CY1126080T1 - Χορηγηση αδενο-σχετιζομενου ιικου διαβιβαστη μικρο-δυστροφινης με εξειδικευση στους μυς για τη θεραπευτικη αντιμετωπιση της μυïκης δυστροφιας - Google Patents

Χορηγηση αδενο-σχετιζομενου ιικου διαβιβαστη μικρο-δυστροφινης με εξειδικευση στους μυς για τη θεραπευτικη αντιμετωπιση της μυïκης δυστροφιας

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Publication number
CY1126080T1
CY1126080T1 CY20231100291T CY231100291T CY1126080T1 CY 1126080 T1 CY1126080 T1 CY 1126080T1 CY 20231100291 T CY20231100291 T CY 20231100291T CY 231100291 T CY231100291 T CY 231100291T CY 1126080 T1 CY1126080 T1 CY 1126080T1
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Cyprus
Prior art keywords
muscle
muscular dystrophy
dystrofin
virus
administration
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CY20231100291T
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English (en)
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Louise RODINO-KLAPAC
Jerry R. Mendell
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Research Institute At Nationwide Children's Hospital
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Publication of CY1126080T1 publication Critical patent/CY1126080T1/el

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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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Abstract

Η εφεύρεση παρέχει διαβιβαστές γονιδιακής θεραπείας, όπως αδενο-σχετιζόμενους ιικούς (AAV) διαβιβαστές, που εκφράζουν ένα μικροσκοπικό ανθρώπινο γονίδιο μικρο-δυστροφίνης και μέθοδο χρήσης αυτών των διαβιβαστών για την έκφραση της μικρο-δυστροφίνης σε σκελετικούς μυς, συμπεριλαμβανομένου του διαφράγματος και του καρδιακού μυός και για την προστασία των μυϊκών ινών από βλάβη, την αύξηση της μυϊκής δύναμης και για τη μείωση και/ή την πρόληψη της ίνωσης σε υποκείμενα που πάσχουν από μυϊκή δυστροφία.
CY20231100291T 2017-03-17 2023-06-21 Χορηγηση αδενο-σχετιζομενου ιικου διαβιβαστη μικρο-δυστροφινης με εξειδικευση στους μυς για τη θεραπευτικη αντιμετωπιση της μυïκης δυστροφιας CY1126080T1 (el)

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
CY1126080T1 true CY1126080T1 (el) 2023-11-15

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CY20231100291T CY1126080T1 (el) 2017-03-17 2023-06-21 Χορηγηση αδενο-σχετιζομενου ιικου διαβιβαστη μικρο-δυστροφινης με εξειδικευση στους μυς για τη θεραπευτικη αντιμετωπιση της μυïκης δυστροφιας

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US (2) US20200199621A1 (el)
EP (2) EP4245852A3 (el)
JP (2) JP2020513811A (el)
CN (1) CN110997923B (el)
AR (1) AR111292A1 (el)
AU (1) AU2018233732A1 (el)
BR (1) BR112019019248A2 (el)
CA (1) CA3056638A1 (el)
CO (1) CO2019011250A2 (el)
CY (1) CY1126080T1 (el)
DK (1) DK3596222T3 (el)
EA (1) EA201992201A1 (el)
ES (1) ES2948233T3 (el)
FI (1) FI3596222T3 (el)
HR (1) HRP20230522T1 (el)
HU (1) HUE062476T2 (el)
IL (2) IL311713A (el)
LT (1) LT3596222T (el)
MA (1) MA52112B1 (el)
MD (1) MD3596222T2 (el)
MX (1) MX2019011046A (el)
PL (1) PL3596222T3 (el)
PT (1) PT3596222T (el)
RS (1) RS64299B1 (el)
SG (1) SG11201908575SA (el)
SI (1) SI3596222T1 (el)
TW (1) TW201840850A (el)
WO (1) WO2018170408A1 (el)
ZA (1) ZA201906251B (el)

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SG11201908575SA (en) 2019-10-30
JP2020513811A (ja) 2020-05-21
CA3056638A1 (en) 2018-09-20
DK3596222T3 (da) 2023-06-19
JP2023053254A (ja) 2023-04-12
IL269391A (en) 2019-11-28
US20220364117A1 (en) 2022-11-17
BR112019019248A2 (pt) 2020-04-28
EP3596222B1 (en) 2023-04-12
FI3596222T3 (fi) 2023-06-08
EP4245852A2 (en) 2023-09-20
AR111292A1 (es) 2019-06-26
IL311713A (en) 2024-05-01
RS64299B1 (sr) 2023-07-31
CO2019011250A2 (es) 2020-02-28
HUE062476T2 (hu) 2023-11-28
ZA201906251B (en) 2024-01-31
MA52112A (fr) 2020-01-22
EA201992201A1 (ru) 2020-03-19
MA52112B1 (fr) 2023-08-31
AU2018233732A1 (en) 2019-10-03
LT3596222T (lt) 2023-07-25
PL3596222T3 (pl) 2023-10-09
MD3596222T2 (ro) 2023-08-31
CN110997923B (zh) 2024-01-02
EP3596222A1 (en) 2020-01-22
PT3596222T (pt) 2023-06-20
US20200199621A1 (en) 2020-06-25
HRP20230522T1 (hr) 2023-08-04
IL269391B1 (en) 2024-05-01
SI3596222T1 (sl) 2024-05-31
EP3596222A4 (en) 2021-01-06
WO2018170408A1 (en) 2018-09-20
KR20190130591A (ko) 2019-11-22
CN110997923A (zh) 2020-04-10
ES2948233T3 (es) 2023-09-06
EP4245852A3 (en) 2023-11-22
MX2019011046A (es) 2019-10-17
TW201840850A (zh) 2018-11-16

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