MX2018007330A - Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. - Google Patents

Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.

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Publication number
MX2018007330A
MX2018007330A MX2018007330A MX2018007330A MX2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A
Authority
MX
Mexico
Prior art keywords
mucolipidosis type
iii
vectors
raav
adeno
Prior art date
Application number
MX2018007330A
Other languages
English (en)
Spanish (es)
Inventor
Yew Nelson
Jin Dong-Kyu
Original Assignee
Genzyme Corp
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 filed Critical Genzyme Corp
Publication of MX2018007330A publication Critical patent/MX2018007330A/es

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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
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    • 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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/10—Laxatives
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
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    • A61P25/00—Drugs for disorders of the nervous system
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    • 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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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
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    • 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
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    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • 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
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    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/00—Animals characterised by purpose
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
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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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  • Diabetes (AREA)
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MX2018007330A 2015-12-15 2016-12-14 Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. MX2018007330A (es)

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
MX2018007330A true MX2018007330A (es) 2019-03-14

Family

ID=57737987

Family Applications (1)

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MX2018007330A MX2018007330A (es) 2015-12-15 2016-12-14 Vectores virales adeno-asociados para tratar mucolipidosis de tipo 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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US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP1073471A4 (en) * 1998-04-27 2005-06-01 Univ Temple METHOD OF TREATING ENDOTOXEMIA
US6534300B1 (en) * 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
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CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
JP2005523942A (ja) * 2002-04-30 2005-08-11 アヴィオル セラピューティクス インコーポレイテッド 免疫療法のためのアデノウイルスベクター
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
PT1625210E (pt) 2003-05-21 2011-03-15 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
PT3252161T (pt) * 2007-06-06 2022-02-01 Genzyme Corp Terapia genética para doenças do armazenamento lisossomal
CN102803478B (zh) 2009-06-16 2016-04-27 建新公司 用于纯化重组aav载体的改进方法
DK2588130T3 (en) * 2010-06-25 2016-10-24 Shire Human Genetic Therapies Cns delivery of therapeutic agents
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JP6224459B2 (ja) 2011-02-17 2017-11-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
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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
CA3008268A1 (en) 2017-06-22
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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