ATE315637T1 - Aav-capsidvehikel für den molekularen transport - Google Patents

Aav-capsidvehikel für den molekularen transport

Info

Publication number
ATE315637T1
ATE315637T1 AT95924751T AT95924751T ATE315637T1 AT E315637 T1 ATE315637 T1 AT E315637T1 AT 95924751 T AT95924751 T AT 95924751T AT 95924751 T AT95924751 T AT 95924751T AT E315637 T1 ATE315637 T1 AT E315637T1
Authority
AT
Austria
Prior art keywords
aav
production
capsids
aav capsid
vitro
Prior art date
Application number
AT95924751T
Other languages
English (en)
Inventor
Richard J Samulski
Forrest K Ferrari
Original Assignee
Univ Pittsburgh
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 Pittsburgh filed Critical Univ Pittsburgh
Application granted granted Critical
Publication of ATE315637T1 publication Critical patent/ATE315637T1/de

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10344—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
    • C12N2750/14123—Virus like particles [VLP]
    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00—Nanotechnology
    • Y10S977/70—Nanostructure
    • Y10S977/788—Of specified organic or carbon-based composition
    • Y10S977/802—Virus-based particle
    • Y10S977/803—Containing biological material in its interior
    • Y10S977/804—Containing nucleic acid
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00—Nanotechnology
    • Y10S977/84—Manufacture, treatment, or detection of nanostructure
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00—Nanotechnology
    • Y10S977/902—Specified use of nanostructure
    • Y10S977/904—Specified use of nanostructure for medical, immunological, body treatment, or diagnosis
    • Y10S977/918—Immunological
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S977/00—Nanotechnology
    • Y10S977/902—Specified use of nanostructure
    • Y10S977/904—Specified use of nanostructure for medical, immunological, body treatment, or diagnosis
    • Y10S977/92—Detection of biochemical

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Obesity (AREA)
  • Emergency Medicine (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Addition Polymer Or Copolymer, Post-Treatments, Or Chemical Modifications (AREA)
AT95924751T 1994-06-30 1995-06-28 Aav-capsidvehikel für den molekularen transport ATE315637T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US08/268,430 US6204059B1 (en) 1994-06-30 1994-06-30 AAV capsid vehicles for molecular transfer

Publications (1)

Publication Number Publication Date
ATE315637T1 true ATE315637T1 (de) 2006-02-15

Family

ID=23022972

Family Applications (1)

Application Number Title Priority Date Filing Date
AT95924751T ATE315637T1 (de) 1994-06-30 1995-06-28 Aav-capsidvehikel für den molekularen transport

Country Status (8)

Country Link
US (3) US6204059B1 (de)
EP (1) EP0766569B1 (de)
JP (1) JPH10502526A (de)
AT (1) ATE315637T1 (de)
AU (1) AU705564B2 (de)
CA (1) CA2193802A1 (de)
DE (1) DE69534746T2 (de)
WO (1) WO1996000587A1 (de)

Families Citing this family (440)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
IL115880A (en) * 1995-11-06 2008-11-26 Hadasit Med Res Service Construct for infecting mammalian cells comprising the sv40 vp1 capsid protein and a histone - free constituent, methods of construction and uses thereof
ES2283015T3 (es) * 1996-04-19 2007-10-16 Merial Ltd. Vacunacion con acidos nucleicos para infecciones parvoviricas.
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
AU771545B2 (en) * 1996-09-11 2004-03-25 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
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6541258B2 (en) 1996-12-18 2003-04-01 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
PT1071806E (pt) * 1998-04-24 2004-09-30 Univ Florida Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica
GB9811171D0 (en) * 1998-05-22 1998-07-22 Royal Free Hosp School Med Viral vector
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
AU762220B2 (en) 1998-05-28 2003-06-19 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The AAV5 vector and uses thereof
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
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
ATE454445T1 (de) * 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US20040043488A1 (en) * 1999-02-17 2004-03-04 University Of Pittsburgh Of The Commonwealth System Of Higher Education Adeno-associated viral gene-transfer vector system
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
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
DE19933719A1 (de) * 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
AU2001239884B2 (en) * 2000-02-25 2006-08-10 Vegenics Limited Materials and methods involving hybrid vascular endothelial growth factor DNAs and proteins and screening methods for modulators
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
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US6368602B1 (en) * 2000-06-16 2002-04-09 Hadasit Medical Research Services And Development Ltd Mucosal immunization against hepatitis A virus (HAV) through rectal administration of HAV vaccine
FR2813085A1 (fr) * 2000-08-18 2002-02-22 Aventis Pharma Sa Systeme de regulation in vivo de l'expression d'un transgene par inhibition conditionnelle
IL154508A0 (en) * 2000-08-18 2003-09-17 Gencell Sa System for regulating in vivo the expression of a transgene by conditional inhibition
AU2002360355B2 (en) * 2001-11-09 2005-07-07 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
US20060292117A1 (en) * 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
US8802080B2 (en) * 2002-05-01 2014-08-12 University Of Florida Research Foundation, Inc. Raav expression systems for genetic modification of specific capsid proteins
US20060093589A1 (en) * 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
EP1670415A4 (de) * 2003-09-12 2007-12-05 Vertex Pharma Tiermodell für die proteaseaktivität und leberschäden
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
ATE507240T1 (de) * 2004-03-05 2011-05-15 Vegenics Pty Ltd Materialien und verfahren für wachstumsfaktorbindende konstrukte
US7319015B2 (en) * 2004-03-16 2008-01-15 The Regents Of The University Of Michigan Methods and compositions for using alveolar macrophage phospholipase A2
US7582442B2 (en) 2004-03-16 2009-09-01 The Regents Of The University Of Michigan Methods and compositions for using aleveolar macrophage phospholipase A2
EP1773303A2 (de) * 2004-05-25 2007-04-18 Chimeracore, Inc. Selbstmontierendes nanopartikel-wirkstofffreisetzungssystem
AU2005287393A1 (en) * 2004-06-18 2006-03-30 The University Of Montana AAV mediated gene delivery to cochlear cells
WO2006014798A2 (en) * 2004-07-27 2006-02-09 Mount Sinai School Of Medicine Methods and compositions for using sax2
FI20050753A7 (fi) 2004-09-03 2006-03-04 Licentia Oy Uudet peptidit
AU2005316476A1 (en) * 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
WO2006101629A2 (en) * 2005-02-17 2006-09-28 Vertex Pharmaceuticals Incorporated SODIUM CHANNEL PROTEIN TYPE III α-SUBUNIT SPLICE VARIANT
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US7972813B2 (en) * 2005-09-30 2011-07-05 Vertex Pharmaceuticals Incorporated Tetrodotoxin-resistant sodium channel alpha subunit
US20080200408A1 (en) * 2005-09-30 2008-08-21 Mccormack Kenneth Deletion mutants of tetrodotoxin-resistant sodium channel alpha subunit
AU2005339101B2 (en) 2005-12-14 2013-01-10 Herantis Pharma Plc. Novel neurotrophic factor protein and uses thereof
EP1981548A4 (de) * 2006-01-20 2010-03-24 Univ North Carolina Verstärkte produktion von infektiösen parvovirus-vektoren in insektenzellen
US20090214496A1 (en) * 2006-01-30 2009-08-27 Licentia Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
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
ATE460922T1 (de) * 2006-04-07 2010-04-15 Chimeros Inc Zusammensetzungen und verfahren zur behandlung von b-zellen-malignomen
DK2548578T3 (da) 2006-05-17 2014-10-06 Ludwig Inst Cancer Res Målretning af VEGF-B-regulering af fedtsyretransportører til modulering af humane sygdomme
CN101511373B (zh) 2006-06-19 2015-12-09 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子ⅷ和因子ⅸ基因和载体
EP2061891B1 (de) 2006-08-24 2012-04-11 Virovek, Inc. Expression von genen mit überlappenden offenen leserastern in insektenzellen, verfahren und zusammensetzungen dafür
CA2683063A1 (en) * 2007-04-09 2008-10-16 Chimeros, Inc. Self-assembling nanoparticle drug delivery system
EP2012122A1 (de) * 2007-07-06 2009-01-07 Medigene AG Strukturproteine von mutierten Parvoviren als Impfstoffe
FI20070808A0 (fi) 2007-10-25 2007-10-25 Mart Saarma GDNF:n silmukointivariantit ja niiden käytöt
US20090196854A1 (en) * 2008-02-04 2009-08-06 Kytos Biosystems S.A. Methods and compositions for use of crl 5803 cells for expression of biotherapeutics and encapsulated cell-based delivery
FI20080326A0 (fi) 2008-04-30 2008-04-30 Licentia Oy Neurotroofinen tekijä MANF ja sen käytöt
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP2318036B1 (de) 2008-06-30 2015-06-03 The Regents of the University of Michigan Aktivität von lysosomaler phospholipase a2 (lpla2) als diagnostisches und therapeutisches target zur identifizierung und behandlung von systemischem lupus erythematodes
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US9096659B2 (en) 2009-03-18 2015-08-04 Wake Forest University Health Sciences Flagellin fusion proteins and use thereof to induce immune responses against Pseudomonas aeruginosa
WO2010120874A2 (en) 2009-04-14 2010-10-21 Chimeros, Inc. Chimeric therapeutics, compositions, and methods for using same
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2010141312A2 (en) 2009-06-01 2010-12-09 Wake Forest University Health Sciences Flagellin fusion proteins and conjugates comprising pneumococcus antigens and methods of using the same
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
KR20130040844A (ko) 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
EP3567106A1 (de) 2010-04-23 2019-11-13 University of Massachusetts Auf das zns abzielende aav-vektoren und verfahren zur verwendung davon
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
EP3219313B1 (de) 2011-04-18 2020-10-28 National Center of Neurology and Psychiatry Arzneimittelabgabepartikel und verfahren zur herstellung davon
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
WO2012158989A2 (en) 2011-05-19 2012-11-22 The Regents Of The University Of Michigan Integrin alpha-2 binding agents and use thereof to inhibit cancer cell proliferation
JP2014527072A (ja) 2011-09-09 2014-10-09 バイオメド リアルティー， エル．ピー． ウイルスタンパク質の集合を制御するための方法および組成物
US9365496B2 (en) 2011-11-30 2016-06-14 Ludwig Institute For Cancer Research iNKT cell modulators and methods of using the same
GB201120860D0 (en) 2011-12-05 2012-01-18 Cambridge Entpr Ltd Cancer immunotherapy
JP5998229B2 (ja) 2011-12-08 2016-09-28 ビロベック・インコーポレイテッドＶｉｒｏｖｅｋ，Ｉｎｃ． 毒性遺伝子を保有するベクター、そのための方法および使用
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
SMT202000445T1 (it) 2012-06-19 2020-09-10 Univ Florida Composizioni e metodi per trattare patologie
EP2692868A1 (de) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
EP2892617B1 (de) 2012-09-06 2018-06-13 The University of Chicago Antisense-polynukleotide zur induktion von exon-skipping sowie verfahren zur behandlung von muskeldystrophien
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
MY199344A (en) 2013-02-18 2023-10-24 Vegenics Pty Ltd Ligand binding molecules and uses thereof
EP2968612B1 (de) 2013-03-14 2018-02-14 Asklepios Biopharmaceutical, Inc. Modifizierte lösliche vegf-rezeptor-1-gene und vektoren zur gentherapie
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
EP3517612A1 (de) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetische adenoassoziierte virusinvertierte endwiederholungssequenzen
WO2014191630A2 (en) 2013-05-28 2014-12-04 Helsingin Yliopisto Non-human animal model encoding a non-functional manf gene
EP3770169A1 (de) 2013-09-12 2021-01-27 BioMarin Pharmaceutical Inc. Adeno-assoziierte virusfaktor-viii-vektoren
NZ718926A (en) 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
JP6822841B2 (ja) 2014-03-09 2021-01-27 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 オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物
CA3276269A1 (en) 2014-03-18 2026-03-02 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson Ldlr variants and their use in compositions for reducing cholesterol levels
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
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
MY186389A (en) 2014-05-13 2021-07-22 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016041588A1 (en) 2014-09-16 2016-03-24 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (de) 2014-10-03 2020-09-09 University of Massachusetts Hocheffiziente bibliotheksidentifizierte aav-vektoren
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP4344741A3 (de) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Auf das zentrale nervensystem gerichtete aav-vektoren
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3242945B1 (de) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Einzelvektor-genkonstrukt mit insulin- und glucokinasegenen
AU2016206624B2 (en) 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2016127057A1 (en) 2015-02-06 2016-08-11 The University Of North Carolina At Chapel Hill Optimized human clotting factor viii gene expression cassettes and their use
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2016131009A1 (en) * 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
PT3283500T (pt) 2015-04-08 2021-01-28 Univ Chicago Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
JP6851319B2 (ja) 2015-04-27 2021-03-31 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 ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
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
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
US12221615B2 (en) 2015-08-06 2025-02-11 The Trustees Of The University Of Pennsylvania GLP-1 and use thereof in compositions for treating metabolic diseases
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
CA2995733A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
EP3359663A4 (de) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania Zusammensetzung und verfahren zur behandlung einer komplementvermittelten erkrankung
PE20181338A1 (es) 2015-09-24 2018-08-21 Biomarin Pharm Inc Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
EP4089175A1 (de) 2015-10-13 2022-11-16 Duke University Genom-engineering mit typ-i-crispr-systemen in eukaryotischen zellen
WO2017068077A1 (en) 2015-10-20 2017-04-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods and products for genetic engineering
EP4636083A3 (de) 2015-10-20 2026-01-21 Institut National de la Santé et de la Recherche Médicale (INSERM) Verfahren und produkte für gentechnologie
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
EP3364996B1 (de) 2015-10-22 2021-08-25 University of Massachusetts Gegen die prostata gerichtete adeno-assoziierte virusserotypvektoren
JP2019501115A (ja) 2015-10-28 2019-01-17 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 遺伝子治療のためのアデノ随伴ウイルスベクターのくも膜下腔内投与
EP3368054A4 (de) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Regulierbare expression unter verwendung des adeno-assoziierten virus (aav)
PE20181156A1 (es) 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica
RU2761264C2 (ru) 2015-11-12 2021-12-06 Рисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал Способы лечения мышечной дистрофии
EP3377042A4 (de) 2015-11-16 2019-05-29 The Research Institute at Nationwide Children's Hospital Materialien und verfahren zur behandlung von titinbasierten myopathien und anderen titinopathien
CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
AU2016370590B2 (en) 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
WO2017106202A2 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
BR112018011881A2 (pt) 2015-12-14 2018-12-04 The University Of North Carolina At Chapel Hill proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017165859A1 (en) * 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
EP3443081A4 (de) 2016-04-13 2019-10-30 Duke University Crispr/cas9-basierte repressoren zur in-vivo-ausschaltung von gen-targets und verfahren zur verwendung
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017190031A1 (en) 2016-04-28 2017-11-02 Indiana University Research And Technology Corporation Methods and compositions for resolving components of a virus preparation
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
KR102799528B1 (ko) 2016-07-08 2025-04-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
EP4275747A3 (de) 2016-07-19 2024-01-24 Duke University Therapeutische anwendungen von cpf1-basierter genomeditierung
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
AU2017301600B2 (en) 2016-07-26 2022-08-04 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
SG10201913002QA (en) 2016-08-15 2020-03-30 Genzyme Corp Methods for detecting aav
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
EP3548065B1 (de) 2016-12-01 2022-11-09 INSERM - Institut National de la Santé et de la Recherche Médicale Pharmazeutische zusammensetzungen zur behandlung von degenerativen netzhauterkrankungen
KR20190118163A (ko) 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
MX2019010275A (es) 2017-02-28 2019-10-15 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este.
BR112019017697A2 (pt) 2017-02-28 2020-04-07 The Trustees Of The University Of Pennsylvania vacinas contra influenza baseadas em vetores de vírus adenoassociado (aav)
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
KR20230093072A (ko) 2017-03-01 2023-06-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 안구 장애에 대한 유전자 치료
IL316259A (en) 2017-03-15 2024-12-01 Univ North Carolina Chapel Hill Polyploid gland associated with virus vectors and methods for preparing and using the same.
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
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
EP3622073A4 (de) 2017-05-09 2021-01-06 University of Massachusetts Verfahren zur behandlung von amyotropher lateralsklerose (als)
US20210189432A1 (en) * 2017-05-10 2021-06-24 University Of Utah Research Foundation Compositions and methods of use of arc capsids
AU2018265414B2 (en) 2017-05-10 2022-01-20 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (APP)-dependence of viruses
BR112019023303A2 (pt) 2017-05-11 2020-06-16 The Trustees Of The University Of Pennsylvania Terapia de gene para lipofuscinoses ceroides neuronais
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (ｆｇｆ21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
CN110913886A (zh) 2017-05-24 2020-03-24 巴塞罗那自治大学 包含成纤维细胞生长因子21(fgf21)编码序列的病毒表达构建体
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
AU2018280116B2 (en) 2017-06-05 2025-03-27 Research Institute At Nationwide Children's Hospital Enhanced modified viral capsid proteins
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
US20200297871A1 (en) 2017-09-15 2020-09-24 King's College London Compositions and methods for enhancing gamma delta t cells in the gut
US11668719B2 (en) 2017-09-20 2023-06-06 The Trustees Of Indiana University Methods for resolving lipoproteins with mass spectrometry
US11739330B2 (en) 2017-09-22 2023-08-29 University Of Massachusetts SOD1 dual expression vectors and uses thereof
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (de) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose (als)
US12043848B2 (en) 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
CN111819281B (zh) * 2017-10-23 2024-12-31 普利维尔治疗公司 用于神经变性疾病的基因疗法
BR112020012336A2 (pt) 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
US11232941B2 (en) 2018-01-12 2022-01-25 The Trustees Of Indiana University Electrostatic linear ion trap design for charge detection mass spectrometry
AR114350A1 (es) 2018-01-31 2020-08-26 Res Institute At Nationwide Children’S Hospital Terapia genética para la distrofia muscular de cinturas del tipo 2c
AU2019228504B2 (en) 2018-02-27 2025-06-26 The Trustees Of The University Of Pennsylvania Novel adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor
CA3092107A1 (en) 2018-02-28 2019-09-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
WO2019195444A1 (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
IT201800004253A1 (it) 2018-04-05 2019-10-05 Composizioni e metodi per il trattamento della tachicardia ventricolare polimorfa catecolaminergica dominante ereditaria.
US12129476B2 (en) 2018-04-27 2024-10-29 Voyager Therapeutics, Inc. Methods for measuring the potency of AADC viral vectors
CA3099704A1 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
JP2021523198A (ja) 2018-05-14 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド 幼若対象におけるａａｖベクターの安定的発現
EP3794126A1 (de) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von morbus parkinson
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3793615A2 (de) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Gerichtete entwicklung von aav zur verbesserung des tropismus für zns
EP3793686A1 (de) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Aav-serotypen zur gehirnspezifischen payload-übertragung
WO2019236142A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Ion trap array for high throughput charge detection mass spectrometry
WO2019236139A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Interface for transporting ions from an atmospheric pressure environment to a low pressure environment
EP4376051A3 (de) 2018-06-04 2024-09-25 The Trustees of Indiana University Ladungsdetektionsmassenspektrometrie mit echtzeitanalyse und signaloptimierung
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
WO2019236141A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for capturing ions in an electrostatic linear ion trap
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
MX2020013450A (es) 2018-06-11 2021-08-11 Univ Florida Materiales y métodos para tratar el cancer y los trastornos relacionados con el estrés.
US12410441B2 (en) 2018-06-12 2025-09-09 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
WO2020006458A1 (en) 2018-06-29 2020-01-02 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
CA3103963A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
JP2021530548A (ja) 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 遺伝子治療製剤を生産するための系および方法
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
BR112021006060A2 (pt) 2018-10-01 2021-07-20 The Trustees Of The University Of Pennsylvania composições úteis para tratamento de gangliosidose gm1
EP3861010A1 (de) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Umlenkung des tropismus von aav-kapsiden
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
JP2022512621A (ja) 2018-10-05 2022-02-07 ボイジャー セラピューティクス インコーポレイテッド Ａａｖ産生タンパク質をコードする操作された核酸コンストラクト
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3867389A1 (de) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expressionsvektoren zur massenproduktion von raav im baculovirus/sf9-system
EP3884510A1 (de) 2018-11-20 2021-09-29 The Trustees of Indiana University Orbitrap für einzelteilchen-massenspektrometrie
MX2021005824A (es) 2018-11-26 2021-10-13 UNIV AUTòNOMA DE BARCELONA Genoterapia con factor 21 de crecimiento de fibroblastos (fgf21).
US11562896B2 (en) 2018-12-03 2023-01-24 The Trustees Of Indiana University Apparatus and method for simultaneously analyzing multiple ions with an electrostatic linear ion trap
MX2021008542A (es) 2019-01-18 2021-11-12 Voyager Therapeutics Inc Métodos y sistemas para la producción de partículas de aav.
MX2021010134A (es) 2019-02-22 2021-09-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
SG11202108480UA (en) 2019-02-26 2021-09-29 Univ Pennsylvania Compositions useful in treatment of krabbe disease
CA3131390A1 (en) 2019-02-26 2020-09-03 Research Institute Of Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
US11942317B2 (en) 2019-04-23 2024-03-26 The Trustees Of Indiana University Identification of sample subspecies based on particle mass and charge over a range of sample temperatures
US20220194992A1 (en) 2019-04-26 2022-06-23 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
AU2020270421A1 (en) 2019-05-03 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
WO2020227515A1 (en) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
WO2020232044A1 (en) 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
KR20220016100A (ko) 2019-05-31 2022-02-08 유니버시타트 아우토노마 데 바르셀로나 인슐린 유전자 요법
EP3995571A4 (de) 2019-07-03 2023-08-09 Nippon Medical School Foundation Verfahren zur herstellung von nukleinsäureverkapselten aav-hohlteilchen
EP3914720B9 (de) 2019-07-10 2023-09-27 Masonic Medical Research Laboratory Vgll4 mit ucp-1-cis-regulierendem element und verfahren zu dessen verwendung
US20220396806A1 (en) 2019-07-26 2022-12-15 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
US20220290182A1 (en) 2019-08-09 2022-09-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
EP4488287A3 (de) 2019-08-21 2025-03-05 Research Institute at Nationwide Children's Hospital Adeno-assoziierte virusvektorverabreichung von alpha-sarcoglycan und behandlung von muskeldystrophie
WO2021041485A1 (en) 2019-08-26 2021-03-04 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021061650A1 (en) 2019-09-25 2021-04-01 The Trustees Of Indiana University Apparatus and method for pulsed mode charge detection mass spectrometry
KR20220066164A (ko) 2019-09-27 2022-05-23 바이오마린 파머수티컬 인크. 크기 배제 크로마토그래피 및 다각도 광 산란 기술을 사용한 유전자 치료 바이러스 입자의 특성화
KR102849777B1 (ko) 2019-10-10 2025-08-22 더 트러스티즈 오브 인디애나 유니버시티 입자들을 식별, 선택 및 정제하기 위한 시스템 및 방법
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
AU2020384294A1 (en) 2019-11-14 2022-06-02 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with liver-specific gene therapy vectors
MX2022007135A (es) 2019-12-10 2022-09-19 Takeda Pharmaceuticals Co Vectores de virus adenoasociados para el tratamiento del síndrome de hunter.
CA3165106A1 (en) 2019-12-18 2021-06-24 The Trustees Of Indiana University Mass spectrometer with charge measurement arrangement
IL293972A (en) 2019-12-19 2022-08-01 Univ Pennsylvania Preparations for the treatment of Friedrich's ataxia
EP4100991B1 (de) 2020-02-03 2025-01-29 The Trustees of Indiana University Ein massenspektrometer zur ladungsdetektion und entsprechende methode
EP4100056A4 (de) * 2020-02-07 2024-03-06 Vecprobio, Inc. Rekombinante adeno-assoziierte virusvektoren in pflanzen
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
WO2021188960A1 (en) 2020-03-20 2021-09-23 University Of Florida Research Foundation, Inc. Gene therapy for cockayne syndrome
WO2021202651A1 (en) 2020-04-01 2021-10-07 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
MX2022013819A (es) 2020-05-05 2023-01-30 Univ North Carolina Chapel Hill Capsides de virus adeno-asociado 5 modificado y usos del mismo.
AU2021273447B2 (en) 2020-05-13 2026-03-05 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
AR122078A1 (es) 2020-05-13 2022-08-10 Akouos Inc Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
MX2022014754A (es) 2020-05-26 2023-01-16 UNIV AUTòNOMA DE BARCELONA Terapia genica del factor de crecimiento de fibroblastos 21 (fgf21) para trastornos del sistema nervioso central.
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
IL299771A (en) 2020-07-10 2023-03-01 Inst Nat Sante Rech Med Methods and compounds for the treatment of epilepsy
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
BR112023003310A2 (pt) 2020-08-24 2023-05-02 Univ Pennsylvania Vetores virais que codificam fusões de agonistas do receptor de glp-1 e usos dos mesmos no tratamento de doenças metabólicas
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
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
US20230364206A1 (en) 2020-10-07 2023-11-16 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
EP4228610A1 (de) 2020-10-13 2023-08-23 Kriya Therapeutics, Inc. Virale vektorkonstrukte zur abgabe von cytokin-codierenden nukleinsäuren und verwendungen davon zur behandlung von krebs
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
AU2021369480B2 (en) 2020-10-28 2025-05-22 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV2.5 vector
WO2022093769A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
CA3196499A1 (en) 2020-10-29 2022-05-05 James M. Wilson Aav capsids and compositions containing same
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
IL302608A (en) 2020-11-03 2023-07-01 Pfizer Methods for purification of aav vectors by anion exchange chromatography
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of aav vectors by affinity chromatography
KR20230127263A (ko) 2020-12-29 2023-08-31 아카우오스, 인크. Clrn1-연관된 청력손실 및/또는 시력손실을 치료하기위한 조성물 및 방법
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向ｔ細胞之ａａｖ載體
WO2022164923A1 (en) 2021-01-26 2022-08-04 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same
WO2022162067A1 (en) 2021-01-30 2022-08-04 Universitat Autònoma De Barcelona Gene therapy for monogenic diabetes
EP4284335A1 (de) 2021-02-01 2023-12-06 RegenxBio Inc. Gentherapie für neuronale ceroid-lipofuscinosen
WO2022183052A1 (en) 2021-02-26 2022-09-01 Takeda Pharmaceutical Company Limited Composition and methods for the treatment of fabry disease
US11760788B2 (en) 2021-03-02 2023-09-19 Pathways Neuro Pharma, Inc. Neuroreceptor compositions and methods of use
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187546A1 (en) 2021-03-03 2022-09-09 Shape Therapeutics Inc. Auxotrophic cells for virus production and compositions and methods of making
EP4301768A2 (de) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Kontrollierte expression von viralen proteinen
EP4301860A1 (de) 2021-03-04 2024-01-10 Kriya Therapeutics, Inc. Virale vektorkonstrukte mit dna zur hemmung toll-ähnlicher rezeptoren und verfahren zur verwendung davon
AU2022258312A1 (en) 2021-04-12 2023-10-26 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
WO2022221462A1 (en) 2021-04-13 2022-10-20 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic vlcad or mcad and methods of using the same
CA3216491A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
US20240181084A1 (en) 2021-04-23 2024-06-06 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas Fusion Protein and Methods of Treatment
EP4089171A1 (de) 2021-05-12 2022-11-16 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Rekombinante tert-codierende virale genome und vektoren
KR20240023638A (ko) 2021-06-22 2024-02-22 화이자 인코포레이티드 곤충 세포에서의 아데노-연관 바이러스 벡터의 생산
US20240358794A1 (en) 2021-08-11 2024-10-31 King’S College London Compositions and methods for improved treatment of disorders affecting the central nervous system
WO2023023220A1 (en) 2021-08-20 2023-02-23 Alexion Pharmaceuticals, Inc. Methods for treating sickle cell disease or beta thalassemia using a complement alternative pathway inhibitor
WO2023023227A1 (en) 2021-08-20 2023-02-23 Alexion Pharmaceuticals, Inc. Methods for treating sickle cell disease or beta thalassemia using complement alternative pathway inhibitors
IL311096A (en) 2021-08-31 2024-04-01 Scout Bio Inc Antigen binding molecules and their uses
EP4396201A1 (de) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav-kapsid-zusammensetzungen und verfahren zur freisetzung
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240376496A1 (en) 2021-09-03 2024-11-14 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240384242A1 (en) 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20250122530A1 (en) 2021-09-03 2025-04-17 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
EP4405396A2 (de) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von her2-positivem krebs
AU2022356427A1 (en) 2021-09-30 2024-05-09 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
US20240408240A1 (en) 2021-10-01 2024-12-12 Biomarin Pharmaceutical Inc. Treatment of hereditary angioedema with aav gene therapy vectors and therapeutic formulations
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
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
IL311861A (en) 2021-11-02 2024-06-01 Voyager Therapeutics Inc Aav capsid variants and uses thereof
AU2022387778A1 (en) 2021-11-09 2024-05-30 The Francis Crick Institute Limited Viral adaptors and uses thereof
US20250011372A1 (en) 2021-11-17 2025-01-09 Voyager Thrapeutics, Inc. Aav capsid variants and uses thereof
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
US20250059527A1 (en) 2021-12-16 2025-02-20 University Of Dundee Targeted degradation of alpha-synuclein
MX2024008509A (es) 2022-01-09 2024-07-19 Kriya Therapeutics Inc Construcciones de vectores para la distribucion de acidos nucleicos que codifican anticuerpos terapeuticos anti-igf-1r y metodos para usarlos.
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
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
US20250346920A1 (en) 2022-01-25 2025-11-13 Voyager Therapeutics, Inc. Baculovirus expression system
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
EP4473011A1 (de) 2022-02-02 2024-12-11 Akouos, Inc. Anti-vegf-antikörperkonstrukte und zugehörige verfahren zur behandlung von vestibulärem schwannoma-assoziierten symptomen
TW202342759A (zh) 2022-02-04 2023-11-01 美商史崔德生物公司 重組腺相關病毒載體及其使用方法
AU2023217698A1 (en) 2022-02-08 2024-07-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
WO2023187728A1 (en) 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2023212683A1 (en) 2022-04-29 2023-11-02 Kriya Therapeutics, Inc. Insulin and glucokinase gene therapy compositions and its use for treating diabetes
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
EP4532515A1 (de) 2022-06-02 2025-04-09 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
KR20250044484A (ko) 2022-06-24 2025-03-31 튠 쎄라퓨틱스, 인코포레이티드 표적 유전자 억제를 통해 저밀도 지단백질을 감소시키기 위한 조성물, 시스템, 및 방법
US20250215453A1 (en) 2022-06-28 2025-07-03 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
CA3264923A1 (en) 2022-08-25 2024-02-29 Takeda Pharmaceutical Company Limited COMPOSITION INTENDED FOR USE IN THE TREATMENT OF FABRY'S DISEASE
EP4584383A1 (de) 2022-09-07 2025-07-16 Universitat Autònoma De Barcelona Beta-hexosaminidase-vektoren
US20260085328A1 (en) 2022-09-08 2026-03-26 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024054993A1 (en) 2022-09-09 2024-03-14 Kriya Therapeutics, Inc. Vector constructs for delivery of nucleic acids encoding therapeutic anti-ctla4 antibodies and methods of using the same
CN120019071A (zh) 2022-09-15 2025-05-16 沃雅戈治疗公司 Tau结合化合物
WO2024056902A2 (en) 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
KR20250108100A (ko) 2022-09-22 2025-07-15 디나코 아게 Aav 유전자 치료법 벡터를 이용한 심근병증의 치료
TW202421788A (zh) 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用ａａｖ基因療法載體治療致心律不整性心肌病
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
AU2023392154A1 (en) 2022-12-14 2025-06-26 King's College London Compositions and methods for treating neurological diseases
WO2024130007A2 (en) 2022-12-16 2024-06-20 Kriya Therapeutics, Inc. Synthetic htert promoters and uses thereof
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
AU2023417630A1 (en) 2022-12-29 2025-05-15 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024147114A1 (en) 2023-01-06 2024-07-11 Bloomsbury Genetic Therapies Limited Compositions and methods for treating parkinson's disease
WO2024151568A1 (en) 2023-01-09 2024-07-18 Biomarin Pharmaceutical Inc. Epigenetic modifiers improve aav gene therapy durability
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024161022A2 (en) 2023-02-03 2024-08-08 King's College London Compositions and methods for treating neurological diseases
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
GB202304767D0 (en) 2023-03-30 2023-05-17 Ucl Business Ltd Synthetic AAV Capsid
WO2024211394A1 (en) 2023-04-03 2024-10-10 Decibel Therapeutics, Inc. Modified rep-cap plasmids and uses thereof
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
WO2024227074A1 (en) 2023-04-28 2024-10-31 Kriya Therapeutics, Inc. Riboswitches for regulating gene expression and therapeutic methods of using the same
US20240390521A1 (en) 2023-04-28 2024-11-28 Kriya Therapeutics, Inc. Chimeric modified ion channels and uses thereof for treatment of trigeminal nerve disorders
WO2024229173A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2024229164A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
AU2024265526A1 (en) 2023-05-03 2025-12-11 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
EP4704807A1 (de) 2023-05-03 2026-03-11 IOX Therapeutics Limited Liposomale inkt-zellmodulatorzusammensetzungen und verfahren zur verwendung
EP4705317A1 (de) 2023-05-03 2026-03-11 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von erkrankungen im zusammenhang mit cdkl5-mangel
EP4705488A1 (de) 2023-05-04 2026-03-11 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
WO2024233999A2 (en) 2023-05-11 2024-11-14 University Hospitals Cleveland Medical Center Anxiolytic therapy
WO2024238591A2 (en) 2023-05-15 2024-11-21 Biomarin Pharmaceutical Inc. Methods of treating anti-aav seropositive hemophilia patients
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
KR20260051103A (ko) 2023-07-07 2026-04-15 크리야 테라퓨틱스, 인크. 안과 병리상태를 치료하기 위한 aav 벡터의 안구주위 전달
EP4743583A1 (de) 2023-07-11 2026-05-20 Kriya Therapeutics Inc Vektorkonstrukte zur abgabe von uricase-codierenden nukleinsäuren und verfahren zur verwendung davon
WO2025015317A2 (en) 2023-07-13 2025-01-16 Kriya Therapeutics, Inc. Complement pathway inhibitor fusions and methods of using the same
TW202511280A (zh) 2023-07-21 2025-03-16 義大利商博雷亞醫療有限責任公司 製備表面修飾之病毒外殼之方法
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025038795A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025038800A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025038802A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN122029285A (zh) 2023-09-06 2026-05-12 迪纳柯公司 用aav基因治疗载体和治疗调配物治疗肥厚性心肌病的方法
WO2025082618A1 (en) * 2023-10-20 2025-04-24 Fuse Vectors Aps In vitro parvovirus capsid manufacturing
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
AU2024370040A1 (en) 2023-11-01 2026-04-23 Kriya Therapeutics, Inc. Aav mediated insulin and glucokinase gene therapy for treating diabetes
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
TW202540200A (zh) 2023-12-01 2025-10-16 美商基利科學股份有限公司 抗ｆａｐ－ｌｉｇｈｔ融合蛋白及其用途
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
TW202540436A (zh) 2023-12-05 2025-10-16 美商航海家醫療公司 用於治療與共濟蛋白(frataxin)缺陷相關之病症之組合物及方法
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025122543A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2025122536A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025126153A2 (en) 2023-12-14 2025-06-19 Aviadobio Ltd. Compositions and methods for treating sod1-mediated neurological diseases
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
EP4600264A1 (de) 2024-02-08 2025-08-13 Ecole Polytechnique Fédérale de Lausanne (EPFL) Manipulierte typ-2-cytokinsignalisierung zur verbesserung der car-t-zelleffektorfunktionen
TW202548022A (zh) 2024-03-03 2025-12-16 美商帕西奇生物公司 用於治療神經退化性病症之重組腺相關病毒
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
US20260056209A1 (en) 2024-06-14 2026-02-26 Gilead Sciences, Inc. Anti-ccr8 antibodies and uses thereof
WO2025262311A1 (en) 2024-06-21 2025-12-26 Institut National De La Sante Et De La Recherche Medicale Methods and products for prime editing genetic engineering
WO2025262314A1 (en) 2024-06-21 2025-12-26 Institut National De La Sante Et De La Recherche Medicale Methods and products for prime editing genetic engineering
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
WO2026028137A1 (en) 2024-07-31 2026-02-05 Universitat Autònoma De Barcelona Fibroblast growth factor 21 (fgf21) gene therapy
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026096611A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein
WO2026096599A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering syntaxin-binding protein-1
WO2026096600A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein
WO2026096603A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering syntaxin-binding protein-1

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) * 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
WO1991019803A1 (en) * 1990-06-19 1991-12-26 Applied Biotechnology, Incorporated Self assembled, defective, nonself-propagating viral particles
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy

Also Published As

Publication number Publication date
CA2193802A1 (en) 1996-01-11
DE69534746T2 (de) 2006-09-14
AU705564B2 (en) 1999-05-27
DE69534746D1 (de) 2006-04-06
AU2913895A (en) 1996-01-25
JPH10502526A (ja) 1998-03-10
EP0766569A1 (de) 1997-04-09
US6204059B1 (en) 2001-03-20
US20010016355A1 (en) 2001-08-23
WO1996000587A1 (en) 1996-01-11
US5863541A (en) 1999-01-26
EP0766569B1 (de) 2006-01-11
EP0766569A4 (de) 1999-06-02

Similar Documents

Publication Publication Date Title
DE69534746D1 (de) Aav-capsidvehikel für den molekularen transport
FR19C1020I1 (fr) Nouvelles molecules b7-4 et leurs utilisations
ATE366814T1 (de) Gl50 moleküle, sowie verwendungen derselben
ATE214100T1 (de) Herstellung von rekombinanten proteinen in einem rinderadenovirus-expressionsvektorsystem
DK0941318T3 (da) Håndvægtformede konstruerede ekspressionssystemer til genterapi
BR9708387A (pt) Processo para vacinação de um mamìfero a um antìgeno selecionado, células patogênica e hospedeira, composição de vacina, ácido nucleico, e, opsonina engenheirada.
WO2000073316A3 (en) Virus immunologic determinants
PL409838A1 (pl) Rekombinowane wirusy stowarzyszone z adenowirusem (AAV), kompozycje , wyizolowane białko kapsydu, wyizolowana lub syntetyczna cząsteczka kwasu nukleinowego, sposób wytwarzania rekombinowanych wirusów, komórki gospodarza, białka obejmujące fragment białka kapsydu AAV, sztuczne białka, rekombinowane wirusy, cząsteczki, sposoby dostarczania transgenu do komórki , sposób identyfikacji serotypu sekwencji wirusa (AAV), zestaw diagnostyczny, sposób izolacji nowych wirusów, nowe serotypy wirusa, wyizolowane wirusy, rekombinowana komórka, zastosowanie wirusa
WO2003092594A8 (en) Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
ATE465266T1 (de) Zusammensetzungen und verfahren zur verpackung von alpavirus-vektoren
KR880700070A (ko) 비루스 왁찐
IL136634A0 (en) Recombinant noda virus compositions and methods
IL77694A (en) Canine parvovirus vaccines comprising viruses of the canine parvovirus strain 154 or viruses derived therefrom by additional cell culture passaging
O'Callaghan et al. Immunogenicity of foreign peptide epitopes expressed in bacterial envelope proteins
KR890014740A (ko) 재조합 마렉병 비루스 및 백신
ATE315658T1 (de) Genkonstrukte für genimmunisierung
Beyer et al. Recombinant expression of lymphocytic choriomeningitis virus strain WE glycoproteins: a single amino acid makes the difference
PT951552E (pt) Adn e prote?nas ou p?ptidos espec?ficos de bact?rias da esp?cie neisseria meningitidis, processos para a sua obten??o e suas aplica??es biol?gicas
Teigler et al. Hexon hypervariable region-modified adenovirus type 5 (Ad5) vectors display reduced hepatotoxicity but induce T lymphocyte phenotypes similar to Ad5 vectors
WO2001073028A3 (en) A p-selectin glycoprotein ligand (psgl-1) binding protein and uses therefor
ATE480620T1 (de) Neue pgc-1-isoformen und deren verwendungen
EP1157105A4 (de) Regulatorische konstrukte, die das intron 3 des prostat-spezifischen membranatigen-gens enthalten
Wobbe et al. Proteins tightly associated with the termini of replicative form DNA of Kilham rat virus, an autonomous parvovirus.
DE69941099D1 (de) Spezifische Nukleinsäuren und Polypeptide aus krankheitserregenden Stämmen von Neisseria
WO2002026803A3 (en) 22108 and 47916, novel human thioredoxin family members and uses thereof

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties