[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

DK3589730T5 - Klade f adeno-associeret virus (aav) vektor og anvendelse deraf - Google Patents

Klade f adeno-associeret virus (aav) vektor og anvendelse deraf Download PDF

Info

Publication number
DK3589730T5
DK3589730T5 DK18711202.4T DK18711202T DK3589730T5 DK 3589730 T5 DK3589730 T5 DK 3589730T5 DK 18711202 T DK18711202 T DK 18711202T DK 3589730 T5 DK3589730 T5 DK 3589730T5
Authority
DK
Denmark
Prior art keywords
clade
aav
adeno
vector
associated virus
Prior art date
Application number
DK18711202.4T
Other languages
English (en)
Other versions
DK3589730T3 (da
Inventor
James M Wilson
Qiang Wang
April Giles
Kevin Turner
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Application granted granted Critical
Publication of DK3589730T3 publication Critical patent/DK3589730T3/da
Publication of DK3589730T5 publication Critical patent/DK3589730T5/da

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Virology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
DK18711202.4T 2017-02-28 2018-02-27 Klade f adeno-associeret virus (aav) vektor og anvendelse deraf DK3589730T5 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762464748P 2017-02-28 2017-02-28
US201762591002P 2017-11-27 2017-11-27
US201862614002P 2018-01-05 2018-01-05
PCT/US2018/019992 WO2018160582A1 (en) 2017-02-28 2018-02-27 Adeno-associated virus (aav) clade f vector and uses therefor

Publications (2)

Publication Number Publication Date
DK3589730T3 DK3589730T3 (da) 2024-03-04
DK3589730T5 true DK3589730T5 (da) 2024-09-02

Family

ID=61628475

Family Applications (1)

Application Number Title Priority Date Filing Date
DK18711202.4T DK3589730T5 (da) 2017-02-28 2018-02-27 Klade f adeno-associeret virus (aav) vektor og anvendelse deraf

Country Status (28)

Country Link
US (2) US11827906B2 (da)
EP (2) EP4272765A3 (da)
JP (2) JP7455579B2 (da)
KR (1) KR20190132639A (da)
CN (1) CN110741082B (da)
AU (2) AU2018227440B2 (da)
BR (1) BR112019017839A2 (da)
CA (1) CA3053399A1 (da)
CL (1) CL2019002474A1 (da)
CO (1) CO2019009525A2 (da)
DK (1) DK3589730T5 (da)
ES (1) ES2971872T3 (da)
FI (1) FI3589730T3 (da)
HR (1) HRP20240257T1 (da)
HU (1) HUE065885T2 (da)
IL (1) IL268644A (da)
LT (1) LT3589730T (da)
MD (1) MD3589730T2 (da)
MX (1) MX2019010275A (da)
PE (1) PE20191362A1 (da)
PL (1) PL3589730T3 (da)
PT (1) PT3589730T (da)
RS (1) RS65241B1 (da)
SA (1) SA519402565B1 (da)
SG (4) SG10201912973XA (da)
SI (1) SI3589730T1 (da)
WO (1) WO2018160582A1 (da)
ZA (1) ZA201905407B (da)

Families Citing this family (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002361573B2 (en) 2001-11-13 2008-06-12 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
HUE033158T2 (en) 2003-09-30 2017-11-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors containing them, and their use
SG10202107733QA (en) 2015-09-28 2021-09-29 Univ North Carolina Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017123918A1 (en) * 2016-01-15 2017-07-20 American Gene Technologies International Inc. Methods and compositons for the activation of gamma-delta t-cells
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
DK3589730T5 (da) 2017-02-28 2024-09-02 Univ Pennsylvania Klade f adeno-associeret virus (aav) vektor og anvendelse deraf
AU2018265531B2 (en) 2017-05-11 2024-07-11 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
MX2020005663A (es) 2017-11-30 2020-08-20 Univ Pennsylvania Terapia genica para mucopolisacaridosis iii a.
JP7384797B2 (ja) 2017-11-30 2023-11-21 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 ムコ多糖症iiib型のための遺伝子療法
BR112020017278A2 (pt) * 2018-02-27 2020-12-22 The Trustees Of The University Of Pennsylvania Vetores de vírus adenoassociado (aav), vetores de aav tendo desamidação de capsídeos reduzida e usos para os mesmos
JP2021516046A (ja) * 2018-02-27 2021-07-01 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 新規アデノ随伴ウイルス(aav)ベクター、低減されたカプシド脱アミド化を有するaavベクター、およびその使用
AU2019247746B2 (en) 2018-04-03 2024-08-15 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN113438954A (zh) * 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
KR20210126096A (ko) 2019-02-15 2021-10-19 상가모 테라퓨틱스, 인코포레이티드 재조합 aav를 제조하기 위한 조성물 및 방법
TW202045730A (zh) 2019-02-22 2020-12-16 賓州大學委員會 用於治療grn相關的成年發病型神經系統退化症之重組腺相關病毒
AU2020229772A1 (en) 2019-02-26 2021-08-26 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
WO2020219766A1 (en) * 2019-04-24 2020-10-29 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
PE20220930A1 (es) * 2019-05-03 2022-05-31 Univ Pennsylvania Composiciones utiles en el tratamiento de leucodistrofia metacromatica
EP4045525A1 (en) 2019-10-17 2022-08-24 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CA3161367A1 (en) * 2019-12-10 2021-06-17 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
EP4096721A1 (en) 2020-02-02 2022-12-07 The Trustees of The University of Pennsylvania Compositions useful for treating gm1 gangliosidosis
IL298137A (en) 2020-05-12 2023-01-01 Univ Pennsylvania The compositions are useful in the treatment of Krabbe's disease
CA3177407A1 (en) 2020-05-12 2021-11-18 James M. Wilson Compositions for drg-specific reduction of transgene expression
AU2021282257A1 (en) 2020-05-26 2022-12-01 Shape Therapeutics Inc. High throughput engineering of functional AAV capsids
KR20230038503A (ko) 2020-07-13 2023-03-20 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 샤르코-마리-투스 질환의 치료에 유용한 조성물
GB2599212A (en) 2020-07-30 2022-03-30 Shape Therapeutics Inc Stable cell lines for inducible production of rAAV virions
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
MX2023002293A (es) 2020-08-24 2023-05-19 Univ Pennsylvania Vectores virales que codifican fusiones de agonistas del receptor de glp-1 y usos de estos en el tratamiento de enfermedades metabólica.
TW202227633A (zh) 2020-08-26 2022-07-16 賓州大學委員會 用於治療grn相關的成年發病神經退化之重組腺相關病毒
US20230365955A1 (en) 2020-10-09 2023-11-16 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022087153A1 (en) * 2020-10-22 2022-04-28 Scout Bio, Inc. Method of suppressing immune response to vector-delivered therapeutic protein
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
MX2023005113A (es) * 2020-10-29 2023-08-04 Univ Pennsylvania Cápsides de aav y composiciones que las contienen.
US20230390418A1 (en) * 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
CA3200192A1 (en) 2020-12-01 2022-06-09 Justin PERCIVAL Compositions and uses thereof for treatment of angelman syndrome
AU2021390480A1 (en) 2020-12-01 2023-06-22 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
AR125406A1 (es) 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
AU2022274162A1 (en) 2021-05-12 2023-11-30 Fondazione Telethon Vector system
CA3220037A1 (en) * 2021-06-08 2022-12-15 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof
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
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CA3239452A1 (en) 2021-12-01 2023-06-08 Thomas Packard Functional aav capsids for systemic administration
EP4441232A2 (en) 2021-12-01 2024-10-09 Shape Therapeutics Inc. Functional aav capsids for intravitreal administration
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
MX2024009186A (es) 2022-01-25 2024-09-30 Univ Pennsylvania Cápsides de aav para mejorar la transducción cardiaca y desdireccionamiento del hígado.
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
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
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023220695A2 (en) * 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024173802A2 (en) 2023-02-17 2024-08-22 Biogen Ma Inc. Rgd-containing peptides for delivering payloads

Family Cites Families (141)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
NZ261277A (en) 1993-02-05 1997-04-24 Laporte Group Australia Ltd Slag defoaming composition comprising a carbon source; an exothermic material (eg aluminium) and a dense inert granular material (eg calcium silicate)
US6972193B1 (en) 1993-02-12 2005-12-06 Board Of Trustees Of Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
PL310327A1 (en) 1993-02-12 1995-12-11 Univ Leland Stanford Junior Adjustable transcription of target genes and other biological processes
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US6140120A (en) 1993-02-12 2000-10-31 Board Of Trustees Of Leland Stanford Jr. University Regulated transcription of targeted genes and other biological events
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US6150137A (en) 1994-05-27 2000-11-21 Ariad Pharmaceuticals, Inc. Immunosuppressant target proteins
US6476200B1 (en) 1994-06-27 2002-11-05 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic multimerizing agents
ES2245781T3 (es) 1994-08-18 2006-01-16 Ariad Gene Therapeutics, Inc. Nuevos agentes de multimerizacion.
US6150527A (en) 1994-08-18 2000-11-21 Ariad Pharmaceuticals, Inc. Synthetic multimerizing agents
US5786464C1 (en) 1994-09-19 2012-04-24 Gen Hospital Corp Overexpression of mammalian and viral proteins
ATE544776T1 (de) 1994-12-29 2012-02-15 Massachusetts Inst Technology Chimäre dna-bindeproteine
US6326166B1 (en) 1995-12-29 2001-12-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
WO1996041865A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamcycin-based regulation of biological events
US5811524A (en) 1995-06-07 1998-09-22 Idec Pharmaceuticals Corporation Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic use thereof
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
AU2192797A (en) 1996-02-28 1997-09-16 Ariad Gene Therapeutics, Inc. Synthetic derivatives of rapamycin as multimerising agents for chimeric proteins with immunophilin derived domains
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
EP0937082A2 (en) 1996-07-12 1999-08-25 Ariad Pharmaceuticals, Inc. Materials and method for treating or preventing pathogenic fungal infection
EP0972081B1 (en) 1997-04-01 2007-06-13 Solexa Ltd. Method of nucleic acid amplification
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
WO1999010508A1 (en) 1997-08-27 1999-03-04 Ariad Gene Therapeutics, Inc. Chimeric transcriptional activators and compositions and uses related thereto
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
AU9036198A (en) 1997-08-26 1999-03-16 Ariad Gene Therapeutics, Inc. Fusion proteins comprising a dimerization, trimerization or tetramerization domain and an additional heterologous transcription activation, transcription repression, dna binding or ligand binding domain
WO1999036553A2 (en) 1998-01-15 1999-07-22 Ariad Gene Therapeutics, Inc. Regulation of biological events using multimeric chimeric proteins
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
WO1999041258A1 (en) 1998-02-13 1999-08-19 President And Fellows Of Harvard College Novel dimerizing agents, their production and use
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
US6258603B1 (en) 1998-06-17 2001-07-10 Rohm And Haas Company Ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US7109317B1 (en) 1998-11-06 2006-09-19 President And Fellows Of Harvard College FK506-based regulation of biological events
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
US6780639B1 (en) 1999-08-24 2004-08-24 Universite Libre De Bruxelles Antibiotic inducible/repressible genetic construct for gene therapy or gene immunization
ES2219388T3 (es) 1999-08-24 2004-12-01 Ariad Gene Therapeutics, Inc. 28-epi-rapalogos.
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
JP3961737B2 (ja) 2000-02-29 2007-08-22 株式会社小糸製作所 車両用灯具およびその製造方法
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
WO2001070816A2 (en) 2000-03-22 2001-09-27 Rohm And Haas Company Ecdysone receptor-based inducible gene expression system
ES2327609T3 (es) 2000-06-01 2009-11-02 University Of North Carolina At Chapel Hill Procedimientos y compuestos para controlar la liberacion de vectores de parvovirus reconbinantes.
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
EP3470520A2 (en) 2001-02-20 2019-04-17 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
ATE545705T1 (de) 2001-02-20 2012-03-15 Intrexon Corp Neues induzierbares genexpressionssystem auf ecdysonrezeptor/invertebraten-retinoid-x-rezept r-basis
ES2392508T3 (es) 2001-02-20 2012-12-11 Intrexon Corporation Receptores X retinoides quiméricos y su uso en un sistema inducible de expresión génica basado en receptores de ecdisona novedoso
US9249207B2 (en) 2001-02-20 2016-02-02 Intrexon Corporation Substitution mutant receptors and their use in an ecdysone receptor-based inducible gene expression system
WO2002095073A1 (en) 2001-05-18 2002-11-28 Wisconsin Alumni Research Foundation Method for the synthesis of dna sequences
AU2002361573B2 (en) 2001-11-13 2008-06-12 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
JP4810062B2 (ja) 2001-12-17 2011-11-09 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 アデノ随伴ウイルス(aav)血清型8の配列
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
WO2005013090A2 (en) 2003-08-01 2005-02-10 Dna Twopointo Inc. Systems and methods for biopolymer engineering
HUE033158T2 (en) 2003-09-30 2017-11-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors containing them, and their use
US20060246079A1 (en) 2003-11-14 2006-11-02 Morrow Phillip R Neutralizing human antibodies to anthrax toxin
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
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
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
ES2545223T3 (es) 2005-06-23 2015-09-09 Isis Pharmaceuticals, Inc. Composiciones y procedimientos para modular el corte y empalme de SMN2
US8877187B2 (en) 2005-07-25 2014-11-04 Avianax, Llc Therapeutic antibodies for treatment and prophylaxis of transmittable viral diseases
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
JP5268890B2 (ja) * 2006-04-28 2013-08-21 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 Aavの規模適応性の製造方法
SI2029742T1 (sl) 2006-06-07 2016-11-30 Genzyme Corporation Genska terapija amiotrofične lateralne skleroze in drugih motenj hrbtenjače
AU2008218925A1 (en) 2007-02-20 2008-08-28 Anaptysbio, Inc. Somatic hypermutation systems
CN102046654A (zh) 2007-03-13 2011-05-04 胡马斯有限公司 针对甲型流感病毒h5n1株系的抗体
WO2008124934A1 (en) 2007-04-17 2008-10-23 National Research Council Of Canada Constructs for enhancement of gene expression in muscle
WO2008156763A2 (en) 2007-06-15 2008-12-24 The Board Of Trustees Of The Leland Stanford Junior University Human neutralizing monoclonal antibodies to h5n1 influenza a virus
US7863425B2 (en) 2007-09-26 2011-01-04 Cornell University Compositions and methods for inhibiting Yersinia pestis infection
PT2220116E (pt) 2007-11-12 2012-12-07 Theraclone Sciences Inc Composições e métodos para a terapia e o diagnóstico da gripe
RU2010127156A (ru) 2007-12-06 2012-01-20 Дана-Фарбер Кэнсер Инститьют, Инк. (Us) Антитела против вируса гриппа и их применение
ITTO20080204A1 (it) 2008-03-17 2009-09-18 Pomona Biotechnologies Llc Anticorpi monoclonali atti a reagire con una pluralita di sottotipi del virus influenzale a
CA2719201A1 (en) 2008-03-28 2009-10-01 Sea Lane Biotechnologies, Llc. Neutralizing molecules to viral antigens
ITTO20080398A1 (it) 2008-05-27 2009-11-28 Pomona Biotechnologies Llc Anticorpi monoclonali aventi proprieta' di cross-neutralizzazione omosubtipica per virus influenzali di tipo a sottotipo h1
WO2010044921A2 (en) 2008-06-03 2010-04-22 Vaxin Inc. Intranasal administration of receptor-binding ligands or genes encoding such ligands as a therapeutic regimen for mitigating infections caused by respiratory pathogens
CN102216327A (zh) 2008-07-25 2011-10-12 生物医学研究所 中和抗甲型流感病毒抗体及其用途
US8871207B2 (en) 2008-07-25 2014-10-28 Humabs, LLC Neutralizing anti-influenza A virus antibodies and uses thereof
KR101734955B1 (ko) 2008-11-07 2017-05-12 메사추세츠 인스티튜트 오브 테크놀로지 아미노알콜 리피도이드 및 그의 용도
US8211631B2 (en) 2008-12-18 2012-07-03 Wisconsin Alumni Research Foundation In vitro model of spinal muscular atrophy
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
BRPI1012749B1 (pt) 2009-05-11 2022-01-11 Janssen Vaccines & Prevention B.V. Anticorpo monoclonal humano, seu uso e seu método de produção, imunoconjugado, e composição farmacêutica
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
IT1395961B1 (it) 2009-06-01 2012-11-02 Pomona Biotechnologies Llc Anticorpi monoclonali come medicamento per il trattamento terapeutico e/o profilattico delle infezioni da virus influenzale a (h1n1) di origine suina (s-oiv)
EP2442816A4 (en) 2009-06-17 2013-10-02 Isis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATING SMN2 SPLICING IN A SUBJECT
NZ617191A (en) 2009-06-25 2015-06-26 Medimmune Llc Swine influenza hemagglutinin variants
US8598331B2 (en) 2009-09-28 2013-12-03 The University Of British Columbia CLDN5 mini-promoters
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ Pennsylvania Pharmacologically induced transgene ablation system
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
BR112013021494B1 (pt) 2011-02-22 2021-09-08 California Institute Of Technology Vetor viral, polinucleotídeo isolado, uso de um vírus adeno-associado (aav) recombinante e método para produzir o mesmo
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
WO2012145759A2 (en) 2011-04-21 2012-10-26 Arizona Board Of Regents, A Body Corporate Of The State Of Arizona Acting For And On Behalf Of Arizona State University Methods of protein production and compositions thereof
EP2717893B1 (en) 2011-06-08 2019-05-08 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
EP2731967B1 (en) 2011-07-14 2016-10-12 Janssen Vaccines & Prevention B.V. Human binding molecules capable of neutralizing influenza a viruses of phylogenetic group 1 and phylogenetic group 2 and influenza b viruses
US9181328B2 (en) 2012-01-31 2015-11-10 Osaka University Human monoclonal antibodies broadly protective against influenza B virus and methods of using the same
CA2865594C (en) 2012-03-08 2021-07-27 Crucell Holland B.V. Human binding molecules capable of binding to and neutralizing influenza b viruses and uses thereof
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
ES2826203T3 (es) 2012-06-08 2021-05-17 Ethris Gmbh Suministro pulmonar de ARN mensajero
JP6417322B2 (ja) 2012-06-21 2018-11-07 アソシアシオン・アンスティテュ・ドゥ・ミオロジーAssociation Institut De Myologie 遺伝子治療ベクターの広範な遺伝子送達
PL2880053T3 (pl) 2012-08-01 2021-01-11 Ikaika Therapeutics, Llc Łagodzenie uszkodzenia tkanek i włóknienia przez przeciwciało przeciwko ltbp4
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
BR112015027336A2 (pt) 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
US20170183396A1 (en) 2014-02-19 2017-06-29 Jody Berry Ebola monoclonal antibodies
US10746742B2 (en) 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3134115A4 (en) 2014-04-25 2017-10-25 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
JP6741590B2 (ja) 2014-04-25 2020-08-19 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 コレステロールレベルを低下させるためのldlr変異体および組成物中でのそれらの使用
CN106470736B (zh) 2014-05-13 2021-05-28 宾夕法尼亚州大学信托人 包括表达双抗体构建体的aav的组合物及其用途
DK3198018T3 (da) 2014-09-24 2021-03-01 Hope City Vektorvarianter af adenoassocieret virus til højeffektiv genomredigering og fremgangsmåder dertil
SG10201902924RA (en) 2014-10-03 2019-05-30 Massachusetts Inst Technology Antibodies that bind ebola glycoprotein and uses thereof
EP3253411A1 (en) 2015-02-05 2017-12-13 Janssen Vaccines & Prevention B.V. Binding molecules directed against influenza hemagglutinin and uses thereof
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
CN116196410A (zh) 2015-10-28 2023-06-02 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US20180363000A1 (en) 2015-12-14 2018-12-20 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
JP7082050B2 (ja) 2015-12-14 2022-06-07 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 脊髄性筋萎縮症の処置において有用なアデノ-関連ウイルスベクター
US20190002915A1 (en) 2015-12-14 2019-01-03 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
WO2017136500A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
CN110168080B (zh) * 2016-08-15 2024-05-31 建新公司 检测aav的方法
US20190240328A1 (en) 2016-09-24 2019-08-08 The Trustees Of The University Of Pennsylvania Novel humanized anti-ebola antibodies useful in preventing ebola infections
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
DK3589730T5 (da) 2017-02-28 2024-09-02 Univ Pennsylvania Klade f adeno-associeret virus (aav) vektor og anvendelse deraf
UY37620A (es) 2017-02-28 2018-08-31 Univ Pennsylvania Vacunas contra la gripe mediadas por aav novedosas

Also Published As

Publication number Publication date
KR20190132639A (ko) 2019-11-28
JP2023089134A (ja) 2023-06-27
ZA201905407B (en) 2020-11-25
CA3053399A1 (en) 2018-09-07
DK3589730T3 (da) 2024-03-04
PL3589730T3 (pl) 2024-09-02
SG11201907714UA (en) 2019-09-27
BR112019017839A2 (pt) 2020-04-14
WO2018160582A1 (en) 2018-09-07
FI3589730T3 (fi) 2024-02-22
CO2019009525A2 (es) 2019-09-18
EP4272765A2 (en) 2023-11-08
US20200056159A1 (en) 2020-02-20
SA519402565B1 (ar) 2023-12-05
WO2018160582A8 (en) 2019-09-12
EP3589730B1 (en) 2023-12-27
AU2024219391A1 (en) 2024-10-03
JP2020510429A (ja) 2020-04-09
SG10201912973XA (en) 2020-03-30
HRP20240257T1 (hr) 2024-05-24
ES2971872T3 (es) 2024-06-10
US11827906B2 (en) 2023-11-28
RS65241B1 (sr) 2024-03-29
PT3589730T (pt) 2024-02-28
IL268644A (en) 2019-10-31
MX2019010275A (es) 2019-10-15
AU2018227440B2 (en) 2024-06-06
AU2018227440A1 (en) 2019-08-29
EP4272765A3 (en) 2024-02-21
CL2019002474A1 (es) 2019-11-29
SG10201912976WA (en) 2020-02-27
EP3589730A1 (en) 2020-01-08
JP7455579B2 (ja) 2024-03-26
HUE065885T2 (hu) 2024-06-28
SI3589730T1 (sl) 2024-04-30
CN110741082A (zh) 2020-01-31
MD3589730T2 (ro) 2024-04-30
US20240117322A1 (en) 2024-04-11
PE20191362A1 (es) 2019-10-01
CN110741082B (zh) 2024-09-06
SG10201912969XA (en) 2020-02-27
LT3589730T (lt) 2024-03-12

Similar Documents

Publication Publication Date Title
DK3589730T3 (da) Klade f adeno-associeret virus (aav) vektor og anvendelse deraf
DK3684423T3 (da) Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
DK3472183T3 (da) Variant adeno-associerede virus og fremgangsmåder til anvendelse
DK3445773T5 (da) Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
IL282885A (en) AAV viral vectors and their uses
IL263801A (en) Novel adeno-associated virus capsid proteins
DK3442600T5 (da) ADENOASSOCIERET VIRUSVEKTORFREMFØRING AF ß-SARCOGLYCAN OG MIKRORNA-29 OG BEHANDLING AF MUSKELDYSTROFI
DK3408382T3 (da) Onkolytiske virale vektorer og anvendelser deraf
EP3585883A4 (en) PROTEINS OF MODIFIED AAV CAPSIDES AND THEIR USES
DK3380620T3 (da) Optimerede lentivirale transfervektorer og anvendelse heraf
EP3368054A4 (en) REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
IL282053A (en) Constructions of glut1-linked recombinant aav vectors, preparations and kits containing them and their use
IL287214A (en) Adeno-associated virus vector formulations and methods
EP3262162A4 (en) Regulatable expression using adeno-associated virus (aav)
DK3347472T3 (da) Sammensætninger som indeholder cyp76ad1- clade polypeptider og anvendelse heraf.
DK3265571T3 (da) Fler-vektorsystem og anvendelse heraf
DK3108000T3 (da) Adeno-associeret virusvektor
MA42934A (fr) Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci
DK3253876T3 (da) Vektor, der co-ekspresserer vaccine og costimulerende molekyler
DK3408398T3 (da) Rekombinante adenovirus-vektoriserede fmdv-vacciner og anvendelser deraf
SG11201900439XA (en) Recombinant vaccinia virus and use thereof
IL253103B (en) Viral vectors associated with adapted g6pc-encoded adeno and uses thereof
DK3743106T3 (da) Influenzavirusvacciner og anvendelser deraf
SG11201704919SA (en) Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav)
DK3710590T3 (da) Virale vektorer omfattende rdh12-kodende områder og fremgangsmåder til behandling af nethindedystrofier