AR111292A1 - Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular - Google Patents

Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular

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Publication number
AR111292A1
AR111292A1 ARP180100629A ARP180100629A AR111292A1 AR 111292 A1 AR111292 A1 AR 111292A1 AR P180100629 A ARP180100629 A AR P180100629A AR P180100629 A ARP180100629 A AR P180100629A AR 111292 A1 AR111292 A1 AR 111292A1
Authority
AR
Argentina
Prior art keywords
vectors
microdistrophin
muscular dystrophy
virus
adeo
Prior art date
Application number
ARP180100629A
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English (en)
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Res Institute At Nationwide Children’S Hospital
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Application filed by Res Institute At Nationwide Children’S Hospital filed Critical Res Institute At Nationwide Children’S Hospital
Publication of AR111292A1 publication Critical patent/AR111292A1/es

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    • 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
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0029Parenteral nutrition; Parenteral nutrition compositions as drug carriers
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nutrition Science (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente proporciona vectores de tratamiento génico, tales como vectores del virus adenoasociado (AAV), que expresan un gen de microdistrofina humana miniaturizado y un método para usar estos vectores en la expresión de microdistrofina en músculos esqueléticos, que incluyen el músculo de diafragma y cardiaco, y para proteger las fibras musculares de lesiones, aumentar la fuerza muscular y reducir y/o prevenir la fibrosis en sujetos que tienen distrofia muscular.
ARP180100629A 2017-03-17 2018-03-19 Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular AR111292A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762473148P 2017-03-17 2017-03-17

Publications (1)

Publication Number Publication Date
AR111292A1 true AR111292A1 (es) 2019-06-26

Family

ID=63523321

Family Applications (1)

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ARP180100629A AR111292A1 (es) 2017-03-17 2018-03-19 Administración de vectores del virus adenoasociado de microdistrofina específica del músculo para tratar la distrofia muscular

Country Status (30)

Country Link
US (2) US20200199621A1 (es)
EP (2) EP4245852A3 (es)
JP (2) JP2020513811A (es)
KR (2) KR20240112964A (es)
CN (2) CN110997923B (es)
AR (1) AR111292A1 (es)
AU (1) AU2018233732A1 (es)
BR (1) BR112019019248A2 (es)
CA (1) CA3056638A1 (es)
CO (1) CO2019011250A2 (es)
CY (1) CY1126080T1 (es)
DK (1) DK3596222T5 (es)
EA (1) EA201992201A1 (es)
ES (1) ES2948233T3 (es)
FI (1) FI3596222T3 (es)
HR (1) HRP20230522T1 (es)
HU (1) HUE062476T2 (es)
IL (2) IL269391B2 (es)
LT (1) LT3596222T (es)
MA (1) MA52112B1 (es)
MD (1) MD3596222T2 (es)
MX (1) MX2019011046A (es)
PL (1) PL3596222T3 (es)
PT (1) PT3596222T (es)
RS (1) RS64299B1 (es)
SG (1) SG11201908575SA (es)
SI (1) SI3596222T1 (es)
TW (1) TW201840850A (es)
WO (1) WO2018170408A1 (es)
ZA (1) ZA201906251B (es)

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EP4108263A3 (en) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
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Publication number Publication date
ZA201906251B (en) 2024-01-31
EP4245852A3 (en) 2023-11-22
CN110997923B (zh) 2024-01-02
EP4245852A2 (en) 2023-09-20
SG11201908575SA (en) 2019-10-30
JP2023053254A (ja) 2023-04-12
HRP20230522T1 (hr) 2023-08-04
MD3596222T2 (ro) 2023-08-31
CN118497275A (zh) 2024-08-16
KR102683682B1 (ko) 2024-07-09
US20220364117A1 (en) 2022-11-17
MA52112B1 (fr) 2023-08-31
IL269391B2 (en) 2024-09-01
IL311713A (en) 2024-05-01
ES2948233T3 (es) 2023-09-06
KR20190130591A (ko) 2019-11-22
MA52112A (fr) 2020-01-22
FI3596222T3 (fi) 2023-06-08
IL269391B1 (en) 2024-05-01
DK3596222T5 (da) 2024-09-02
DK3596222T3 (da) 2023-06-19
CA3056638A1 (en) 2018-09-20
SI3596222T1 (sl) 2024-05-31
PT3596222T (pt) 2023-06-20
CY1126080T1 (el) 2023-11-15
EP3596222B1 (en) 2023-04-12
PL3596222T3 (pl) 2023-10-09
US20200199621A1 (en) 2020-06-25
AU2018233732A1 (en) 2019-10-03
EP3596222A4 (en) 2021-01-06
BR112019019248A2 (pt) 2020-04-28
CN110997923A (zh) 2020-04-10
CO2019011250A2 (es) 2020-02-28
MX2019011046A (es) 2019-10-17
LT3596222T (lt) 2023-07-25
RS64299B1 (sr) 2023-07-31
EP3596222A1 (en) 2020-01-22
TW201840850A (zh) 2018-11-16
EA201992201A1 (ru) 2020-03-19
KR20240112964A (ko) 2024-07-19
JP2020513811A (ja) 2020-05-21
HUE062476T2 (hu) 2023-11-28
IL269391A (en) 2019-11-28
WO2018170408A1 (en) 2018-09-20

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