MX2018003234A - Metodos y materiales para la terapia genica galgt2. - Google Patents

Metodos y materiales para la terapia genica galgt2.

Info

Publication number
MX2018003234A
MX2018003234A MX2018003234A MX2018003234A MX2018003234A MX 2018003234 A MX2018003234 A MX 2018003234A MX 2018003234 A MX2018003234 A MX 2018003234A MX 2018003234 A MX2018003234 A MX 2018003234A MX 2018003234 A MX2018003234 A MX 2018003234A
Authority
MX
Mexico
Prior art keywords
methods
gene therapy
galgt2
raav
materials
Prior art date
Application number
MX2018003234A
Other languages
English (en)
Spanish (es)
Inventor
taylor martin Paul
Original Assignee
Res Institute At Nationwide Children´S 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 Institute At Nationwide Children´S Hospital filed Critical Res Institute At Nationwide Children´S Hospital
Publication of MX2018003234A publication Critical patent/MX2018003234A/es

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45Transferases (2)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1048Glycosyltransferases (2.4)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1048Glycosyltransferases (2.4)
    • C12N9/1051Hexosyltransferases (2.4.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/01Hexosyltransferases (2.4.1)
    • C12Y204/01165N-Acetylneuraminylgalactosylglucosylceramide beta-1,4-N-acetylgalactosaminyltransferase (2.4.1.165)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • 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)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (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)
MX2018003234A 2015-09-17 2016-09-16 Metodos y materiales para la terapia genica galgt2. MX2018003234A (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
MX2018003234A true MX2018003234A (es) 2018-09-07

Family

ID=58289891

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018003234A MX2018003234A (es) 2015-09-17 2016-09-16 Metodos y materiales para la terapia genica galgt2.
MX2023013509A MX2023013509A (es) 2015-09-17 2018-03-14 Composiciones farmaceuticas que comprenden raav para la terapia genica galgt2 para trastornos neuromusculares y uso de las mismas.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023013509A MX2023013509A (es) 2015-09-17 2018-03-14 Composiciones farmaceuticas que comprenden raav para la terapia genica galgt2 para trastornos neuromusculares y uso de las mismas.

Country Status (14)

Country Link
US (3) US10980897B2 (cg-RX-API-DMAC7.html)
EP (1) EP3350331A4 (cg-RX-API-DMAC7.html)
JP (3) JP7338970B2 (cg-RX-API-DMAC7.html)
KR (1) KR102799163B1 (cg-RX-API-DMAC7.html)
CN (1) CN108136049A (cg-RX-API-DMAC7.html)
AU (3) AU2016323575B2 (cg-RX-API-DMAC7.html)
CA (1) CA2998636A1 (cg-RX-API-DMAC7.html)
CO (1) CO2018003960A2 (cg-RX-API-DMAC7.html)
EA (1) EA201890543A1 (cg-RX-API-DMAC7.html)
HK (1) HK1257519A1 (cg-RX-API-DMAC7.html)
IL (2) IL314884A (cg-RX-API-DMAC7.html)
MX (2) MX2018003234A (cg-RX-API-DMAC7.html)
SA (1) SA518391113B1 (cg-RX-API-DMAC7.html)
WO (1) WO2017049031A1 (cg-RX-API-DMAC7.html)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3350331A4 (en) 2015-09-17 2019-01-23 Research Institute at Nationwide Children's Hospital METHOD AND MATERIALS FOR GALGT2 GENE THERAPY
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
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
UY37679A (es) * 2017-04-14 2018-11-30 Regenxbio Inc Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas
MA50836A (fr) 2017-10-18 2020-08-26 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
MY207563A (en) 2017-10-20 2025-03-04 Res Inst Nationwide Childrens Hospital Methods and materials for nt-3 gene therapy
PT3807413T (pt) 2018-06-18 2025-08-19 Res Inst Nationwide Childrens Hospital Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular
CA3104471A1 (en) * 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
CN114828858A (zh) * 2019-10-25 2022-07-29 奥登茨治疗公司 用于治疗糖原贮积症的组合物和方法
EP4051324A4 (en) * 2019-10-28 2023-11-29 University Of Florida Research Foundation, Incorporated Gene therapy vectors
MX2023001411A (es) * 2020-08-07 2023-05-15 Janssen Biotech Inc Formulaciones para particulas virales altamente purificadas.
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
US20250025578A1 (en) 2025-01-23
CN108136049A (zh) 2018-06-08
KR20180084747A (ko) 2018-07-25
KR102799163B1 (ko) 2025-04-21
CO2018003960A2 (es) 2018-10-10
JP7338970B2 (ja) 2023-09-05
CA2998636A1 (en) 2017-03-23
MX2023013509A (es) 2024-01-08
EP3350331A4 (en) 2019-01-23
SA518391113B1 (ar) 2022-10-25
AU2023200499B2 (en) 2025-05-22
HK1257519A1 (zh) 2019-10-25
IL258005A (en) 2018-05-31
AU2025213610A1 (en) 2025-08-28
US12121595B2 (en) 2024-10-22
NZ740707A (en) 2025-06-27
US20180250423A1 (en) 2018-09-06
NZ780253A (en) 2025-06-27
WO2017049031A1 (en) 2017-03-23
JP2018527365A (ja) 2018-09-20
JP2024109934A (ja) 2024-08-14
IL314884A (en) 2024-10-01
EP3350331A1 (en) 2018-07-25
AU2023200499A1 (en) 2023-06-15
AU2016323575A1 (en) 2018-04-05
EA201890543A1 (ru) 2018-08-31
IL258005B2 (en) 2025-01-01
US20210220484A1 (en) 2021-07-22
IL258005B1 (en) 2024-09-01
JP2022010079A (ja) 2022-01-14
AU2016323575B2 (en) 2022-10-27
BR112018005177A2 (pt) 2018-10-09
US10980897B2 (en) 2021-04-20

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