MX2019011046A - Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular. - Google Patents

Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular.

Info

Publication number
MX2019011046A
MX2019011046A MX2019011046A MX2019011046A MX2019011046A MX 2019011046 A MX2019011046 A MX 2019011046A MX 2019011046 A MX2019011046 A MX 2019011046A MX 2019011046 A MX2019011046 A MX 2019011046A MX 2019011046 A MX2019011046 A MX 2019011046A
Authority
MX
Mexico
Prior art keywords
dystrophin
adeno
associated virus
muscular dystrophy
virus vector
Prior art date
Application number
MX2019011046A
Other languages
English (en)
Inventor
R Mendell Jerry
Rodino-Klapac Louise
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 MX2019011046A publication Critical patent/MX2019011046A/es

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Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0029—Parenteral nutrition; Parenteral nutrition compositions as drug carriers
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/63—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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Abstract

La invención proporciona vectores de tratamiento génico, tales como vectores de virus adenoasociado (AAV), que expresan un gen de microdistrofina humana miniaturizado y un método de uso de estos vectores para expresar la microdistrofina en músculos esqueléticos que incluyen el diafragma y el músculo cardíaco, y para proteger las fibras musculares de una lesión, aumentar la fuerza muscular y reducir y/o prevenir la fibrosis en sujetos que padecen distrofia muscular.
MX2019011046A 2017-03-17 2018-03-16 Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular. MX2019011046A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762473148P 2017-03-17 2017-03-17
PCT/US2018/022881 WO2018170408A1 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Publications (1)

Publication Number Publication Date
MX2019011046A true MX2019011046A (es) 2019-10-17

Family

ID=63523321

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019011046A MX2019011046A (es) 2017-03-17 2018-03-16 Administracion de vectores del virus adenoasociado de microdistrofina especifica del musculo para tratar la distrofia muscular.

Country Status (28)

Country Link
US (2) US20200199621A1 (es)
EP (2) EP3596222B1 (es)
JP (2) JP2020513811A (es)
KR (1) KR20190130591A (es)
CN (1) 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) DK3596222T3 (es)
EA (1) EA201992201A1 (es)
ES (1) ES2948233T3 (es)
FI (1) FI3596222T3 (es)
HR (1) HRP20230522T1 (es)
HU (1) HUE062476T2 (es)
IL (1) IL269391A (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)
TW (1) TW201840850A (es)
WO (1) WO2018170408A1 (es)

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Publication number Publication date
PL3596222T3 (pl) 2023-10-09
MA52112A (fr) 2020-01-22
TW201840850A (zh) 2018-11-16
JP2020513811A (ja) 2020-05-21
SG11201908575SA (en) 2019-10-30
PT3596222T (pt) 2023-06-20
KR20190130591A (ko) 2019-11-22
IL269391A (en) 2019-11-28
CO2019011250A2 (es) 2020-02-28
RS64299B1 (sr) 2023-07-31
MA52112B1 (fr) 2023-08-31
EA201992201A1 (ru) 2020-03-19
BR112019019248A2 (pt) 2020-04-28
AR111292A1 (es) 2019-06-26
EP4245852A3 (en) 2023-11-22
JP2023053254A (ja) 2023-04-12
CY1126080T1 (el) 2023-11-15
CN110997923A (zh) 2020-04-10
EP3596222A1 (en) 2020-01-22
EP3596222B1 (en) 2023-04-12
DK3596222T3 (da) 2023-06-19
LT3596222T (lt) 2023-07-25
EP4245852A2 (en) 2023-09-20
FI3596222T3 (fi) 2023-06-08
MD3596222T2 (ro) 2023-08-31
CA3056638A1 (en) 2018-09-20
AU2018233732A1 (en) 2019-10-03
EP3596222A4 (en) 2021-01-06
HUE062476T2 (hu) 2023-11-28
ES2948233T3 (es) 2023-09-06
CN110997923B (zh) 2024-01-02
WO2018170408A1 (en) 2018-09-20
US20200199621A1 (en) 2020-06-25
US20220364117A1 (en) 2022-11-17
HRP20230522T1 (hr) 2023-08-04

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