SG11201804994QA - Adeno-associated viral vectors for treating mucolipidosis type ii - Google Patents

Adeno-associated viral vectors for treating mucolipidosis type ii

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Publication number
SG11201804994QA
SG11201804994QA SG11201804994QA SG11201804994QA SG11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA
Authority
SG
Singapore
Prior art keywords
international
mucolipidosis type
vectors
iii
pct
Prior art date
Application number
SG11201804994QA
Other languages
English (en)
Inventor
Nelson Yew
Dong-Kyu Jin
Original Assignee
Genzyme Corp
Samsung Life Public Welfare Foundation
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 Corp, Samsung Life Public Welfare Foundation filed Critical Genzyme Corp
Publication of SG11201804994QA publication Critical patent/SG11201804994QA/en

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    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1288—Transferases for other substituted phosphate groups (2.7.8)
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
    • C12Y207/08017—UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Physical Education & Sports Medicine (AREA)
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  • Animal Husbandry (AREA)
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  • Diabetes (AREA)
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SG11201804994QA 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii SG11201804994QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267502P 2015-12-15 2015-12-15
PCT/US2016/066611 WO2017106313A1 (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

Publications (1)

Publication Number Publication Date
SG11201804994QA true SG11201804994QA (en) 2018-07-30

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SG11201804994QA SG11201804994QA (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii
SG10201912935WA SG10201912935WA (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

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SG10201912935WA SG10201912935WA (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

Country Status (13)

Country Link
US (1) US20200261598A1 (https=)
EP (1) EP3390644B1 (https=)
JP (3) JP7347932B2 (https=)
KR (1) KR102833376B1 (https=)
CN (1) CN108603199A (https=)
AU (2) AU2016372035B2 (https=)
CA (1) CA3008268A1 (https=)
ES (1) ES2991277T3 (https=)
IL (1) IL259964B (https=)
MX (1) MX2018007330A (https=)
RU (1) RU2742612C2 (https=)
SG (2) SG11201804994QA (https=)
WO (1) WO2017106313A1 (https=)

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WO2020106916A1 (en) * 2018-11-21 2020-05-28 Stridebio, Inc. Recombinant viral vectors and nucleic acids for producing the same
CA3158131A1 (en) * 2019-10-18 2021-04-22 Nicolas Sebastien Wein Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
EP4103214B1 (en) * 2020-02-13 2026-04-29 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease

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SG10201912935WA (en) 2020-02-27
AU2023204116A1 (en) 2023-07-13
US20200261598A1 (en) 2020-08-20
WO2017106313A1 (en) 2017-06-22
AU2023204116B2 (en) 2026-03-12
ES2991277T3 (es) 2024-12-03
MX2018007330A (es) 2019-03-14
JP2019500034A (ja) 2019-01-10
AU2016372035B2 (en) 2023-03-30
KR20180098294A (ko) 2018-09-03
IL259964A (en) 2018-07-31
RU2018124448A3 (https=) 2020-08-14
BR112018012015A2 (pt) 2018-12-04
IL259964B (en) 2022-09-01
JP7347932B2 (ja) 2023-09-20
EP3390644A1 (en) 2018-10-24
CN108603199A (zh) 2018-09-28
JP2022046635A (ja) 2022-03-23
KR102833376B1 (ko) 2025-07-11
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