CO2021010088A2 - Construcciones de terapia génica para el tratamiento de la enfermedad de wilson - Google Patents

Construcciones de terapia génica para el tratamiento de la enfermedad de wilson

Info

Publication number
CO2021010088A2
CO2021010088A2 CONC2021/0010088A CO2021010088A CO2021010088A2 CO 2021010088 A2 CO2021010088 A2 CO 2021010088A2 CO 2021010088 A CO2021010088 A CO 2021010088A CO 2021010088 A2 CO2021010088 A2 CO 2021010088A2
Authority
CO
Colombia
Prior art keywords
disease
gene therapy
wilson
treatment
atp7b
Prior art date
Application number
CONC2021/0010088A
Other languages
English (en)
Inventor
Christine Livingston
Samuel Wadsworth
Original Assignee
Ultragenyx Pharmaceutical Inc
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 Ultragenyx Pharmaceutical Inc filed Critical Ultragenyx Pharmaceutical Inc
Publication of CO2021010088A2 publication Critical patent/CO2021010088A2/es

Links

Classifications

    • 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
    • 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
    • A61K48/005—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • 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
    • 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

RESUMEN DE LA INVENCIÓN Esta solicitud se refiere generalmente a vectores virales adenoasociados que codifican un ATP7B truncado aunque funcional para usarse en la terapia génica para tratar la enfermedad de Wilson (WD); el ATP7B truncado descrito en este documento tiene varias ventajas sobre el ATP7B de tipo salvaje, tales como una mayor eficacia y un rendimiento de fabricación mejorado.
CONC2021/0010088A 2019-01-04 2021-07-29 Construcciones de terapia génica para el tratamiento de la enfermedad de wilson CO2021010088A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962788324P 2019-01-04 2019-01-04
US201962834830P 2019-04-16 2019-04-16
PCT/US2020/012131 WO2020142653A1 (en) 2019-01-04 2020-01-03 Gene therapy constructs for treating wilson disease

Publications (1)

Publication Number Publication Date
CO2021010088A2 true CO2021010088A2 (es) 2021-09-20

Family

ID=69528954

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0010088A CO2021010088A2 (es) 2019-01-04 2021-07-29 Construcciones de terapia génica para el tratamiento de la enfermedad de wilson

Country Status (17)

Country Link
US (1) US20220090131A1 (es)
EP (2) EP3906066B1 (es)
JP (2) JP2022516283A (es)
KR (1) KR20210112339A (es)
CN (1) CN113518628A (es)
AU (1) AU2020204679A1 (es)
BR (1) BR112021013140A2 (es)
CA (1) CA3124880A1 (es)
CO (1) CO2021010088A2 (es)
DK (1) DK3906066T3 (es)
FI (1) FI3906066T3 (es)
IL (1) IL284554A (es)
MX (1) MX2021008135A (es)
PT (1) PT3906066T (es)
SG (1) SG11202106855YA (es)
TW (1) TW202043479A (es)
WO (1) WO2020142653A1 (es)

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KR102604096B1 (ko) 2016-12-30 2023-11-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 윌슨병을 치료하기 위한 유전자 치료
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
CN114072514A (zh) * 2019-05-14 2022-02-18 杜克大学 用于治疗atp酶介导的疾病的组合物和方法
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CN115216481A (zh) * 2021-04-21 2022-10-21 四川至善唯新生物科技有限公司 一种表达量提高的截短型atp7b基因及其应用
CN113694218A (zh) * 2021-08-30 2021-11-26 昆明理工大学 一种atp7b基因p992l突变的基因修复治疗载体
WO2023108159A1 (en) * 2021-12-10 2023-06-15 University Of Florida Research Foundation, Incorporated Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector
WO2023108130A2 (en) * 2021-12-10 2023-06-15 Aavantibio, Inc. Compositions comprising kozak sequences selected for enhanced expression

Family Cites Families (14)

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US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
PT1625210E (pt) 2003-05-21 2011-03-15 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CN105838737A (zh) 2010-01-28 2016-08-10 费城儿童医院 用于病毒载体纯化的可扩缩生产平台和用于基因治疗中的如此纯化的病毒载体
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
ES2856090T3 (es) 2014-09-24 2021-09-27 Hope City Variantes de vectores de virus adenoasociados para la edición genómica de alta eficacia y sus métodos
IL252917B2 (en) * 2014-12-17 2023-10-01 Fundacion Para La Investig Medica Aplicada Vectors and vectors for gene therapy for use in the treatment of Wilson's disease
KR102604096B1 (ko) * 2016-12-30 2023-11-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 윌슨병을 치료하기 위한 유전자 치료

Also Published As

Publication number Publication date
JP2024054426A (ja) 2024-04-16
EP3906066A1 (en) 2021-11-10
FI3906066T3 (fi) 2024-01-30
US20220090131A1 (en) 2022-03-24
EP4269579A2 (en) 2023-11-01
TW202043479A (zh) 2020-12-01
SG11202106855YA (en) 2021-07-29
EP4269579A3 (en) 2024-01-24
JP2022516283A (ja) 2022-02-25
DK3906066T3 (da) 2024-02-05
IL284554A (en) 2021-08-31
PT3906066T (pt) 2024-02-06
AU2020204679A1 (en) 2021-07-15
KR20210112339A (ko) 2021-09-14
CN113518628A (zh) 2021-10-19
EP3906066B1 (en) 2023-11-01
WO2020142653A1 (en) 2020-07-09
CA3124880A1 (en) 2020-07-09
BR112021013140A2 (pt) 2021-11-30
MX2021008135A (es) 2021-08-11

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