BR112016008644A2 - Inibidor de dinamina 2 para uso no tratamento de uma miopatia centronuclear; método para identificar ou triar moléculas úteis no tratamento de uma miopatia centronuclear, de preferência, xlcnm; e composição farmacêutica - Google Patents

Inibidor de dinamina 2 para uso no tratamento de uma miopatia centronuclear; método para identificar ou triar moléculas úteis no tratamento de uma miopatia centronuclear, de preferência, xlcnm; e composição farmacêutica

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Publication number
BR112016008644A2
BR112016008644A2 BR112016008644A BR112016008644A BR112016008644A2 BR 112016008644 A2 BR112016008644 A2 BR 112016008644A2 BR 112016008644 A BR112016008644 A BR 112016008644A BR 112016008644 A BR112016008644 A BR 112016008644A BR 112016008644 A2 BR112016008644 A2 BR 112016008644A2
Authority
BR
Brazil
Prior art keywords
treatment
centronuclear myopathy
dynamine
inhibitors
identifying
Prior art date
Application number
BR112016008644A
Other languages
English (en)
Inventor
Laporte Jocelyn
Cowling Belinda
Tasfaout Hichem
Original Assignee
Centre Nat Rech Scient
Inst Nat Sante Rech Med
Univ Strasbourg
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, Inst Nat Sante Rech Med, Univ Strasbourg filed Critical Centre Nat Rech Scient
Publication of BR112016008644A2 publication Critical patent/BR112016008644A2/pt

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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical 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
    • G01N33/5044—Chemical 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
    • G01N33/5061—Muscle cells
    • 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 N.A.
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)

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  • 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Analytical Chemistry (AREA)
  • Toxicology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)

Abstract

INIBIDOR DE DINAMINA 2 PARA O TRATAMENTO DE MIOPATIAS CENTRONUCLEARES. A presente revelação se refere a um inibidor de Dinamina 2 para uso no tratamento de miopatias centronucleares. A presente revelação se refere a composições farmacêuticas que contêm inibidor de Dinamina 2 e a seu uso para o tratamento de miopatias centronucleares. Também trata de um método para identificar ou triar moléculas úteis no tratamento de uma miopatia centronuclear.
BR112016008644A 2013-10-18 2014-10-20 Inibidor de dinamina 2 para uso no tratamento de uma miopatia centronuclear; método para identificar ou triar moléculas úteis no tratamento de uma miopatia centronuclear, de preferência, xlcnm; e composição farmacêutica BR112016008644A2 (pt)

Applications Claiming Priority (2)

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
PCT/EP2014/072466 WO2015055859A1 (en) 2013-10-18 2014-10-20 Dynamin 2 inhibitor for the treatment of centronuclear myopathies

Publications (1)

Publication Number Publication Date
BR112016008644A2 true BR112016008644A2 (pt) 2017-10-03

Family

ID=49510094

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112016008644A BR112016008644A2 (pt) 2013-10-18 2014-10-20 Inibidor de dinamina 2 para uso no tratamento de uma miopatia centronuclear; método para identificar ou triar moléculas úteis no tratamento de uma miopatia centronuclear, de preferência, xlcnm; e composição farmacêutica

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 (14)

* 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
JP7301047B2 (ja) 2017-11-11 2023-06-30 ユニヴェルシテ・ドゥ・ストラスブール Ｘ連鎖性中心核ミオパチーを処置するための組成物及び方法
US10865414B2 (en) * 2018-01-15 2020-12-15 Ionis Pharmaceuticals, Inc. Modulators of DNM2 expression
EP3830130A4 (en) * 2018-08-02 2022-05-18 Dyne Therapeutics, Inc. MUSCLE TARGET COMPLEXES AND THEIR USES IN THE TREATMENT OF CENTRONUCLEAR MYOPATHY
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP2022525944A (ja) * 2019-03-20 2022-05-20 サントル ナショナル ドゥ ラ ルシェルシュ シアンティフィック 常染色体優性中心核ミオパシーを処置するためのアンフィファイジン／ｂｉｎ１
IL296522A (en) 2020-03-20 2022-11-01 Abivax Compounds for treating or preventing coronaviridae infection and methods and uses for assessing the occurrence of coronaviridae infection
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
US9993055B2 (en) 2011-07-01 2018-06-12 Mark A. Waltrip Bottle holder and related methods
EP2726109A4 (en) * 2011-07-01 2014-11-26 Univ Texas 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
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
US10647986B2 (en) 2020-05-12
CN105940108A (zh) 2016-09-14
JP2020055867A (ja) 2020-04-09
US20160264976A1 (en) 2016-09-15
JP6920408B2 (ja) 2021-08-18
JP2016540732A (ja) 2016-12-28
US20200308589A1 (en) 2020-10-01
AU2014336070A1 (en) 2016-05-19
HRP20181450T1 (hr) 2018-11-16
EP3058071B1 (en) 2018-08-15
LT3058071T (lt) 2018-10-25
JP6641270B2 (ja) 2020-02-05
EP3058071A1 (en) 2016-08-24
AU2014336070B2 (en) 2020-11-19
ES2689862T3 (es) 2018-11-16
SI3058071T1 (sl) 2018-11-30
PL3058071T3 (pl) 2018-12-31
DK3058071T3 (en) 2018-10-22
CN105940108B (zh) 2019-06-28
RS57860B1 (sr) 2018-12-31
PT3058071T (pt) 2018-10-25
EP2862928A1 (en) 2015-04-22
CA2927240A1 (en) 2015-04-23
WO2015055859A1 (en) 2015-04-23
US11499155B2 (en) 2022-11-15
CA2927240C (en) 2022-12-13

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