muscular dystrophy
exon skipping
oligomers
skipping oligomers
exon
Prior art date
Application number
MX2018016052A
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English (en)
Inventor
Elizabeth Frank Diane
K Bestwick Richard
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Sarepta Therapeutics Inc
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Publication of MX2018016052A publication Critical patent/MX2018016052A/es

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65583Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system each of the hetero rings containing nitrogen as ring hetero atom
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C07F9/65616Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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

Abstract

La presente invencion se refiere a oligomeros antisentido complementarios a un sitio objetivo seleccionado en el gen de distrofina humana para inducir la omision del exon 45.
MX2018016052A 2016-06-30 2017-06-29 Oligomeros de omision de exon para distrofia muscular. MX2018016052A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662357072P 2016-06-30 2016-06-30
US201662356923P 2016-06-30 2016-06-30
PCT/US2017/040017 WO2018005805A1 (en) 2016-06-30 2017-06-29 Exon skipping oligomers for muscular dystrophy

Publications (1)

Publication Number Publication Date
MX2018016052A true MX2018016052A (es) 2019-05-02

Family

ID=59315756

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018016052A MX2018016052A (es) 2016-06-30 2017-06-29 Oligomeros de omision de exon para distrofia muscular.

Country Status (15)

Country Link
US (2) US20190262375A1 (es)
EP (1) EP3478697A1 (es)
JP (1) JP2019525742A (es)
KR (1) KR20190024977A (es)
CN (1) CN109311919A (es)
AU (1) AU2017290231A1 (es)
BR (1) BR112019000006A2 (es)
CA (1) CA3025575A1 (es)
CO (1) CO2018014029A2 (es)
IL (1) IL263892A (es)
MA (1) MA45618A (es)
MX (1) MX2018016052A (es)
SG (1) SG11201810143PA (es)
TW (1) TW201811807A (es)
WO (1) WO2018005805A1 (es)

Families Citing this family (9)

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TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CN109152792B (zh) * 2016-06-30 2021-09-14 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
WO2019209764A2 (en) * 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
US10765760B2 (en) * 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3810150A4 (en) * 2018-06-14 2023-01-25 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATE FOR MUSCLE DYSTROPHY
EP3946376A1 (en) * 2019-03-28 2022-02-09 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy with casimersen
WO2021025899A1 (en) 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
TW202307208A (zh) * 2021-06-23 2023-02-16 日商日本新藥股份有限公司 反義寡聚物之組合

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EP3478697A1 (en) 2019-05-08
CN109311919A (zh) 2019-02-05
AU2017290231A1 (en) 2019-02-07
TW201811807A (zh) 2018-04-01
BR112019000006A2 (pt) 2019-04-16
SG11201810143PA (en) 2019-01-30
WO2018005805A1 (en) 2018-01-04
US20190262375A1 (en) 2019-08-29
US20210187003A1 (en) 2021-06-24
CA3025575A1 (en) 2018-01-04
JP2019525742A (ja) 2019-09-12
CO2018014029A2 (es) 2019-03-18
MA45618A (fr) 2019-05-08
IL263892A (en) 2019-01-31
KR20190024977A (ko) 2019-03-08

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