CO2018003960A2 - Virus adeno-asociado recombinante que comprende un ácido nucleico galgt2 - Google Patents

Virus adeno-asociado recombinante que comprende un ácido nucleico galgt2

Info

Publication number
CO2018003960A2
CO2018003960A2 CONC2018/0003960A CO2018003960A CO2018003960A2 CO 2018003960 A2 CO2018003960 A2 CO 2018003960A2 CO 2018003960 A CO2018003960 A CO 2018003960A CO 2018003960 A2 CO2018003960 A2 CO 2018003960A2
Authority
CO
Colombia
Prior art keywords
galgt2
associated virus
recombinant adeno
nucleic acid
raav
Prior art date
Application number
CONC2018/0003960A
Other languages
English (en)
Inventor
Paul Taylor Martin
Original Assignee
Res Inst Nationwide Childrens Hospital
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 Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of CO2018003960A2 publication Critical patent/CO2018003960A2/es

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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86—Viral vectors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1048—Glycosyltransferases (2.4)
    • C12N9/1051—Hexosyltransferases (2.4.1)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
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    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01165—N-Acetylneuraminylgalactosylglucosylceramide beta-1,4-N-acetylgalactosaminyltransferase (2.4.1.165)
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Virology (AREA)
  • Analytical Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente divulgación se refiere a la administración de virus adeno-asociado recombinante (rAAV) de un polinucleótido GALGT2. La divulgación proporciona rAAV y métodos de uso de rAAV para la terapia génica GALGT2 de trastornos neuromusculares. Los trastornos neuromusculares de ejemplo incluyen, pero no se limitan a, distrofias musculares tales como Distrofia muscular de Duchenne, Distrofia muscular congénita 1A y Distrofia muscular de la columna y extremidades 2D.
CONC2018/0003960A 2015-09-17 2018-04-13 Virus adeno-asociado recombinante que comprende un ácido nucleico galgt2 CO2018003960A2 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562220107P 2015-09-17 2015-09-17
US201562221068P 2015-09-20 2015-09-20
US201662301260P 2016-02-29 2016-02-29
PCT/US2016/052051 WO2017049031A1 (en) 2015-09-17 2016-09-16 Methods and materials for galgt2 gene therapy

Publications (1)

Publication Number Publication Date
CO2018003960A2 true CO2018003960A2 (es) 2018-10-10

Family

ID=58289891

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2018/0003960A CO2018003960A2 (es) 2015-09-17 2018-04-13 Virus adeno-asociado recombinante que comprende un ácido nucleico galgt2

Country Status (15)

Country Link
US (2) US10980897B2 (es)
EP (1) EP3350331A4 (es)
JP (2) JP7338970B2 (es)
KR (1) KR20180084747A (es)
CN (1) CN108136049A (es)
AU (2) AU2016323575B2 (es)
BR (1) BR112018005177A2 (es)
CA (1) CA2998636A1 (es)
CO (1) CO2018003960A2 (es)
EA (1) EA201890543A1 (es)
HK (1) HK1257519A1 (es)
IL (1) IL258005A (es)
MX (1) MX2018003234A (es)
SA (1) SA518391113B1 (es)
WO (1) WO2017049031A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
US11534501B2 (en) 2017-10-18 2022-12-27 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
CN111433367A (zh) 2017-10-20 2020-07-17 全国儿童医院研究所 Nt-3基因疗法的方法和材料
EP3807309A1 (en) * 2018-06-18 2021-04-21 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
EP4051324A4 (en) * 2019-10-28 2023-11-29 University Of Florida Research Foundation, Incorporated GENE THERAPY VECTORS
EP4230196A1 (en) 2022-02-21 2023-08-23 Som Innovation Biotech, S.A. Compounds for use in the treatment of dystrophinopathies

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Also Published As

Publication number Publication date
WO2017049031A1 (en) 2017-03-23
CN108136049A (zh) 2018-06-08
SA518391113B1 (ar) 2022-10-25
AU2016323575A1 (en) 2018-04-05
EA201890543A1 (ru) 2018-08-31
JP2022010079A (ja) 2022-01-14
CA2998636A1 (en) 2017-03-23
AU2023200499A1 (en) 2023-06-15
JP2018527365A (ja) 2018-09-20
US20180250423A1 (en) 2018-09-06
MX2018003234A (es) 2018-09-07
KR20180084747A (ko) 2018-07-25
BR112018005177A2 (pt) 2018-10-09
EP3350331A1 (en) 2018-07-25
US10980897B2 (en) 2021-04-20
EP3350331A4 (en) 2019-01-23
US20210220484A1 (en) 2021-07-22
IL258005A (en) 2018-05-31
JP7338970B2 (ja) 2023-09-05
HK1257519A1 (zh) 2019-10-25
AU2016323575B2 (en) 2022-10-27

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