MX2022004352A - Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c. - Google Patents

Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.

Info

Publication number
MX2022004352A
MX2022004352A MX2022004352A MX2022004352A MX2022004352A MX 2022004352 A MX2022004352 A MX 2022004352A MX 2022004352 A MX2022004352 A MX 2022004352A MX 2022004352 A MX2022004352 A MX 2022004352A MX 2022004352 A MX2022004352 A MX 2022004352A
Authority
MX
Mexico
Prior art keywords
adeno
niemann
treatment
viral vectors
disease type
Prior art date
Application number
MX2022004352A
Other languages
English (en)
Inventor
Daniel Mccoy
David Dismuke
Garrett Berry
Original Assignee
Stridebio Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Stridebio Inc filed Critical Stridebio Inc
Publication of MX2022004352A publication Critical patent/MX2022004352A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • 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
    • 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
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14044—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • C12N2750/14122—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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención proporciona composiciones para terapia génica y métodos para tratar, prevenir y/o curar NPC1. Más específicamente, la invención proporciona vectores de virus adenoasociados (AAV) para la entrega de ácidos nucleicos y ácidos nucleicos (incluidos casetes de transferencia de AAV) para tratar, prevenir y/o curar NPC1.
MX2022004352A 2019-10-17 2020-10-16 Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c. MX2022004352A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962916749P 2019-10-17 2019-10-17
US201962923253P 2019-10-18 2019-10-18
US202063082425P 2020-09-23 2020-09-23
US202063082899P 2020-09-24 2020-09-24
PCT/US2020/056031 WO2021076925A1 (en) 2019-10-17 2020-10-16 Adeno-associated viral vectors for treatment of niemann-pick disease type c

Publications (1)

Publication Number Publication Date
MX2022004352A true MX2022004352A (es) 2022-07-19

Family

ID=73452269

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022004352A MX2022004352A (es) 2019-10-17 2020-10-16 Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.

Country Status (14)

Country Link
US (2) US11905523B2 (es)
EP (1) EP4045525A1 (es)
JP (1) JP2022551739A (es)
KR (1) KR20220083714A (es)
CN (1) CN114787180A (es)
AU (1) AU2020367532A1 (es)
BR (1) BR112022006530A2 (es)
CA (1) CA3157700A1 (es)
CL (1) CL2022000939A1 (es)
CO (1) CO2022004649A2 (es)
IL (1) IL292297A (es)
MX (1) MX2022004352A (es)
TW (1) TW202128736A (es)
WO (1) WO2021076925A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
WO2023230579A2 (en) * 2022-05-25 2023-11-30 Metagenomi, Inc. Supplementation of liver enzyme expression

Family Cites Families (256)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
US4501729A (en) 1982-12-13 1985-02-26 Research Corporation Aerosolized amiloride treatment of retained pulmonary secretions
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
EP0322417A1 (en) 1986-09-08 1989-07-05 Applied Biotechnology, Inc. Empty viral capsid vaccines
US4968603A (en) 1986-12-31 1990-11-06 The Regents Of The University Of California Determination of status in neoplastic disease
EP0442926A1 (en) 1988-11-10 1991-08-28 Imperial Cancer Research Technology Limited Polypeptides
US5916563A (en) 1988-11-14 1999-06-29 United States Of America Parvovirus protein presenting capsids
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
ES2026826A6 (es) 1991-03-26 1992-05-01 Ercros Sa Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados.
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
US5869248A (en) 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5599706A (en) 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
CA2223837C (en) 1995-06-07 2010-05-25 University Of North Carolina At Chapel Hill Aav transduction of myoblasts
ATE354957T1 (de) 1995-12-15 2006-03-15 Intronn Inc Durch trans-spaltung erhaltene therapeutische molekule
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6426198B1 (en) 1997-07-03 2002-07-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Genes for Niemann-Pick type C disease
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
CA2745131C (en) 1998-05-28 2016-08-09 John A. Chiorini Aav5 vector and uses thereof
US6562958B1 (en) 1998-06-09 2003-05-13 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Acinetobacter baumannii for diagnostics and therapeutics
US8299321B2 (en) 1998-06-16 2012-10-30 Monsanto Technology Llc Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20100293663A2 (en) 1998-06-16 2010-11-18 Thomas La Rosa Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
NZ511171A (en) 1998-09-22 2004-02-27 Univ Florida Methods for large-scale production of recombinant AAV vectors
GB9822763D0 (en) 1998-10-20 1998-12-16 Univ Sheffield Immunoglobin variant
AU768729B2 (en) 1998-11-05 2004-01-08 Trustees Of The University Of Pennsylvania, The Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
US6822071B1 (en) 1998-11-12 2004-11-23 The Regents Of The University Of California Polypeptides from Chlamydia pneumoniae and their use in the diagnosis, prevention and treatment of disease
US6387368B1 (en) * 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
EP1887081A2 (en) 1999-02-25 2008-02-13 Ceres Incorporated DNA Sequences
EP1033405A3 (en) 1999-02-25 2001-08-01 Ceres Incorporated Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
US20080229439A1 (en) 1999-05-06 2008-09-18 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20110093981A9 (en) 1999-05-06 2011-04-21 La Rosa Thomas J Nucleic acid molecules and other molecules associated with transcription in plants and uses thereof for plant improvement
US20110214206A1 (en) 1999-05-06 2011-09-01 La Rosa Thomas J Nucleic acid molecules and other molecules associated with plants
US20130326723A1 (en) 1999-05-06 2013-12-05 Thomas J. La Rosa Soy nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20090087878A9 (en) 1999-05-06 2009-04-02 La Rosa Thomas J Nucleic acid molecules associated with plants
US20040031072A1 (en) 1999-05-06 2004-02-12 La Rosa Thomas J. Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
AU2138601A (en) 1999-12-22 2001-07-03 Aventis Pasteur Limited Chlamydia antigens and corresponding DNA fragments and uses thereof
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US20140130203A1 (en) 2000-04-19 2014-05-08 Thomas J. La Rosa Rice nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US20110131679A2 (en) 2000-04-19 2011-06-02 Thomas La Rosa Rice Nucleic Acid Molecules and Other Molecules Associated with Plants and Uses Thereof for Plant Improvement
JP2004520803A (ja) 2000-04-21 2004-07-15 コリクサ コーポレイション 尋常性挫瘡の治療および診断のための組成物および方法
WO2001091803A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7214786B2 (en) 2000-12-14 2007-05-08 Kovalic David K Nucleic acid molecules and other molecules associated with plants and uses thereof for plant improvement
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
AU2002256388A1 (en) 2001-04-30 2002-11-11 Cell Genesys, Inc. Viral-mediated delivery and in vivo expression of polynucleotides encoding anti-angiogenic proteins
AU2002345250A1 (en) 2001-06-22 2003-01-08 Syngenta Participations Ag Plant disease resistance genes
AU2002341541A1 (en) 2001-06-22 2003-03-03 Syngenta Participations Ag Abiotic stress responsive polynucleotides and polypeptides
US6623729B2 (en) 2001-07-09 2003-09-23 Korea Advanced Institute Of Science And Technology Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer
WO2002010210A2 (en) 2001-08-28 2002-02-07 Bayer Cropscience Ag Polypeptides for identifying herbicidally active compounds
WO2003033515A1 (en) 2001-10-15 2003-04-24 Corixa Corporation Compositions and methods for the therapy and diagnosis of acne vulgaris
US7271002B2 (en) 2001-11-09 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Production of adeno-associated virus in insect cells
ATE520707T1 (de) 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
US7071172B2 (en) 2002-04-30 2006-07-04 The University Of North Carolina At Chapel Hill Secretion signal vectors
ATE405295T1 (de) 2002-05-01 2008-09-15 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
JP2005185101A (ja) 2002-05-30 2005-07-14 National Institute Of Agrobiological Sciences 植物の全長ｃＤＮＡおよびその利用
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
CA2510895A1 (en) 2002-12-20 2004-07-15 Applera Corporation Genetic polymorphisms associated with stenosis, methods of detection and uses thereof
PT2270048E (pt) 2002-12-24 2016-02-10 Rinat Neuroscience Corp Anticorpos anti-ngf e métodos de utilização dos mesmos
DK1633767T3 (en) 2003-06-02 2019-03-25 Univ Massachusetts METHODS AND COMPOSITIONS FOR MANAGING THE EFFECT OF RNA SILENCING
ES2862206T3 (es) 2003-06-19 2021-10-07 Genzyme Corp Viriones de AAV con inmunorreactividad disminuida y sus usos
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US7473531B1 (en) 2003-08-08 2009-01-06 Colora Corporation Pancreatic cancer targets and uses thereof
US20060107345A1 (en) 2003-09-30 2006-05-18 Nickolai Alexandrov Sequence-determined DNA fragments and corresponding polypeptides encoded thereby
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
HUE037549T2 (hu) 2004-04-07 2018-09-28 Rinat Neuroscience Corp Eljárások csontrákfájdalom kezelésére idegi növekedési faktor antagonista antitest beadásával
US20060171926A1 (en) 2004-04-30 2006-08-03 Passini Marco A Gene therapy for neurometabolic disorders
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
JP4346526B2 (ja) 2004-08-31 2009-10-21 株式会社東芝 半導体集積回路装置
EP1791432A4 (en) 2004-09-09 2010-07-07 Gen Hospital Corp MODULATION OF PHOSPHATASE ACTIVITY IN CARDIAC CELLS
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
JP4798663B2 (ja) 2005-01-05 2011-10-19 塩野義製薬株式会社 新規血管新生抑制因子
US20140199313A1 (en) 2005-03-02 2014-07-17 Metanomics Gmbh Process for the Production of Fine Chemicals
CA2598792A1 (en) 2005-03-02 2006-09-08 Metanomics Gmbh Process for the production of fine chemicals
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
WO2006119137A1 (en) 2005-04-29 2006-11-09 The University Of North Carolina At Chapel Hill Methods and compositions for regulated expression of nucleic acid at post-transcriptional level
EP2478760A1 (en) 2005-05-10 2012-07-25 Monsanto Technology LLC Genes and uses for plant improvement
US20100095387A1 (en) 2005-07-15 2010-04-15 Martin Smith Methods and reagents for screening new drugs and for treating ion pump associated disorders and diseases
US20090191597A1 (en) 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
BRPI0707590A2 (pt) 2006-02-08 2011-05-10 Genzyme Corp terapia gÊnica para a doenÇa de niemann-pick do tipo a
EP1994043A4 (en) 2006-02-10 2009-05-20 Univ Cincinnati PROTEIN-A PHOSPHATASE INHIBITOR AS REGULATOR OF CARDIAC FUNCTION
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
CN103849629B (zh) 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2014193716A2 (en) 2013-05-21 2014-12-04 University Of Florida Research Foundation, Inc. Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
HUE030719T2 (en) 2007-04-09 2017-05-29 Univ Florida RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use
KR100911624B1 (ko) 2007-05-14 2009-08-12 연세대학교 산학협력단 Ｉｌ－１２ 및 ｉｌ－２３의 효율적인 공동발현 방법
CN103695459A (zh) 2007-09-18 2014-04-02 巴斯夫植物科学有限公司 产量提高的植物
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US10526627B2 (en) 2007-11-30 2020-01-07 Corn Products Development, Inc Method for producing high molecular weight reduced viscosity starch pastes
CA2715397A1 (en) 2008-02-20 2009-08-27 Ceres, Inc. Nucleotide sequences and corresponding polypeptides conferring improved nitrogen use efficiency characteristics in plants
US20090221620A1 (en) 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
MX2010012592A (es) 2008-05-20 2011-05-05 Eos Neuroscience Inc Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas.
JP2010002479A (ja) 2008-06-18 2010-01-07 Crossfor:Kk メガネレンズ用装飾体およびメガネ用装飾体装着具
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US20120252877A1 (en) 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
CA3066596A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
WO2011140279A1 (en) * 2010-05-04 2011-11-10 Wayne State University Aav-mediated subcellular targeting of heterologous rhodopsins in retinal ganglion cells
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
CA2816990A1 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Stabilized step function opsin proteins and methods of using the same
US10415056B2 (en) 2010-11-10 2019-09-17 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
WO2012112578A2 (en) 2011-02-14 2012-08-23 The Children's Hospital Of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
US9598468B2 (en) 2011-05-18 2017-03-21 University Of Florida Research Foundation, Incorporated Polypeptides and vectors for targeting HER2/neu expressing cells and uses thereof
JP2014523884A (ja) 2011-06-24 2014-09-18 セントローズ， エルエルシー 細胞外標的化薬物複合体
US9206425B2 (en) 2011-07-28 2015-12-08 Trustees Of Dartmouth College Methods for treating Niemann-Pick type C disease
US9976157B2 (en) 2011-08-23 2018-05-22 Evogene Ltd. Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
CA2856137A1 (en) 2011-11-22 2013-05-30 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
FI124176B (en) 2011-12-01 2014-04-15 Helsingin Yliopisto Polypeptide, polynucleotide, expression cassette, vector, host cell, plant and uses
US20140350087A9 (en) 2012-03-22 2014-11-27 Halozyme, Inc. Oncovector Nucleic Acid Molecules and Methods of Use
SG11201405545XA (en) 2012-03-27 2014-11-27 Curevac Gmbh Artificial nucleic acid molecules comprising a 5'top utr
RU2683497C2 (ru) 2012-04-18 2019-03-28 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
EP2492347A1 (en) 2012-05-22 2012-08-29 Laboratorios Del. Dr. Esteve, S.A. Methods for the production of vectors
US20150182637A1 (en) 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
WO2014007858A1 (en) 2012-07-06 2014-01-09 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
HUE030799T2 (en) 2012-07-11 2017-06-28 Sangamo Biosciences Inc Methods and preparations for the treatment of lysosomal storage diseases
WO2014045674A1 (ja) 2012-09-19 2014-03-27 国立大学法人東京大学 神経活動依存的プロモータ活性を有するdna、及びこれを含むベクター
US9683268B2 (en) 2012-09-19 2017-06-20 Beth Israel Deaconess Viruses associated with immunodeficiency and enteropathy and methods using same
CN111621507A (zh) 2012-09-28 2020-09-04 北卡罗来纳-查佩尔山大学 靶向少突胶质细胞的aav载体
US9066966B2 (en) 2013-02-01 2015-06-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
EP2954051B1 (en) 2013-02-08 2019-03-27 The Trustees Of The University Of Pennsylvania Modified aav8 capsid for gene transfer for retinal therapies
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US9983200B2 (en) 2013-03-14 2018-05-29 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Compositions and methods for predicting age of onset of a lysosomal storage disease or a disease associated with a lysosomal defect
AU2014227766B2 (en) 2013-03-15 2018-10-04 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
SG10201809075XA (en) 2013-07-22 2018-11-29 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
US20160256571A1 (en) 2013-09-30 2016-09-08 Sanofi Invention
ES2857751T3 (es) 2013-10-11 2021-09-29 Massachusetts Eye & Ear Infirmary Métodos para predecir secuencias de virus ancestrales y usos de los mismos
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
US20170007720A1 (en) 2014-02-21 2017-01-12 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US10746742B2 (en) 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
MX2016014220A (es) 2014-05-02 2017-02-06 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc.
WO2015181823A1 (en) 2014-05-28 2015-12-03 Evogene Ltd. Isolated polynucleotides, polypeptides and methods of using same for increasing abiotic stress tolerance, biomass and yield of plants
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
ES2834556T3 (es) 2014-06-25 2021-06-17 Acuitas Therapeutics Inc Lípidos y formulaciones de nanopartículas lipídicas novedosos para la entrega de ácidos nucleicos
US9818383B2 (en) 2014-07-28 2017-11-14 University Of Connecticut Methods and systems for non-destructive analysis of objects and production of replica objects
ES2856090T3 (es) 2014-09-24 2021-09-27 Hope City Variantes de vectores de virus adenoasociados para la edición genómica de alta eficacia y sus métodos
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
RU2727015C2 (ru) 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Векторы aav, нацеленные на центральную нервную систему
CA2972807C (en) 2015-01-14 2024-01-16 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3632923A1 (en) 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
GB201502305D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
GB201502306D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
WO2016134338A1 (en) 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders
WO2016141078A1 (en) 2015-03-02 2016-09-09 Avalanche Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
WO2016150964A1 (en) 2015-03-23 2016-09-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
WO2016154344A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
EP3280451B8 (en) 2015-04-08 2019-09-25 The United States of America, as represented by the Secretary, Department of Health and Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
CA3019315A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
AU2016259976B2 (en) 2015-05-08 2021-10-14 Children's Medical Research Institute Promoters for expression of heterologous genes
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
JP6805174B2 (ja) 2015-05-12 2020-12-23 アメリカ合衆国 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質
US20180216133A1 (en) 2015-07-17 2018-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
EP3907231A1 (en) 2015-07-19 2021-11-10 Yeda Research and Development Co., Ltd. Selective inhibitors of alpha2-containing isoforms of na,k-atpase and use thereof for reducing intraocular pressure
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
EP3872085B1 (en) 2015-07-30 2023-03-01 Massachusetts Eye & Ear Infirmary Ancestral aav sequences and uses thereof
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
RU2743792C2 (ru) 2015-11-05 2021-02-26 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
BR112018011193A2 (pt) 2015-12-01 2018-11-21 Spark Therapeutics Inc métodos escalonáveis para produzir vetor viral adeno-associado (aav) recombinante em sistema de cultura celular em suspensão isento de soro adequado para uso clínico
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
MX2018007237A (es) 2015-12-14 2018-12-11 Univ North Carolina Chapel Hill Proteinas de cápside modificadas para un suministro mejorado de vectores de parvovirus.
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
EP3417055B1 (en) 2016-02-16 2021-10-13 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
JP7231922B2 (ja) 2016-02-22 2023-03-02 ザ ユニヴァーシティー オブ ノース カロライナ アト チャペル ヒル Ｍｐｓ－ｉ関連失明の治療のためのａａｖ－ｉｄｕａベクター
CA3019423A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Novel aav8 mutant capsids and compositions containing same
US11866462B2 (en) 2016-05-04 2024-01-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
EP3488007B1 (en) 2016-07-21 2023-12-20 Spark Therapeutics, Inc. Scalable high recovery methods for producing high yield recombinant adeno-associated viral (raav) vector and recombinant adeno-associated viral (raav) vectors produced thereby
EP3491008A2 (en) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
EP3500676A4 (en) 2016-08-16 2020-07-01 The University of North Carolina at Chapel Hill METHOD AND COMPOSITIONS FOR TARGETED GENE TRANSFER
EP4248979A3 (en) 2016-09-08 2023-12-20 2seventy bio, Inc. Pd-1 homing endonuclease variants, compositions, and methods of use
WO2018064624A1 (en) 2016-09-29 2018-04-05 University Of Florida Research Foundation, Incorporated Aavrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
WO2018075798A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
EP3558393A4 (en) 2016-12-22 2020-11-11 Oregon Health & Science University ADENO-ASSOCIATED VIRAL VECTORS
AU2018221738B2 (en) 2017-02-15 2024-06-27 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
SG11201907714UA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JP7132934B2 (ja) 2017-03-15 2022-09-07 ザ 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 ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
US20210254056A1 (en) 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
GB201707212D0 (en) * 2017-05-05 2017-06-21 Ucl Business Plc Gene therapy for ciliopathies
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質（ａａｐ）依存性を改変する方法および組成物
BR112019025732A2 (pt) 2017-06-06 2020-06-30 University Of Massachusetts vetores de aav auto-reguladores para expressão segura de mecp2 na síndrome de rett
US20200377906A1 (en) 2017-06-20 2020-12-03 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Codon-optimized human npc1 genes for the treatment of niemann-pick type c1 deficiency and related conditions
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
WO2019025984A1 (en) 2017-07-31 2019-02-07 Reflection Biotechnologies Limited CELLULAR MODELS AND THERAPIES FOR OCULAR DISEASES
BR112020014625A2 (pt) 2018-01-17 2020-12-08 Meiragtx Uk Ii Limited Proteína de capsídeo de raav modificada para terapia gênica
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
WO2019169004A1 (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
EP3759218A4 (en) 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR ANTIBODY AVOIDING VIRAL VECTORS
EP3762500A1 (en) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
WO2019173538A1 (en) 2018-03-06 2019-09-12 University Of Florida Research Foundation, Incorporated Aav chimeras
JP7304878B2 (ja) 2018-03-16 2023-07-07 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル キャプシド改変による組織特異的遺伝子送達の増加
KR20210006358A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 안 조직을 표적으로 하기 위한 바이러스 벡터
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
WO2020106916A1 (en) 2018-11-21 2020-05-28 Stridebio, Inc. Recombinant viral vectors and nucleic acids for producing the same
JP2022516283A (ja) 2019-01-04 2022-02-25 ウルトラジェニックス ファーマシューティカル インコーポレイテッド ウイルソン病を処置するための遺伝子治療構築物
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
BR112021022951A2 (pt) 2019-05-14 2022-01-25 Univ Duke Composições e métodos para o tratamento de doenças mediadas por atpase
CN114585743A (zh) 2019-10-17 2022-06-03 斯特里迪比奥公司 Aav转移盒
IL300728A (en) 2020-08-19 2023-04-01 Sarepta Therapeutics Inc Adeno-associated virus vectors for the treatment of Rett syndrome

Also Published As

Publication number Publication date
IL292297A (en) 2022-06-01
TW202128736A (zh) 2021-08-01
KR20220083714A (ko) 2022-06-20
BR112022006530A2 (pt) 2022-07-05
CL2022000939A1 (es) 2023-01-20
US20210115474A1 (en) 2021-04-22
CO2022004649A2 (es) 2022-04-29
US11905523B2 (en) 2024-02-20
JP2022551739A (ja) 2022-12-13
AU2020367532A1 (en) 2022-05-12
CA3157700A1 (en) 2021-04-22
WO2021076925A1 (en) 2021-04-22
EP4045525A1 (en) 2022-08-24
US20230392163A1 (en) 2023-12-07
CN114787180A (zh) 2022-07-22

Similar Documents

Publication Publication Date Title
MX2022004352A (es) Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
MX2021006646A (es) Vector viral adenoasociado recombinante para el suministro de genes.
EA202191418A1 (ru) Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения
WO2019006418A3 (en) ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
BR112019001887A2 (pt) composições e métodos para o tratamento de doença associada a cep290
WO2020123645A8 (en) Combination therapy for treating muscular dystrophy
MX2021014338A (es) Particulas virales modificadas y usos de estas.
MX2021013364A (es) Composiciones utiles para el tratamiento de la enfermedad de pompe.
MX2019008105A (es) Virus.
EA202090700A1 (ru) Компетентные по репликации аденовирусные векторы
MX2021009696A (es) Vectores de genoterapia para el tratamiento de la enfermedad de danon.
MX2022009982A (es) Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.
CO2022004652A2 (es) Casete de transferencia de aav
MX2021005980A (es) Adenovirus y metodos para usar adenovirus.
CL2021002236A1 (es) Inserción de vectores de virus adeno-asociados de b-sarcoglicano y el tratamiento de distrofia muscular
NZ745489A (en) Gene therapy for the treatment of a retinal degeneration disease
IL287614A (en) Adeno-associated virus (aav) particles modified for gene therapy
IL299380A (en) An improved adenovirus as a vector for gene therapy
MX2022016332A (es) Administracion de vectores de virus adenoasociados para distrofias musculares.
MX2021005435A (es) Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2).
CL2023001650A1 (es) Tratamiento de la enfermedad de danon
MX2021007379A (es) Metodos y composiciones para el tratamiento de enfermedades de almacenamiento de glucogeno.
AR120249A1 (es) Vectores virales adeno-asociados para el tratamiento de la enfermedad de niemann-pick tipo c
WO2021102435A8 (en) Materials and methods for treatment of disorders associated with the ighmbp2 gene
BR112017022621A2 (pt) liberação do gene de smad7 como uma substância terapêutica