DE69824859D1 - Methoden zur erhöhung der effizienz rekombinanter aav produkte - Google Patents

Methoden zur erhöhung der effizienz rekombinanter aav produkte

Info

Publication number
DE69824859D1
DE69824859D1 DE69824859T DE69824859T DE69824859D1 DE 69824859 D1 DE69824859 D1 DE 69824859D1 DE 69824859 T DE69824859 T DE 69824859T DE 69824859 T DE69824859 T DE 69824859T DE 69824859 D1 DE69824859 D1 DE 69824859D1
Authority
DE
Germany
Prior art keywords
methods
aav
production
expression
compositions
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
DE69824859T
Other languages
English (en)
Other versions
DE69824859T2 (de
Inventor
J Samulski
Xiao Xiao
Richard Snyder
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.)
University of North Carolina at Chapel Hill
Cell Genesys Inc
University of North Carolina System
Original Assignee
University of North Carolina at Chapel Hill
Cell Genesys Inc
University of North Carolina System
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 University of North Carolina at Chapel Hill, Cell Genesys Inc, University of North Carolina System filed Critical University of North Carolina at Chapel Hill
Application granted granted Critical
Publication of DE69824859D1 publication Critical patent/DE69824859D1/de
Publication of DE69824859T2 publication Critical patent/DE69824859T2/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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    • 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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14151—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
DE69824859T 1997-04-14 1998-04-14 Methoden zur erhöhung der effizienz rekombinanter aav produkte Expired - Lifetime DE69824859T2 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4354797P 1997-04-14 1997-04-14
US43547P 1997-04-14
PCT/US1998/007654 WO1998046728A1 (en) 1997-04-14 1998-04-14 Methods for increasing the efficiency of recombinant aav product

Publications (2)

Publication Number Publication Date
DE69824859D1 true DE69824859D1 (de) 2004-08-05
DE69824859T2 DE69824859T2 (de) 2005-08-04

Family

ID=21927717

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69824859T Expired - Lifetime DE69824859T2 (de) 1997-04-14 1998-04-14 Methoden zur erhöhung der effizienz rekombinanter aav produkte

Country Status (10)

Country Link
US (2) US6548286B1 (de)
EP (1) EP1007637B1 (de)
JP (2) JP4135120B2 (de)
AT (1) ATE270324T1 (de)
AU (1) AU728220B2 (de)
CA (1) CA2287478C (de)
DE (1) DE69824859T2 (de)
ES (1) ES2224375T3 (de)
NZ (1) NZ500546A (de)
WO (1) WO1998046728A1 (de)

Families Citing this family (127)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6548286B1 (en) * 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production
US6995006B2 (en) * 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
US6346415B1 (en) * 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US7078387B1 (en) 1998-12-28 2006-07-18 Arch Development Corp. Efficient and stable in vivo gene transfer to cardiomyocytes using recombinant adeno-associated virus vectors
AU7841400A (en) * 1999-10-01 2001-05-10 Genovo, Incorporated Production of recombinant aav using adenovirus comprising aav rep/cap genes
US7115391B1 (en) * 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
JP2002153278A (ja) * 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
US20040197312A1 (en) 2003-04-02 2004-10-07 Marina Moskalenko Cytokine-expressing cellular vaccine combinations
US7186699B2 (en) * 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
US7485291B2 (en) * 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
US20050136035A1 (en) * 2003-06-03 2005-06-23 Derek Ko Cell specific replication-competent viral vectors comprising a self processing peptide cleavage site
JP4644663B2 (ja) * 2003-06-03 2011-03-02 セル ジェネシス インコーポレイテッド ペプチド開裂部位を用いた単一ベクターからの組換えポリペプチドの高められた発現のための構成と方法
WO2005089505A2 (en) 2004-03-19 2005-09-29 Yale University Detection, isolation and uses of renalase (monoamine oxidase c)
CA2571159A1 (en) * 2004-06-18 2006-03-30 The University Of Montana Aav mediated gene delivery to cochlear cells
US7714119B2 (en) * 2004-07-13 2010-05-11 Biosante Pharmaceuticals, Inc. AAV vector compositions and methods for enhanced expression of immunoglobulins using the same
WO2006012625A2 (en) * 2004-07-22 2006-02-02 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Stat3 decoy oligonucleotides and uses therefor
EP2484780A1 (de) 2004-07-23 2012-08-08 The University of North Carolina At Chapel Hill Verfahren und Materialien zur Bestimmung der Schmerzempfindlichkeit und Vorhersage und Behandlung zugehöriger Störungen
US20060057127A1 (en) * 2004-09-10 2006-03-16 Pocheng Liu Cytokine-expressing cellular vaccines for treatment of prostate cancer
US20060234347A1 (en) * 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
US7632509B2 (en) * 2005-07-19 2009-12-15 Biosante Pharmaceuticals, Inc. Methods to express recombinant proteins from lentiviral vectors
EP2468881A3 (de) 2005-07-21 2012-08-15 Abbott Laboratories Mehrfache Genexpression mit sORF-Konstrukten und Verfahren mit Polyproteinen, Pro-Proteinen und Proteolyse
EP1981548A4 (de) * 2006-01-20 2010-03-24 Univ North Carolina Verstärkte produktion von infektiösen parvovirus-vektoren in insektenzellen
WO2007123737A2 (en) 2006-03-30 2007-11-01 University Of California Methods and compositions for localized secretion of anti-ctla-4 antibodies
EP2007795B1 (de) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav-capsid-proteine
US7919079B2 (en) * 2006-03-31 2011-04-05 Biosante Pharmaceuticals, Inc. Cancer immunotherapy compositions and methods of use
US20070292922A1 (en) * 2006-03-31 2007-12-20 Cell Genesys, Inc. Regulated expression of recombinant proteins from adeno-associated viral vectors
EP2037892B1 (de) * 2006-06-19 2015-03-18 Asklepios Biopharmaceutical, Inc. Modifizierte fatkor-viii- und faktor-ix-gene und vektoren für gentherapie
US20090028857A1 (en) 2007-07-23 2009-01-29 Cell Genesys, Inc. Pd-1 antibodies in combination with a cytokine-secreting cell and methods of use thereof
US8425897B2 (en) 2007-08-30 2013-04-23 Immutep S.A. Compositions containing LAG-3 and cells that secrete GM-CSF and methods of use
CN101977632A (zh) * 2008-02-19 2011-02-16 塞拉东公司 用于增强病毒载体于心肌中的摄取的组合物
US8840881B2 (en) 2008-08-28 2014-09-23 Aduro Gvax Inc. Methods and compositions for treating prostate cancer or inducing a humoral immune response against prostate cancer
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
EP2493923A1 (de) 2009-10-30 2012-09-05 Abbott Laboratories Sorf-konstrukte und multiple genexpression
WO2011084694A1 (en) 2009-12-17 2011-07-14 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Stabilized stat3 decoy oligonucleotides and uses therefor
AU2011238708B2 (en) 2010-03-29 2016-02-11 The Trustees Of The University Of Pennsylvania Pharmacologically Induced Transgene Ablation system
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
BR112014018732A8 (pt) 2012-02-01 2017-07-11 Univ Arizona Composições imunogênicas contra campylobacter e usos das mesmas
TWI702955B (zh) 2012-05-15 2020-09-01 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ）
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
DE102013004595A1 (de) 2013-03-15 2014-09-18 Emergent Product Development Germany Gmbh RSV-Impfstoffe
WO2014201252A2 (en) * 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
US20160251680A1 (en) 2013-11-05 2016-09-01 Clontech Laboratories, Inc. Dry transfection compositions and methods for making and using the same
GB201322626D0 (en) 2013-12-19 2014-02-05 Immutep S A Combined preparations for the treatment of cancer
KR102390075B1 (ko) 2014-03-09 2022-04-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 오르니틴 트랜스카르바밀라아제（ｏｔｃ） 결핍증의 치료에 유용한 조성물
US10000741B2 (en) 2014-03-17 2018-06-19 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US10780182B2 (en) 2014-04-25 2020-09-22 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
EP3919508A1 (de) 2014-04-25 2021-12-08 The Trustees of The University of Pennsylvania Ldlr-varianten und deren verwendung in zusammensetzungen zur verringerung des cholesterinspiegels
MA44179B1 (fr) 2014-05-13 2020-10-28 Univ Pennsylvania Compositions comprenant un virus adeno-associe exprimant des constructions a double anticorps et leurs utilisations
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
ES2900973T3 (es) 2015-01-07 2022-03-21 UNIV AUTòNOMA DE BARCELONA Constructo genético de vector individual que comprende genes de insulina y glucoquinasa
GB201500374D0 (en) 2015-01-09 2015-02-25 Immutep S A Combined preparations for the treatment of cancer
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
CN107405507B (zh) 2015-03-02 2022-05-03 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
WO2016176191A1 (en) 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
CN108135994B (zh) 2015-05-13 2022-01-25 宾夕法尼亚州大学信托人 Aav-介导的抗-流感抗体的表达及其使用方法
EP3325018A4 (de) 2015-07-22 2019-04-24 Duke University Hochdurchsatz-screening von regulatorischen elementfunktionen mit epigenomeditierungstechnologien
CA2996001A1 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
WO2017040524A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
AU2016328279A1 (en) 2015-09-24 2018-05-10 The University Of North Carolina At Chapel Hill Methods and compositions for reducing metastases
IL296929A (en) 2015-09-24 2022-12-01 Univ Pennsylvania A preparation and method for the treatment of a complement-mediated disease
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
EP4089175A1 (de) 2015-10-13 2022-11-16 Duke University Genom-engineering mit typ-i-crispr-systemen in eukaryotischen zellen
EP3368563A1 (de) 2015-10-28 2018-09-05 The Trustees Of The University Of Pennsylvania Intrathekale verabreichung von adeno-assoziierten viralen vektoren zur gentherapie
EP3400304B1 (de) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Gentherapeutische behandlung der familiären hypercholesterinämie
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
EP3795180A1 (de) 2015-12-14 2021-03-24 The Trustees Of The University Of Pennsylvania Gentherapie für augenerkrankungen
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
ES2908467T3 (es) 2016-04-29 2022-04-29 Adverum Biotechnologies Inc Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante
RU2764920C2 (ru) 2016-07-08 2022-01-24 Зе Трастис Оф Зе Юниверсити Оф Пенсильвания Способы и композиции для лечения нарушений и заболеваний, связанных с rdh12
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
CA3073137A1 (en) 2016-08-19 2018-02-22 Jeffrey S. Bartlett Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus
AU2017345470B2 (en) 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
EP3548065B1 (de) 2016-12-01 2022-11-09 INSERM - Institut National de la Santé et de la Recherche Médicale Pharmazeutische zusammensetzungen zur behandlung von degenerativen netzhauterkrankungen
AU2017371043B2 (en) 2016-12-07 2022-12-15 University Of Florida Research Foundation, Incorporated IL-1Ra cDNAs
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
TW201837170A (zh) 2017-02-28 2018-10-16 賓州大學委員會 新穎ａａｖ媒介的流感疫苗
FI3589730T3 (fi) 2017-02-28 2024-02-22 Univ Pennsylvania Kladin f adenoassosioitunut virus (aav) -vektori ja sen käyttötapoja
BR112019017327A2 (pt) 2017-03-01 2020-04-14 Univ Pennsylvania terapia gênica para distúrbios oculares
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법
CA3058970A1 (en) 2017-04-18 2018-10-25 Glaxosmithkline Intellectual Property Development Limited Methods for adeno-associated viral vector production
GB201706090D0 (en) * 2017-04-18 2017-05-31 Glaxosmithkline Ip Dev Ltd Methods for adeno-associated viral vector production
EP3634986A4 (de) 2017-04-24 2021-09-08 The Trustees of The University of Pennsylvania Gentherapie für augenerkrankungen
KR20200023280A (ko) 2017-05-11 2020-03-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 신경 세로이드 지질갈색소증에 대한 유전자 요법
JP2020530977A (ja) 2017-05-24 2020-11-05 ウニベルシダッド アウトノマ デ バルセロナ 線維芽細胞増殖因子２１（ｆｇｆ２１）コーディング配列を含むウイルス発現コンストラクト
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
JP2020527335A (ja) 2017-06-14 2020-09-10 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 眼疾患のための遺伝子療法
JP2021509253A (ja) 2017-10-10 2021-03-25 ナントバイオ，インコーポレイテッド ウイルス生成ペイロードに対して低い毒性を有する改変ｅｃ７細胞
CA3086045A1 (en) 2017-12-19 2019-06-27 Akouos, Inc. Aav-mediated delivery of therapeutic antibodies to the inner ear
GB201800903D0 (en) 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
PE20211596A1 (es) 2018-10-01 2021-08-18 Univ Pennsylvania Composiciones utiles para tratar la gangliosidosis gm1
BR112021010038A2 (pt) 2018-11-26 2021-08-24 Universitat Autonoma De Barcelona Terapia gênica com fator de crescimento de fibroblastos 21 (fgf21)
JP2022523766A (ja) 2019-02-22 2022-04-26 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 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
WO2020176562A1 (en) 2019-02-26 2020-09-03 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
KR20220016100A (ko) 2019-05-31 2022-02-08 유니버시타트 아우토노마 데 바르셀로나 인슐린 유전자 요법
EP4007814A1 (de) 2019-07-26 2022-06-08 Akouos, Inc. Verfahren zur behandlung von hörverlust unter verwendung eines sekretierten zielproteins
KR20220112283A (ko) 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Hunter 질환 치료용 아데노-연합된 바이러스 벡터
CA3183171A1 (en) 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating slc26a4-associated hearing loss
KR20230017845A (ko) 2020-05-26 2023-02-06 유니버시타트 아우토노마 데 바르셀로나 중추 신경계 장애에 대한 섬유아세포 성장 인자 21(fgf21) 유전자 요법
US20230372539A1 (en) 2020-08-24 2023-11-23 The Trustees Of The University Of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
AR123358A1 (es) 2020-08-26 2022-11-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio adulto asociada a la grn
US20230364206A1 (en) 2020-10-07 2023-11-16 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
GB202016254D0 (en) * 2020-10-13 2020-11-25 Freeline Therapeutics Ltd Plasmid system
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
CA3196499A1 (en) 2020-10-29 2022-05-05 James M. Wilson Aav capsids and compositions containing same
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
WO2022119839A1 (en) 2020-12-01 2022-06-09 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2022146839A1 (en) 2020-12-29 2022-07-07 Akouos, Inc. Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
CA3206590A1 (en) 2021-01-30 2022-08-04 Maria Fatima Bosch Tubert Gene therapy for monogenic diabetes
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
BR112023016983A2 (pt) 2021-02-26 2023-11-07 Takeda Pharmaceuticals Co Vetor de vírus adeno-associado recombinante, método para tratar doença de fabry, composição farmacêutica, célula, e, método de expressão da enzima ¿-gal em uma célula
EP4323520A1 (de) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Zusammensetzungen zur behandlung von spinaler und bulbusarer muskelatrophie (sbma)
JP2024515715A (ja) 2021-04-23 2024-04-10 ユニバーシティ オブ ロチェスター レトロウイルスインテグラーゼ－Ｃａｓ融合タンパク質を使用した指向性非相同ＤＮＡ挿入によるゲノム編集及び治療の方法
EP4089171A1 (de) 2021-05-12 2022-11-16 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Rekombinante tert-codierende virale genome und vektoren
CA3233097A1 (en) 2021-09-30 2023-04-06 Katherine Diane GRIBBLE Compositions and methods for treating kcnq4-associated hearing loss
TW202325845A (zh) 2021-10-02 2023-07-01 賓州大學委員會 新穎aav衣殼及含其之組成物
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023150142A1 (en) 2022-02-02 2023-08-10 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2023187728A1 (en) 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024042485A1 (en) 2022-08-25 2024-02-29 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024052413A1 (en) 2022-09-07 2024-03-14 Universitat Autònoma De Barcelona Beta-hexosaminidase vectors

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) * 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5116742A (en) 1986-12-03 1992-05-26 University Patents, Inc. RNA ribozyme restriction endoribonucleases and methods
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
PT728214E (pt) * 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6027931A (en) 1995-08-03 2000-02-22 Avigen, Inc. High-efficiency AA V helper functions
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
EP1983057A3 (de) 1995-09-08 2009-01-07 Genzyme Corporation Verbesserte AAV-Vektoren für die Gentherapie
US6086913A (en) * 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
US6548286B1 (en) * 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production

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JP4135120B2 (ja) 2008-08-20
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