DK3286317T3 - Dynamin 2-inhibitor til behandlingen af duchennes muskeldystrofi - Google Patents

Dynamin 2-inhibitor til behandlingen af duchennes muskeldystrofi Download PDF

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Publication number
DK3286317T3
DK3286317T3 DK16722076.3T DK16722076T DK3286317T3 DK 3286317 T3 DK3286317 T3 DK 3286317T3 DK 16722076 T DK16722076 T DK 16722076T DK 3286317 T3 DK3286317 T3 DK 3286317T3
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DK
Denmark
Prior art keywords
dyrophy
duchenn
dynamin
muscle
inhibitor
Prior art date
Application number
DK16722076.3T
Other languages
English (en)
Inventor
Jocelyn Laporte
Belinda Cowling
Original Assignee
Univ Strasbourg
Inst Nat Sante Rech Med
Centre Nat Rech Scient
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 Univ Strasbourg, Inst Nat Sante Rech Med, Centre Nat Rech Scient filed Critical Univ Strasbourg
Application granted granted Critical
Publication of DK3286317T3 publication Critical patent/DK3286317T3/da

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05—Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
    • C12Y306/05005—Dynamin GTPase (3.6.5.5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
DK16722076.3T 2015-04-22 2016-04-22 Dynamin 2-inhibitor til behandlingen af duchennes muskeldystrofi DK3286317T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP15305615.5A EP3085785A1 (en) 2015-04-22 2015-04-22 Dynamin 2 inhibitor for the treatment of duchenne's muscular dystrophy
PCT/EP2016/059090 WO2016170162A1 (en) 2015-04-22 2016-04-22 Dynamin 2 inhibitor for the treatment of duchenne's muscular dystrophy

Publications (1)

Publication Number Publication Date
DK3286317T3 true DK3286317T3 (da) 2021-05-17

Family

ID=53005533

Family Applications (1)

Application Number Title Priority Date Filing Date
DK16722076.3T DK3286317T3 (da) 2015-04-22 2016-04-22 Dynamin 2-inhibitor til behandlingen af duchennes muskeldystrofi

Country Status (10)

Country Link
US (2) US10822400B2 (da)
EP (2) EP3085785A1 (da)
JP (1) JP6791877B2 (da)
CN (1) CN107532169B (da)
AU (1) AU2016251973B2 (da)
CA (1) CA2983211A1 (da)
DK (1) DK3286317T3 (da)
ES (1) ES2869978T3 (da)
PT (1) PT3286317T (da)
WO (1) WO2016170162A1 (da)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10947540B2 (en) * 2016-11-29 2021-03-16 Association Institut De Myologie Allele-specific silencing therapy for Dynamin 2-related diseases
WO2018115477A1 (en) * 2016-12-23 2018-06-28 Universite De Strasbourg Dynamin 2 inhibitor for the treatment of myotonic dystrophy
BR112019015081A2 (pt) * 2017-01-23 2020-03-10 Rutgers, The State University Of New Jersey Composições para reduzir a expressão da sarcolipina e prevenir e tratar a distrofia muscular e a cardiomiopatia e os métodos de utilização
CA3088522A1 (en) * 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
EP4215614A1 (en) * 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
EP4311546A1 (en) 2022-07-26 2024-01-31 Dynacure Combination therapy for myopathies
WO2025085810A2 (en) * 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
GB0602768D0 (en) * 2006-02-10 2006-03-22 Vastox Plc Treatment of muscular dystrophy
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
EP3133160B1 (en) * 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
CN103826620A (zh) * 2011-05-27 2014-05-28 Md制药公司 新的治疗方法
WO2013006558A2 (en) * 2011-07-01 2013-01-10 Board Of Regents, The University Of Texas System Compositions and methods for treating skeletal myopathy
EP2862928A1 (en) * 2013-10-18 2015-04-22 Université de Strasbourg Dynamin 2 inhibitor for the treatment of centronuclear myopathies

Also Published As

Publication number Publication date
US10822400B2 (en) 2020-11-03
JP2018518456A (ja) 2018-07-12
EP3286317B1 (en) 2021-02-24
PT3286317T (pt) 2021-05-18
US20180142008A1 (en) 2018-05-24
CA2983211A1 (en) 2016-10-27
EP3085785A1 (en) 2016-10-26
JP6791877B2 (ja) 2020-11-25
WO2016170162A1 (en) 2016-10-27
US20210130447A1 (en) 2021-05-06
EP3286317A1 (en) 2018-02-28
AU2016251973B2 (en) 2022-05-26
CN107532169A (zh) 2018-01-02
CN107532169B (zh) 2021-09-03
AU2016251973A1 (en) 2017-11-09
ES2869978T3 (es) 2021-10-26

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