PT3058071T - Inibidor de dinamina 2 para o tratamento de miopatias centronucleares - Google Patents

Inibidor de dinamina 2 para o tratamento de miopatias centronucleares

Info

Publication number
PT3058071T
PT3058071T PT14786893T PT14786893T PT3058071T PT 3058071 T PT3058071 T PT 3058071T PT 14786893 T PT14786893 T PT 14786893T PT 14786893 T PT14786893 T PT 14786893T PT 3058071 T PT3058071 T PT 3058071T
Authority
PT
Portugal
Prior art keywords
dynamin
inhibitor
treatment
centronuclear myopathies
centronuclear
Prior art date
Application number
PT14786893T
Other languages
English (en)
Inventor
Laporte Jocelyn
Cowling Belinda
Tasfaout Hichem
Original Assignee
Centre Nat Rech Scient
Univ Strasbourg
Inst Nat Sante Rech Med
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 Centre Nat Rech Scient, Univ Strasbourg, Inst Nat Sante Rech Med filed Critical Centre Nat Rech Scient
Publication of PT3058071T publication Critical patent/PT3058071T/pt

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    • 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
    • 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
    • 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)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • G01N33/5061Muscle cells
    • 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 nucleic acids [NA]
    • 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • Toxicology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
PT14786893T 2013-10-18 2014-10-20 Inibidor de dinamina 2 para o tratamento de miopatias centronucleares PT3058071T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP13306440.2A EP2862928A1 (en) 2013-10-18 2013-10-18 Dynamin 2 inhibitor for the treatment of centronuclear myopathies

Publications (1)

Publication Number Publication Date
PT3058071T true PT3058071T (pt) 2018-10-25

Family

ID=49510094

Family Applications (1)

Application Number Title Priority Date Filing Date
PT14786893T PT3058071T (pt) 2013-10-18 2014-10-20 Inibidor de dinamina 2 para o tratamento de miopatias centronucleares

Country Status (16)

Country Link
US (2) US10647986B2 (pt)
EP (2) EP2862928A1 (pt)
JP (2) JP6641270B2 (pt)
CN (1) CN105940108B (pt)
AU (1) AU2014336070B2 (pt)
BR (1) BR112016008644A2 (pt)
CA (1) CA2927240C (pt)
DK (1) DK3058071T3 (pt)
ES (1) ES2689862T3 (pt)
HR (1) HRP20181450T1 (pt)
LT (1) LT3058071T (pt)
PL (1) PL3058071T3 (pt)
PT (1) PT3058071T (pt)
RS (1) RS57860B1 (pt)
SI (1) SI3058071T1 (pt)
WO (1) WO2015055859A1 (pt)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2862928A1 (en) 2013-10-18 2015-04-22 Université de Strasbourg Dynamin 2 inhibitor for the treatment of centronuclear myopathies
EP3085785A1 (en) * 2015-04-22 2016-10-26 Université de Strasbourg Dynamin 2 inhibitor for the treatment of duchenne's muscular dystrophy
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
EP3610016A1 (en) 2017-04-10 2020-02-19 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
CA3079783A1 (en) * 2017-11-11 2019-05-16 Universite De Strasbourg Compositions and method for the treatment of x-linked centronuclear myopathy
US20200392510A1 (en) * 2018-01-15 2020-12-17 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US20210322562A1 (en) * 2018-08-02 2021-10-21 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating centronuclear myopathy
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
EP3941505A1 (en) * 2019-03-20 2022-01-26 Centre national de la recherche scientifique Amphiphysin / bin1 for the treatment of autosomal dominant centronuclear myopathy
CN115605205A (zh) 2020-03-20 2023-01-13 Abivax公司(Fr) 用于治疗或预防冠状病毒科感染的化合物以及用于评估冠状病毒科感染的发生的方法和用途
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
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 (8)

* 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
GB9811692D0 (en) * 1998-06-01 1998-07-29 Medical Res Council Improvements in or relating to uptake of substances by cells
US8491914B2 (en) * 2004-02-13 2013-07-23 Ibc Pharmaceuticals, Inc. Dock-and-lock (DNL) complexes for delivery of interference RNA
EP2670425A4 (en) 2011-01-31 2015-07-01 Oklahoma Med Res Found UBIQUITIN-INTERACTIVE MOTIVE PEPTIDES AS CANCER THERAPEUTICS
WO2013006555A1 (en) 2011-07-01 2013-01-10 Waltrip Mark A Bottle holder and related methods
JP2014520813A (ja) * 2011-07-01 2014-08-25 ボード・オブ・リージエンツ,ザ・ユニバーシテイ・オブ・テキサス・システム 骨格筋ミオパチーを処置するための組成物および方法
EP2862928A1 (en) 2013-10-18 2015-04-22 Université de Strasbourg Dynamin 2 inhibitor for the treatment of centronuclear myopathies
US10947540B2 (en) * 2016-11-29 2021-03-16 Association Institut De Myologie Allele-specific silencing therapy for Dynamin 2-related diseases

Also Published As

Publication number Publication date
HRP20181450T1 (hr) 2018-11-16
DK3058071T3 (en) 2018-10-22
EP3058071B1 (en) 2018-08-15
WO2015055859A1 (en) 2015-04-23
RS57860B1 (sr) 2018-12-31
AU2014336070A1 (en) 2016-05-19
CA2927240A1 (en) 2015-04-23
CA2927240C (en) 2022-12-13
PL3058071T3 (pl) 2018-12-31
SI3058071T1 (sl) 2018-11-30
US10647986B2 (en) 2020-05-12
EP2862928A1 (en) 2015-04-22
CN105940108A (zh) 2016-09-14
US20200308589A1 (en) 2020-10-01
BR112016008644A2 (pt) 2017-10-03
LT3058071T (lt) 2018-10-25
JP6920408B2 (ja) 2021-08-18
ES2689862T3 (es) 2018-11-16
US11499155B2 (en) 2022-11-15
JP2020055867A (ja) 2020-04-09
EP3058071A1 (en) 2016-08-24
JP6641270B2 (ja) 2020-02-05
JP2016540732A (ja) 2016-12-28
AU2014336070B2 (en) 2020-11-19
US20160264976A1 (en) 2016-09-15
CN105940108B (zh) 2019-06-28

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