CA3008268A1 - Adeno-associated viral vectors for treating mucolipidosis type ii - Google Patents

Adeno-associated viral vectors for treating mucolipidosis type ii Download PDF

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Publication number
CA3008268A1
CA3008268A1 CA3008268A CA3008268A CA3008268A1 CA 3008268 A1 CA3008268 A1 CA 3008268A1 CA 3008268 A CA3008268 A CA 3008268A CA 3008268 A CA3008268 A CA 3008268A CA 3008268 A1 CA3008268 A1 CA 3008268A1
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CA
Canada
Prior art keywords
aav
raav
gnptab
mammal
iii
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3008268A
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English (en)
French (fr)
Inventor
Nelson Yew
Dong-Kyu JIN
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Samsung Life Public Welfare Foundation
Genzyme Corp
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Samsung Life Public Welfare Foundation
Genzyme Corp
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Application filed by Samsung Life Public Welfare Foundation, Genzyme Corp filed Critical Samsung Life Public Welfare Foundation
Publication of CA3008268A1 publication Critical patent/CA3008268A1/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
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    • A—HUMAN NECESSITIES
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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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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  • Chemical Kinetics & Catalysis (AREA)
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  • Physical Education & Sports Medicine (AREA)
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  • Diabetes (AREA)
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CA3008268A 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii Pending CA3008268A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267502P 2015-12-15 2015-12-15
US62/267,502 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
CA3008268A1 true CA3008268A1 (en) 2017-06-22

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CA3008268A Pending CA3008268A1 (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

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

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WO2019094061A1 (en) 2017-11-07 2019-05-16 The University Of North Carolina At Chapel Hill Optimized aga genes and expression cassettes and their use
EA202191418A1 (ru) * 2018-11-21 2021-08-05 Страйдбайо, Инк. Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения
US20220389453A1 (en) * 2019-10-18 2022-12-08 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated with the irf2bpl gene
MY209860A (en) * 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease

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JP2019500034A (ja) 2019-01-10
JP2024056832A (ja) 2024-04-23
JP2022046635A (ja) 2022-03-23
EP3390644A1 (en) 2018-10-24
SG10201912935WA (en) 2020-02-27
EP3390644B1 (en) 2024-07-31
ES2991277T3 (es) 2024-12-03
RU2018124448A3 (enExample) 2020-08-14
US20200261598A1 (en) 2020-08-20
KR20180098294A (ko) 2018-09-03
WO2017106313A1 (en) 2017-06-22
SG11201804994QA (en) 2018-07-30
MX2018007330A (es) 2019-03-14
IL259964A (en) 2018-07-31
RU2018124448A (ru) 2020-01-17
AU2016372035B2 (en) 2023-03-30
JP7347932B2 (ja) 2023-09-20
AU2023204116A1 (en) 2023-07-13
KR102833376B1 (ko) 2025-07-11
RU2742612C2 (ru) 2021-02-09
IL259964B (en) 2022-09-01
AU2016372035A1 (en) 2018-08-02
CN108603199A (zh) 2018-09-28
BR112018012015A2 (pt) 2018-12-04

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