CA2944606C - Treatment of myotonic dystrophy - Google Patents

Treatment of myotonic dystrophy Download PDF

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Publication number
CA2944606C
CA2944606C CA2944606A CA2944606A CA2944606C CA 2944606 C CA2944606 C CA 2944606C CA 2944606 A CA2944606 A CA 2944606A CA 2944606 A CA2944606 A CA 2944606A CA 2944606 C CA2944606 C CA 2944606C
Authority
CA
Canada
Prior art keywords
mbnl
polypeptide
modified
use according
mbnl1
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2944606A
Other languages
English (en)
French (fr)
Other versions
CA2944606A1 (en
Inventor
Denis Furling
Nicolas Sergeant
Marie-Laure CAILLET-BOUDIN
Ludovic ARANDEL
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Centre National de la Recherche Scientifique
Université Pierre et Marie Curie
Institut National de la Santé et de la Recherche Médicale
Institute of Myology
Original Assignee
Centre National de la Recherche Scientifique
Université Pierre et Marie Curie
Institut National de la Santé et de la Recherche Médicale
Institute of Myology
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 National de la Recherche Scientifique, Université Pierre et Marie Curie, Institut National de la Santé et de la Recherche Médicale, Institute of Myology filed Critical Centre National de la Recherche Scientifique
Publication of CA2944606A1 publication Critical patent/CA2944606A1/en
Application granted granted Critical
Publication of CA2944606C publication Critical patent/CA2944606C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CA2944606A 2014-04-14 2015-04-14 Treatment of myotonic dystrophy Active CA2944606C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EPPCT/EP2014/057553 2014-04-14
PCT/EP2014/057553 WO2015158365A1 (en) 2014-04-14 2014-04-14 Treatment of myotonic dystrophy
PCT/EP2015/058111 WO2015158740A1 (en) 2014-04-14 2015-04-14 Treatment of myotonic dystrophy

Publications (2)

Publication Number Publication Date
CA2944606A1 CA2944606A1 (en) 2015-10-22
CA2944606C true CA2944606C (en) 2023-06-27

Family

ID=50588655

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2944606A Active CA2944606C (en) 2014-04-14 2015-04-14 Treatment of myotonic dystrophy

Country Status (6)

Country Link
US (1) US10799556B2 (cg-RX-API-DMAC7.html)
EP (1) EP3145529B1 (cg-RX-API-DMAC7.html)
JP (3) JP6953131B2 (cg-RX-API-DMAC7.html)
CA (1) CA2944606C (cg-RX-API-DMAC7.html)
ES (1) ES2970841T3 (cg-RX-API-DMAC7.html)
WO (2) WO2015158365A1 (cg-RX-API-DMAC7.html)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015158365A1 (en) * 2014-04-14 2015-10-22 Association Institut De Myologie Treatment of myotonic dystrophy
AU2020347503B2 (en) * 2019-09-12 2026-07-16 Association Institut De Myologie Nucleic acid sequence for regulation of transgene expression
WO2024249893A1 (en) * 2023-05-31 2024-12-05 The Trustees Of Columbia University In The City Of New York Generation of mature neurons differentiated from pluripotent stem cells

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
CA2777486A1 (en) * 2008-10-15 2010-04-22 4S3 Bioscience Inc. Methods and compositions for treatment of myotonic dystrophy
WO2015158365A1 (en) 2014-04-14 2015-10-22 Association Institut De Myologie Treatment of myotonic dystrophy

Also Published As

Publication number Publication date
WO2015158740A1 (en) 2015-10-22
US20170035841A1 (en) 2017-02-09
CA2944606A1 (en) 2015-10-22
WO2015158365A1 (en) 2015-10-22
JP6953131B2 (ja) 2021-10-27
ES2970841T3 (es) 2024-05-30
JP2023099082A (ja) 2023-07-11
JP7739017B2 (ja) 2025-09-16
EP3145529B1 (en) 2023-11-15
EP3145529A1 (en) 2017-03-29
JP2021101713A (ja) 2021-07-15
US10799556B2 (en) 2020-10-13
JP2017511145A (ja) 2017-04-20
JP7752148B2 (ja) 2025-10-09

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