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.)
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Publication date
Application filed by Univ Strasbourg, Inst Nat Sante Rech Med, Centre Nat Rech Scient filed Critical Univ Strasbourg
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Publication of DK3286317T3 publication Critical patent/DK3286317T3/da

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • C12N15/1137Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/05Hydrolases acting on acid anhydrides (3.6) acting on GTP; involved in cellular and subcellular movement (3.6.5)
    • C12Y306/05005Dynamin GTPase (3.6.5.5)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.

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)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Investigating Or Analysing Biological Materials (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 (6)

* 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
CN110475573A (zh) * 2017-01-23 2019-11-19 新泽西鲁特格斯州立大学 用于减少肌脂蛋白表达以及预防和治疗肌营养不良症和心肌病的组合物及其使用方法
CN111902537A (zh) 2018-01-15 2020-11-06 Ionis制药公司 Dnm2表达的调节剂
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

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
CN102203253B (zh) * 2008-10-24 2016-04-06 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
EP2714020A1 (en) * 2011-05-27 2014-04-09 MD Pharma AB Novel treatments
US20140221464A1 (en) 2011-07-01 2014-08-07 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
EP3286317A1 (en) 2018-02-28
ES2869978T3 (es) 2021-10-26
US10822400B2 (en) 2020-11-03
JP2018518456A (ja) 2018-07-12
AU2016251973B2 (en) 2022-05-26
CN107532169A (zh) 2018-01-02
JP6791877B2 (ja) 2020-11-25
CA2983211A1 (en) 2016-10-27
WO2016170162A1 (en) 2016-10-27
CN107532169B (zh) 2021-09-03
EP3286317B1 (en) 2021-02-24
PT3286317T (pt) 2021-05-18
EP3085785A1 (en) 2016-10-26
US20180142008A1 (en) 2018-05-24
AU2016251973A1 (en) 2017-11-09
US20210130447A1 (en) 2021-05-06

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