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ES2629380T3 - Viriones de AAV con inmunorreactividad reducida y usos de los mismos - Google Patents

Viriones de AAV con inmunorreactividad reducida y usos de los mismos Download PDF

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Publication number
ES2629380T3
ES2629380T3 ES13163182T ES13163182T ES2629380T3 ES 2629380 T3 ES2629380 T3 ES 2629380T3 ES 13163182 T ES13163182 T ES 13163182T ES 13163182 T ES13163182 T ES 13163182T ES 2629380 T3 ES2629380 T3 ES 2629380T3
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ES
Spain
Prior art keywords
aav
amino acid
aav virions
virions
reduced immunoreactivity
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.)
Expired - Lifetime
Application number
ES13163182T
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English (en)
Inventor
Alejandra Elena Arbetman
Peter C. Colosi
Michael A Lochrie
Richard T Surosky
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Genzyme Corp
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Genzyme Corp
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Publication date
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Publication of ES2629380T3 publication Critical patent/ES2629380T3/es
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    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • 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
    • 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
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    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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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
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14161—Methods of inactivation or attenuation
    • C12N2750/14162—Methods of inactivation or attenuation by genetic engineering
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

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Abstract

Uso de una proteína de la cápside de virus adeno-asociado (AAV) recombinante, en un virión de AAV recombinante, para reducir la inmunorreactividad de los viriones en comparación con los correspondientes viriones de tipo salvaje, en donde dicha proteína de la cápside de AAV mutado comprende una sustitución del aminoácido que se produce en una posición correspondiente a la posición en la cápside VPA de AAV-2 seleccionada del grupo que consiste en el aminoácido 357 y el aminoácido 395.
ES13163182T 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos Expired - Lifetime ES2629380T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US48039503P 2003-06-19 2003-06-19
US56731004P 2004-04-30 2004-04-30
US57650104P 2004-06-03 2004-06-03

Publications (1)

Publication Number Publication Date
ES2629380T3 true ES2629380T3 (es) 2017-08-09

Family

ID=33545345

Family Applications (5)

Application Number Title Priority Date Filing Date
ES17162137T Expired - Lifetime ES2862206T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad disminuida y sus usos
ES04755800.2T Expired - Lifetime ES2563064T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES13163168.1T Expired - Lifetime ES2629087T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES13163182T Expired - Lifetime ES2629380T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES11158742.4T Expired - Lifetime ES2627913T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos

Family Applications Before (3)

Application Number Title Priority Date Filing Date
ES17162137T Expired - Lifetime ES2862206T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad disminuida y sus usos
ES04755800.2T Expired - Lifetime ES2563064T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES13163168.1T Expired - Lifetime ES2629087T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES11158742.4T Expired - Lifetime ES2627913T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos

Country Status (11)

Country Link
US (6) US7259151B2 (es)
EP (5) EP2657247B1 (es)
JP (7) JP2007524386A (es)
CY (5) CY1117575T1 (es)
DK (5) DK1633772T3 (es)
ES (5) ES2862206T3 (es)
HU (5) HUE034546T2 (es)
PL (5) PL3235827T3 (es)
PT (5) PT3235827T (es)
SI (5) SI2657248T1 (es)
WO (1) WO2004112727A2 (es)

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* Cited by examiner, † Cited by third party
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US20030215422A1 (en) 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
CA2745131C (en) 1998-05-28 2016-08-09 John A. Chiorini Aav5 vector and uses thereof
EP1190249A2 (en) 1999-06-08 2002-03-27 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
BRPI0214119B8 (pt) 2001-11-13 2021-05-25 Univ Pennsylvania vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
EP1608410A2 (en) 2003-03-31 2005-12-28 University of Iowa Research Foundation Pharmico-gene theraphy of epithelial sodium channel associated disoders
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
ES2862206T3 (es) 2003-06-19 2021-10-07 Genzyme Corp Viriones de AAV con inmunorreactividad disminuida y sus usos
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
WO2006029196A1 (en) * 2004-09-08 2006-03-16 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of H Ealth Transcytosis of adeno-associated viruses
US7892809B2 (en) * 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
EP2359867B1 (en) * 2005-04-07 2014-10-08 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
CA2651180A1 (en) * 2006-04-28 2007-11-08 University Of Iowa Research Foundation Methods and compounds to alter virus infection
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
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