CY1123760T1 - Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα - Google Patents

Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα

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Publication number
CY1123760T1
CY1123760T1 CY20201101100T CY201101100T CY1123760T1 CY 1123760 T1 CY1123760 T1 CY 1123760T1 CY 20201101100 T CY20201101100 T CY 20201101100T CY 201101100 T CY201101100 T CY 201101100T CY 1123760 T1 CY1123760 T1 CY 1123760T1
Authority
CY
Cyprus
Prior art keywords
osteoprotective
has2
osteoarthritis
lubricin
mammals
Prior art date
Application number
CY20201101100T
Other languages
English (en)
Inventor
Monica DIAS FIGUEIREDO
Sirkka RM KYOSTIO-MOORE
Patricia Berthelette
Original Assignee
Genzyme Corporation
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 Genzyme Corporation filed Critical Genzyme Corporation
Publication of CY1123760T1 publication Critical patent/CY1123760T1/el

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    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin, cold insoluble globulin [CIG]
    • 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
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/01—Hexosyltransferases (2.4.1)
    • C12Y204/01212—Hyaluronan synthase (2.4.1.212)
    • 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
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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

Abstract

Η παρούσα εφεύρεση αναφέρεται σε ανασυνδυασμένους ιϊκούς φορείς, σε φαρμακευτικές συνθέσεις που αποτελούνται από τέτοιους ανασυνδυασμένους φορείς, και σε μεθόδους για την πρόληψη και θεραπευτική αγωγή οστεοαρθρίτιδας στα θηλαστικά. Ειδικότερα, αυτή η αποκάλυψη παρέχει φορείς αδενο-σχετιζόμενού ιού (AAV) ικανούς να εκφράζουν, σε έναν ξενιστή, οστεοπροστατευτικές/ χονδροπροστατευτικές βιοδραστικές πρωτεΐνες, που περιλαμβάνουν συνθάση 2 υαλουρονάνης 2 (HAS2) και λουμπρικίνη (PRG4). Μέθοδοι παραγωγής αυτών των AAV παρέχονται, καθώς είναι μέθοδοι αντιμετώπισης της οστεοαρθρίτιδας στις αρθρώσεις θηλαστικών, μέσω της μακροπρόθεσμης γονιδιακής έκφρασης των οστεοπροστατευτικών /χονδροπροστατευτικών πρωτεϊνών, που περιλαμβάνουν HAS2 και PRG4, τόσο σε αρθρικά κύτταρα όσο και σε χονδροκυτταρικά κύτταρα.
CY20201101100T 2016-01-13 2020-11-19 Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα CY1123760T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662278243P 2016-01-13 2016-01-13
PCT/US2017/013419 WO2017123934A1 (en) 2016-01-13 2017-01-13 Recombinant aav vectors expressing osteoprotective genes, including has2 and lubricin, useful in the treatment of osteoarthritis and related joint conditions in mammals

Publications (1)

Publication Number Publication Date
CY1123760T1 true CY1123760T1 (el) 2022-03-24

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CY20201101100T CY1123760T1 (el) 2016-01-13 2020-11-19 Ανασυνδυασμενοι φορεις aav που εκφραζουν οστεοπροστατευτικα γονιδια, που περιλαμβανουν has2 και λουμπρικινη, χρησιμοι στην θεραπευτικη αγωγη οστεοαρθριτιδας και συναφων παθησεων των αρθρωσεων στα θηλαστικα

Country Status (16)

Country Link
US (2) US20170198263A1 (el)
EP (2) EP3402530B1 (el)
JP (2) JP7058603B2 (el)
KR (1) KR20180095720A (el)
CN (1) CN108697813B (el)
AR (1) AR108683A1 (el)
AU (1) AU2017207917B2 (el)
CA (1) CA3011459A1 (el)
CY (1) CY1123760T1 (el)
DK (1) DK3402530T3 (el)
ES (1) ES2835031T3 (el)
HU (1) HUE051774T2 (el)
MX (2) MX2018008651A (el)
RU (1) RU2771490C2 (el)
WO (1) WO2017123934A1 (el)
ZA (1) ZA201804731B (el)

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Also Published As

Publication number Publication date
US20170198263A1 (en) 2017-07-13
MX2021004397A (es) 2021-06-08
ZA201804731B (en) 2019-05-29
AU2017207917A1 (en) 2018-08-16
ES2835031T3 (es) 2021-06-21
EP3769788A1 (en) 2021-01-27
AR108683A1 (es) 2018-09-19
JP2022058602A (ja) 2022-04-12
JP2019506859A (ja) 2019-03-14
US20200231940A1 (en) 2020-07-23
CA3011459A1 (en) 2017-07-20
WO2017123934A1 (en) 2017-07-20
MX2018008651A (es) 2019-03-14
CN108697813A (zh) 2018-10-23
JP7058603B2 (ja) 2022-04-22
CN108697813B (zh) 2024-01-16
US11905531B2 (en) 2024-02-20
EP3402530A1 (en) 2018-11-21
RU2018129159A3 (el) 2020-03-27
DK3402530T3 (da) 2020-11-23
EP3402530B1 (en) 2020-08-19
RU2771490C2 (ru) 2022-05-05
RU2018129159A (ru) 2020-02-13
HUE051774T2 (hu) 2021-03-29
AU2017207917B2 (en) 2024-03-07
KR20180095720A (ko) 2018-08-27

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