PE20191362A1 - Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este - Google Patents

Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este

Info

Publication number
PE20191362A1
PE20191362A1 PE2019001786A PE2019001786A PE20191362A1 PE 20191362 A1 PE20191362 A1 PE 20191362A1 PE 2019001786 A PE2019001786 A PE 2019001786A PE 2019001786 A PE2019001786 A PE 2019001786A PE 20191362 A1 PE20191362 A1 PE 20191362A1
Authority
PE
Peru
Prior art keywords
associated virus
seq
aavhu68
adeno
aav
Prior art date
Application number
PE2019001786A
Other languages
English (en)
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
Publication of PE20191362A1 publication Critical patent/PE20191362A1/es

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    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Microbiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (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)

Abstract

Un vector de virus adenoasociado recombinante (rAAV), que comprende una capside de AAVhu68 producida en un sistema de produccion que comprende una secuencia de nucleotidos de la SEQ ID NO: 1, o una secuencia al menos 75 % identica a esta, que codifica la SEQ ID N0:2. La capside de AAVhu68 comprende subpoblaciones de residuos de asparagina muy desamidados en los pares de asparagina-glicina en la secuencia de aminoacidos de la SEQ ID NO: 2. Tambien se proporcionan composiciones que contienen el rAAV y usos de estas. Ademas, se proporcionan rAAV que tienen una capside de AAV modificada, que comprende al menos una subpoblacion de proteinas vp1 o vp2 con una Val en la posicion de aminoacidos 157, con referencia a la numeracion de vp1 de AAVhu68
PE2019001786A 2017-02-28 2018-02-27 Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este PE20191362A1 (es)

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 (1)

Publication Number Publication Date
PE20191362A1 true PE20191362A1 (es) 2019-10-01

Family

ID=61628475

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2019001786A PE20191362A1 (es) 2017-02-28 2018-02-27 Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este

Country Status (23)

Country Link
US (2) US11827906B2 (es)
EP (2) EP3589730B1 (es)
JP (2) JP7455579B2 (es)
KR (1) KR20190132639A (es)
CN (1) CN110741082A (es)
BR (1) BR112019017839A2 (es)
CA (1) CA3053399A1 (es)
CL (1) CL2019002474A1 (es)
CO (1) CO2019009525A2 (es)
DK (1) DK3589730T3 (es)
FI (1) FI3589730T3 (es)
HR (1) HRP20240257T1 (es)
IL (1) IL268644A (es)
LT (1) LT3589730T (es)
MD (1) MD3589730T2 (es)
MX (1) MX2019010275A (es)
PE (1) PE20191362A1 (es)
PT (1) PT3589730T (es)
RS (1) RS65241B1 (es)
SG (4) SG10201912976WA (es)
SI (1) SI3589730T1 (es)
WO (1) WO2018160582A1 (es)
ZA (1) ZA201905407B (es)

Families Citing this family (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK1310571T3 (da) 2001-11-13 2006-06-19 Univ Pennsylvania Fremgangsmåde til identifikation af ukendte adeno-associerede virussekvenser (AAV-sekvenser) og et kit til fremgangsmåden
ES2411479T3 (es) 2003-09-30 2013-07-05 The Trustees Of The University Of Pennsylvania Clados de virus adeno-asociados (AAV), secuencias, vectores que los contienen, y usos de los mismos
IL257628B2 (en) 2015-09-28 2023-04-01 Univ North Carolina Chapel Hill Methods and compositions for stealth virus antibody vectors
IL279520B2 (en) * 2016-01-15 2023-03-01 American Gene Tech Int Inc Methods and preparations for the activation of gamma-delta t cells
SG10201912976WA (en) 2017-02-28 2020-02-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP7273730B2 (ja) 2017-05-11 2023-05-15 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 神経セロイドリポフスチン症のための遺伝子療法
AU2018375164A1 (en) 2017-11-30 2020-06-04 The Trustees Of The University Of Pennsylvania Gene therapy for Mucopolysaccharidosis IIIA
MX2020005673A (es) 2017-11-30 2020-12-03 Univ Pennsylvania Terapia génica para mucopolisacaridosis iiib.
WO2019168961A1 (en) * 2018-02-27 2019-09-06 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
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
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
MX2021003869A (es) 2018-10-01 2021-09-08 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
US11718834B2 (en) 2019-02-15 2023-08-08 Sangamo Therapeutics, Inc. Compositions and methods for producing recombinant AAV
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
JP2022525848A (ja) 2019-02-26 2022-05-20 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 クラッベ病の治療に有用な組成物
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
WO2020219766A1 (en) * 2019-04-24 2020-10-29 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
EP3962454A4 (en) * 2019-05-03 2023-05-31 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF METACHROMATIC LEUKODYSTROPHY
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for the treatment of Niemann-Pick type c disease
CA3161367A1 (en) * 2019-12-10 2021-06-17 Homology Medicines, Inc. Adeno-associated virus compositions and methods of use thereof
WO2021155337A1 (en) 2020-02-02 2021-08-05 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
MX2022014245A (es) 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2021242909A1 (en) 2020-05-26 2021-12-02 Shape Therapeutics Inc. High throughput engineering of functional aav capsids
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease
GB2599212A (en) 2020-07-30 2022-03-30 Shape Therapeutics Inc Stable cell lines for inducible production of rAAV virions
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.
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
EP4225906A1 (en) 2020-10-09 2023-08-16 The Trustees of The University of Pennsylvania Compositions and methods for treatment of fabry disease
TW202229315A (zh) 2020-10-18 2022-08-01 賓州大學委員會 改良的腺相關病毒(aav)載體及其用途
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2022094255A2 (en) * 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
AU2021369793A1 (en) * 2020-10-29 2023-06-08 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
EP4244209A1 (en) 2020-11-11 2023-09-20 European Molecular Biology Laboratory Modified viral particles for gene therapy
US20240024507A1 (en) 2020-12-01 2024-01-25 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
US20230414785A1 (en) 2020-12-01 2023-12-28 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4323520A1 (en) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
BR112023021971A2 (pt) 2021-04-23 2024-02-20 Univ Pennsylvania Composições com motivos de direcionamento específicos do cérebro e composições contendo os mesmos
EP4337779A1 (en) 2021-05-12 2024-03-20 Fondazione Telethon Vector system
EP4351661A1 (en) * 2021-06-08 2024-04-17 The Trustees of The University of Pennsylvania Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102079A1 (en) 2021-12-01 2023-06-08 Shape Therapeutics Inc. Functional aav capsids for systemic administration
WO2023102078A2 (en) 2021-12-01 2023-06-08 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
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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
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

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
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
JPH08506144A (ja) 1993-02-05 1996-07-02 ラポート グループ オーストラリア リミティド スラグ脱泡複合材料
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
EP1978095A1 (en) 1993-02-12 2008-10-08 The Board Of Trustees Of The Leland Stanford Junior University Chimeric receptor proteins for controlling signal transduction and ligands binding thereto
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
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
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
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
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
ATE299145T1 (de) 1994-08-18 2005-07-15 Ariad Gene Therapeutics Inc Neues multimerisierendes reagenz
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
AU719001B2 (en) 1994-12-29 2000-05-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
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
CA2219080A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamycin-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
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
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
AU731826B2 (en) 1996-02-28 2001-04-05 Ariad Pharmaceuticals, Inc. Synthetic Multimerizing Agents
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
US6258823B1 (en) 1996-07-12 2001-07-10 Ariad Pharmaceuticals, Inc. Materials and method for treating or preventing pathogenic fungal infection
AU6846698A (en) 1997-04-01 1998-10-22 Glaxo Group Limited Method of nucleic acid amplification
ES2224375T3 (es) 1997-04-14 2005-03-01 Cell Genesys, Inc. Metodos para aumentar la eficacia del producto de aav recombinante.
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
EP1017829A2 (en) 1997-08-26 2000-07-12 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
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
EP1003886A1 (en) 1997-08-27 2000-05-31 Ariad Gene Therapeutics, Inc. Chimeric transcriptional activators and compositions and uses related thereto
JP2002508971A (ja) 1998-01-15 2002-03-26 アリアド・ジーン・セラピューティクス・インコーポレーテッド 多量体キメラ蛋白質を使用する生物学的イベントの調節
EP1053241A1 (en) 1998-02-13 2000-11-22 President And Fellows Of Harvard College Novel dimerizing agents, their production and use
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University 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
JP2002538770A (ja) 1998-11-10 2002-11-19 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ウイルスベクターとその製造及び投与の方法
ATE403715T1 (de) 1999-08-09 2008-08-15 Targeted Genetics Corp Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
WO2001014387A1 (en) 1999-08-24 2001-03-01 Ariad Gene Therapeutics, Inc. 28-epirapalogs
US6780639B1 (en) 1999-08-24 2004-08-24 Universite Libre De Bruxelles Antibiotic inducible/repressible genetic construct for gene therapy or gene immunization
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
CA2404253C (en) 2000-03-22 2014-05-13 Rohm And Haas Company Novel ecdysone receptor-based inducible gene expression system
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
JP4955905B2 (ja) 2001-02-20 2012-06-20 イントレキソン コーポレーション キメラレチノイドｘ受容体および新規エクジソン受容体−ベースの誘導性遺伝子発現システムにおけるそれらの使用
US9493540B2 (en) 2001-02-20 2016-11-15 Intrexon Corporation Ecdysone receptor/invertebrate retinoid X receptor-based inducible gene expression system
US8715959B2 (en) 2001-02-20 2014-05-06 Intrexon Corporation Substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
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
EP1392868B2 (en) 2001-05-18 2013-09-04 Wisconsin Alumni Research Foundation Method for the synthesis of dna sequences using photo-labile linkers
DK1310571T3 (da) 2001-11-13 2006-06-19 Univ Pennsylvania Fremgangsmåde til identifikation af ukendte adeno-associerede virussekvenser (AAV-sekvenser) og et kit til fremgangsmåden
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
EP2434420A3 (en) 2003-08-01 2012-07-25 Dna Twopointo Inc. Systems and methods for biopolymer engineering
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
ES2411479T3 (es) 2003-09-30 2013-07-05 The Trustees Of The University Of Pennsylvania Clados de virus adeno-asociados (AAV), secuencias, vectores que los contienen, y usos de los mismos
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
EP3085389A1 (en) 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
EP3470072A1 (en) 2005-06-23 2019-04-17 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing
US8877187B2 (en) 2005-07-25 2014-11-04 Avianax, Llc Therapeutic antibodies for treatment and prophylaxis of transmittable viral diseases
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
CN101528916B (zh) * 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
PL2029742T3 (pl) 2006-06-07 2017-08-31 Genzyme Corporation Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego
US8603950B2 (en) 2007-02-20 2013-12-10 Anaptysbio, Inc. Methods of generating libraries and uses thereof
JP5346820B2 (ja) 2007-03-13 2013-11-20 エイチユーエムエイビーエス・リミテッド・ライアビリティ・カンパニー Ｈ５ｎ１亜型ａ型インフルエンザウィルスに対する抗体
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
WO2009064805A1 (en) 2007-11-12 2009-05-22 Spaltudaq Corporation Compositions and methods for the therapy and diagnosis of influenza
EP3333187B1 (en) 2007-12-06 2022-06-22 Dana-Farber Cancer Institute, Inc. Antibodies against influenza virus and methods of use thereof
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
EP2698380A1 (en) 2008-03-28 2014-02-19 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
EP2296700A2 (en) 2008-06-03 2011-03-23 Vaxin, Inc. Intranasal administration of receptor-binding ligands or genes encoding such ligands as a therapeutic regimen for mitigating infections caused by respiratory pathogens
US8871207B2 (en) 2008-07-25 2014-10-28 Humabs, LLC Neutralizing anti-influenza A virus antibodies and uses thereof
JP5607620B2 (ja) 2008-07-25 2014-10-15 インスティテュート・フォー・リサーチ・イン・バイオメディシン 抗ａ型インフルエンザウイルス中和抗体およびその使用
US8969353B2 (en) 2008-11-07 2015-03-03 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
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
NZ596032A (en) 2009-05-11 2013-09-27 Crucell Holland Bv Human binding molecules capable of neutralizing influenza virus h3n2 and uses thereof
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)
KR20190037378A (ko) 2009-06-17 2019-04-05 바이오젠 엠에이 인코포레이티드 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법
RU2560420C2 (ru) 2009-06-25 2015-08-20 МЕДИММЬЮН, ЭлЭлСи Варианты гемагглютининов вируса свиного гриппа
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
JP5922095B2 (ja) 2010-03-29 2016-05-24 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 薬理学的に誘導される導入遺伝子アブレーション系
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2012115980A1 (en) 2011-02-22 2012-08-30 California Institute Of Technology Delivery of proteins using adeno-associated virus (aav) vectors
US20140031418A1 (en) 2011-04-20 2014-01-30 The Trustees Of The University Of Pennsylvania Regimens and Compositions for AAV-Mediated Passive Immunization of Airborne Pathogens
US20140127749A1 (en) 2011-04-21 2014-05-08 Arizona Borad Of Regents, A Body Corporate Of The State Of Arizona Acting For And On Behalf Of Arizo Methods of protein production and compositions thereof
IL280771B2 (en) 2011-06-08 2024-03-01 Shire Human Genetic Therapies Preparations of lipid nanoparticles and methods for administration of mRNA
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
ES2608321T3 (es) 2011-07-14 2017-04-07 Janssen Vaccines & Prevention B.V. Moléculas de unión humanas capaces de neutralizar virus de la gripe A del grupo filogenético 1 y del grupo filogenético 2 y virus de la gripe B
WO2013114885A1 (en) 2012-01-31 2013-08-08 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
EP3800254A1 (en) 2012-06-08 2021-04-07 Ethris GmbH Pulmonary delivery of messenger rna
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
EP3711771A1 (en) 2012-08-01 2020-09-23 Ikaika Therapeutics, LLC Mitigating tissue damage and fibrosis via latent transforming growth factor beta binding protein (ltbp4)
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
CA2911105A1 (en) 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
WO2015127136A2 (en) 2014-02-19 2015-08-27 Jody Berry Ebola monoclonal antibodies
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson Ldlr variants and their use in compositions for reducing cholesterol levels
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
US10746742B2 (en) * 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
MX365658B (es) 2014-05-13 2019-06-10 Univ Pennsylvania Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos.
BR112017005892A2 (pt) * 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
SG10201902924RA (en) 2014-10-03 2019-05-30 Massachusetts Inst Technology Antibodies that bind ebola glycoprotein and uses thereof
JP6772157B2 (ja) 2015-02-05 2020-10-21 ヤンセン ファッシンズ アンド プリベンション ベーフェーＪａｎｓｓｅｎ Ｖａｃｃｉｎｅｓ ＆ Ｐｒｅｖｅｎｔｉｏｎ Ｂ．Ｖ． インフルエンザヘマグルチニンに対する結合分子及びその使用
EA201792500A1 (ru) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Aav-опосредованная экспрессия антител против гриппа и способы их использования
EP4316512A3 (en) 2015-10-28 2024-04-24 The Trustees of The University of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
WO2017106326A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
US20190002915A1 (en) 2015-12-14 2019-01-03 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
CA3008280A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
BR112018015751A2 (pt) 2016-02-03 2019-02-05 Univ Pennsylvania terapia gênica para tratamento de mucopolissacaridose tipo i
BR112019002934A2 (pt) * 2016-08-15 2019-05-14 Genzyme Corporation métodos para detecção de aav
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
SG10201913833PA (en) 2017-02-28 2020-03-30 Univ Pennsylvania Influenza vaccines based on aav vectors
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SG10201912976WA (en) 2017-02-28 2020-02-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor

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