ES2970841T3 - Polipéptido MBNL para su uso en el tratamiento de la distrofia miotónica - Google Patents

Polipéptido MBNL para su uso en el tratamiento de la distrofia miotónica Download PDF

Info

Publication number
ES2970841T3
ES2970841T3 ES15716781T ES15716781T ES2970841T3 ES 2970841 T3 ES2970841 T3 ES 2970841T3 ES 15716781 T ES15716781 T ES 15716781T ES 15716781 T ES15716781 T ES 15716781T ES 2970841 T3 ES2970841 T3 ES 2970841T3
Authority
ES
Spain
Prior art keywords
mbnl
mbnl1
act3
polypeptide
splicing
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
ES15716781T
Other languages
English (en)
Spanish (es)
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 CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Sorbonne Universite
Original Assignee
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Association Institut de Myologie
Sorbonne Universite
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 CNRS, Institut National de la Sante et de la Recherche Medicale INSERM, Association Institut de Myologie, Sorbonne Universite filed Critical Centre National de la Recherche Scientifique CNRS
Application granted granted Critical
Publication of ES2970841T3 publication Critical patent/ES2970841T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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)
ES15716781T 2014-04-14 2015-04-14 Polipéptido MBNL para su uso en el tratamiento de la distrofia miotónica Active ES2970841T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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 (1)

Publication Number Publication Date
ES2970841T3 true ES2970841T3 (es) 2024-05-30

Family

ID=50588655

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15716781T Active ES2970841T3 (es) 2014-04-14 2015-04-14 Polipéptido MBNL para su uso en el tratamiento de la distrofia miotónica

Country Status (6)

Country Link
US (1) US10799556B2 (https=)
EP (1) EP3145529B1 (https=)
JP (3) JP6953131B2 (https=)
CA (1) CA2944606C (https=)
ES (1) ES2970841T3 (https=)
WO (2) WO2015158365A1 (https=)

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
CA3150413A1 (en) * 2019-09-12 2021-03-18 Denis Furling NUCLEIC ACID SEQUENCE FOR THE 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
WO2010044894A1 (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
JP2021101713A (ja) 2021-07-15
WO2015158740A1 (en) 2015-10-22
US10799556B2 (en) 2020-10-13
JP2023099082A (ja) 2023-07-11
JP7752148B2 (ja) 2025-10-09
EP3145529A1 (en) 2017-03-29
EP3145529B1 (en) 2023-11-15
CA2944606A1 (en) 2015-10-22
US20170035841A1 (en) 2017-02-09
WO2015158365A1 (en) 2015-10-22
JP6953131B2 (ja) 2021-10-27
JP7739017B2 (ja) 2025-09-16
CA2944606C (en) 2023-06-27
JP2017511145A (ja) 2017-04-20

Similar Documents

Publication Publication Date Title
JP7416451B2 (ja) ＣＲＩＳＰＲ－Ｃａｓによる標的化された核内ＲＮＡ切断及びポリアデニル化
JP6453307B2 (ja) デュアルａａｖベクターによる大型遺伝子の効果的送達
US20180256752A1 (en) Selective gene therapy expression system
JP7752148B2 (ja) 筋強直性ジストロフィーの処置
CN111356763B (zh) 变体RNAi
JP2020513831A (ja) ＭｅＣＰ２発現カセット
CN113710693B (zh) Dna结合结构域反式激活因子及其用途
US20200248204A1 (en) Methods of treating genetic hearing loss
EP2212348B1 (en) Materials and methods for gene mediated therapy of psychiatric disorders
US12503495B2 (en) Treatment of myotonic dystrophy
RU2854890C1 (ru) Вектор
WO2024178364A1 (en) Gene therapy for treatment of pain
CN120917144A (zh) 用于靶向致病性蛋白聚集体进行降解的治疗性融合蛋白
HK40118673A (zh) 用於治疗polyq疾病的治疗因子