PT3286317T - Inibidor de dinamina 2 para o tratamento de distrofia muscular de duchenne - Google Patents

Inibidor de dinamina 2 para o tratamento de distrofia muscular de duchenne

Info

Publication number
PT3286317T
PT3286317T PT167220763T PT16722076T PT3286317T PT 3286317 T PT3286317 T PT 3286317T PT 167220763 T PT167220763 T PT 167220763T PT 16722076 T PT16722076 T PT 16722076T PT 3286317 T PT3286317 T PT 3286317T
Authority
PT
Portugal
Prior art keywords
dynamin
duchenne
inhibitor
treatment
muscular dystrophy
Prior art date
Application number
PT167220763T
Other languages
English (en)
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
Publication of PT3286317T publication Critical patent/PT3286317T/pt

Links

Classifications

    • 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)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (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)
PT167220763T 2015-04-22 2016-04-22 Inibidor de dinamina 2 para o tratamento de distrofia muscular de duchenne PT3286317T (pt)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
PT3286317T true PT3286317T (pt) 2021-05-18

Family

ID=53005533

Family Applications (1)

Application Number Title Priority Date Filing Date
PT167220763T PT3286317T (pt) 2015-04-22 2016-04-22 Inibidor de dinamina 2 para o tratamento de distrofia muscular de duchenne

Country Status (10)

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

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
AU2018210990A1 (en) 2017-01-23 2019-08-29 Rutgers, The State University Of New Jersey Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use
EP3740575A1 (en) * 2018-01-15 2020-11-25 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

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
CA3066050A1 (en) * 2008-10-24 2010-04-29 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for dmd
CN103826620A (zh) * 2011-05-27 2014-05-28 Md制药公司 新的治疗方法
CN103764173A (zh) 2011-07-01 2014-04-30 得克萨斯系统大学董事会 用于治疗骨骼肌病的组合物和方法
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
US20180142008A1 (en) 2018-05-24
JP2018518456A (ja) 2018-07-12
EP3286317A1 (en) 2018-02-28
US20210130447A1 (en) 2021-05-06
EP3085785A1 (en) 2016-10-26
AU2016251973B2 (en) 2022-05-26
AU2016251973A1 (en) 2017-11-09
WO2016170162A1 (en) 2016-10-27
ES2869978T3 (es) 2021-10-26
US10822400B2 (en) 2020-11-03
JP6791877B2 (ja) 2020-11-25
DK3286317T3 (da) 2021-05-17
CN107532169A (zh) 2018-01-02
CN107532169B (zh) 2021-09-03
EP3286317B1 (en) 2021-02-24
CA2983211A1 (en) 2016-10-27

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