DK3302522T3 - Midler og fremgangsmåde til behandling af facioscapulohumeral muskeldystrofi (fshd) - Google Patents

Midler og fremgangsmåde til behandling af facioscapulohumeral muskeldystrofi (fshd) Download PDF

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Publication number
DK3302522T3
DK3302522T3 DK16744567.5T DK16744567T DK3302522T3 DK 3302522 T3 DK3302522 T3 DK 3302522T3 DK 16744567 T DK16744567 T DK 16744567T DK 3302522 T3 DK3302522 T3 DK 3302522T3
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DK
Denmark
Prior art keywords
fshd
agent
treatment
muscular dystrophy
facioscapulohumeral muscular
Prior art date
Application number
DK16744567.5T
Other languages
English (en)
Inventor
Der Maarel Silvere Maria Van
Richard Joannes Leonardus Franciscus Lemmers
Judit Balog
Stephen Justice Tapscott
Alrabi Tawil
Original Assignee
Academisch Ziekenhuis Leiden
Univ Rochester
Fred Hutchinson Cancer Center
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Application filed by Academisch Ziekenhuis Leiden, Univ Rochester, Fred Hutchinson Cancer Center filed Critical Academisch Ziekenhuis Leiden
Application granted granted Critical
Publication of DK3302522T3 publication Critical patent/DK3302522T3/da

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
DK16744567.5T 2015-06-02 2016-06-02 Midler og fremgangsmåde til behandling af facioscapulohumeral muskeldystrofi (fshd) DK3302522T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP15170244 2015-06-02
PCT/NL2016/050396 WO2016195493A1 (en) 2015-06-02 2016-06-02 Means and methods for treating facioscapulohumeral muscular dystrophy (fshd).

Publications (1)

Publication Number Publication Date
DK3302522T3 true DK3302522T3 (da) 2022-09-26

Family

ID=53298200

Family Applications (1)

Application Number Title Priority Date Filing Date
DK16744567.5T DK3302522T3 (da) 2015-06-02 2016-06-02 Midler og fremgangsmåde til behandling af facioscapulohumeral muskeldystrofi (fshd)

Country Status (5)

Country Link
US (1) US20180147256A1 (da)
EP (1) EP3302522B1 (da)
DK (1) DK3302522T3 (da)
ES (1) ES2926785T3 (da)
WO (1) WO2016195493A1 (da)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3042700A1 (en) 2016-11-07 2018-05-11 University Of Massachusetts Therapeutic targets for facioscapulohumeral muscular dystrophy
WO2018128587A1 (en) * 2017-01-06 2018-07-12 Agency For Science, Technology And Research Mutant smchd1 for therapy
KR20220007092A (ko) * 2019-05-09 2022-01-18 더 칠드런스 호스피탈 오브 필라델피아 대뇌백질이영양증의 치료용 조성물 및 방법과 그것의 치료에 효과적인 작용제를 확인하기 위한 전체 동물 및 세포 모델
US20230220473A1 (en) * 2020-05-29 2023-07-13 Children's National Medical Center Genetic diagnostic tool for facioscapulohumeral muscular dystrophy (fshd)
IL301187A (en) 2020-09-11 2023-05-01 Arrowhead Pharmaceuticals Inc RNAi agents for suppressing DUX4 expression, preparations containing them and uses thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013120038A2 (en) * 2012-02-10 2013-08-15 Regents Of The University Of Minnesota Morpholino targeting dux4 for treating fshd
WO2013192185A1 (en) * 2012-06-18 2013-12-27 Fred Hutchinson Cancer Research Center Methods compositions related to the smchd1 gene
WO2014071340A1 (en) * 2012-11-05 2014-05-08 University Of Washington Through Its Center For Commercialization Methods and assays for facioscapulohumeral muscular dystrophy

Also Published As

Publication number Publication date
US20180147256A1 (en) 2018-05-31
WO2016195493A1 (en) 2016-12-08
EP3302522B1 (en) 2022-08-31
EP3302522A1 (en) 2018-04-11
ES2926785T3 (es) 2022-10-28

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