ES2874298T3 - Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos - Google Patents

Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos Download PDF

Info

Publication number
ES2874298T3
ES2874298T3 ES17153156T ES17153156T ES2874298T3 ES 2874298 T3 ES2874298 T3 ES 2874298T3 ES 17153156 T ES17153156 T ES 17153156T ES 17153156 T ES17153156 T ES 17153156T ES 2874298 T3 ES2874298 T3 ES 2874298T3
Authority
ES
Spain
Prior art keywords
aav
seq
sequences
factor
capsid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Lifetime
Application number
ES17153156T
Other languages
English (en)
Spanish (es)
Inventor
Guangping Gao
James M Wilson
Luk Vandenberghe
Mauricio R Alvira
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Pennsylvania Penn
Original Assignee
University of Pennsylvania Penn
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=34426044&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ES2874298(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by University of Pennsylvania Penn filed Critical University of Pennsylvania Penn
Application granted granted Critical
Publication of ES2874298T3 publication Critical patent/ES2874298T3/es
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

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)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
ES17153156T 2003-09-30 2004-09-30 Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos Expired - Lifetime ES2874298T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US50822603P 2003-09-30 2003-09-30
US56654604P 2004-04-29 2004-04-29

Publications (1)

Publication Number Publication Date
ES2874298T3 true ES2874298T3 (es) 2021-11-04

Family

ID=34426044

Family Applications (5)

Application Number Title Priority Date Filing Date
ES17153156T Expired - Lifetime ES2874298T3 (es) 2003-09-30 2004-09-30 Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES10182796.2T Expired - Lifetime ES2648241T3 (es) 2003-09-30 2004-09-30 Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES04788580T Expired - Lifetime ES2411479T3 (es) 2003-09-30 2004-09-30 Clados de virus adeno-asociados (AAV), secuencias, vectores que los contienen, y usos de los mismos
ES10182587.5T Expired - Lifetime ES2616016T3 (es) 2003-09-30 2004-09-30 Clados de virus adeno-asociados (AAV), secuencias, vectores que las contienen y usos de los mismos
ES10182098.3T Expired - Lifetime ES2555612T3 (es) 2003-09-30 2004-09-30 Clados de virus adeno-asociados (AAV), secuencias, vectores que las contienen y usos de los mismos

Family Applications After (4)

Application Number Title Priority Date Filing Date
ES10182796.2T Expired - Lifetime ES2648241T3 (es) 2003-09-30 2004-09-30 Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES04788580T Expired - Lifetime ES2411479T3 (es) 2003-09-30 2004-09-30 Clados de virus adeno-asociados (AAV), secuencias, vectores que los contienen, y usos de los mismos
ES10182587.5T Expired - Lifetime ES2616016T3 (es) 2003-09-30 2004-09-30 Clados de virus adeno-asociados (AAV), secuencias, vectores que las contienen y usos de los mismos
ES10182098.3T Expired - Lifetime ES2555612T3 (es) 2003-09-30 2004-09-30 Clados de virus adeno-asociados (AAV), secuencias, vectores que las contienen y usos de los mismos

Country Status (17)

Country Link
US (13) US7906111B2 (enExample)
EP (7) EP2292779B1 (enExample)
JP (9) JP5054975B2 (enExample)
CN (4) CN102174574B (enExample)
AU (2) AU2004278684B2 (enExample)
BE (1) BE2021C533I2 (enExample)
CA (3) CA3072423A1 (enExample)
CY (3) CY1119833T1 (enExample)
DK (4) DK2292779T3 (enExample)
ES (5) ES2874298T3 (enExample)
FR (1) FR21C1037I1 (enExample)
HU (4) HUE054805T2 (enExample)
NZ (1) NZ545628A (enExample)
PL (2) PL2292780T3 (enExample)
PT (2) PT2292780T (enExample)
SI (2) SI3211085T1 (enExample)
WO (1) WO2005033321A2 (enExample)

Families Citing this family (637)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
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
ES2874298T3 (es) * 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
ES2442225T3 (es) 2004-04-28 2014-02-10 The Trustees Of The University Of Pennsylvania Régimen de inmunización con cebado de adenovirus suprimido en E4 y potenciación de adenovirus suprimido en E1
ES2428218T3 (es) 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
AU2007234698B2 (en) 2006-04-07 2013-08-01 The Board Of Regents Of The University Of Texas System Methods and compositions related to adenoassociated virus-phage particles
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
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
EP2191001B1 (en) 2007-04-09 2016-06-08 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
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
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
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
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides 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
DK2425000T3 (da) 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9133479B2 (en) * 2009-06-03 2015-09-15 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
DK2673289T3 (da) * 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
SG10201601110VA (en) 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
SMT202400520T1 (it) 2011-04-22 2025-01-14 Univ California Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo
US20130039888A1 (en) * 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
US9169299B2 (en) 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University AAV capsid proteins for nucleic acid transfer
EP2764119A2 (en) 2011-10-05 2014-08-13 Genelux Corporation Method for detecting replication or colonization of a biological therapeutic
WO2013063019A1 (en) 2011-10-28 2013-05-02 The Wistar Institute Of Anatomy And Biology Methods and compositions for enhancing the therapeutic effect of anti-tumor t cells
WO2013082268A1 (en) 2011-11-30 2013-06-06 The Wistar Institute Of Anatomy And Biology Methods and compositions for regulation of cell aging, carcinogenesis, and reprogramming
EP2601968A1 (en) * 2011-12-06 2013-06-12 Deutsches Krebsforschungszentrum HPV derived polynucleic acids for therapy
WO2013103896A1 (en) * 2012-01-06 2013-07-11 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
US20140087362A1 (en) 2012-03-16 2014-03-27 Aladar A. Szalay Methods for assessing effectiveness and monitoring oncolytic virus treatment
WO2013158265A1 (en) 2012-04-20 2013-10-24 Genelux Corporation Imaging methods for oncolytic virus therapy
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
JP6199965B2 (ja) 2012-07-11 2017-09-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療
EP2879719B1 (en) 2012-08-01 2018-07-04 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
US9636370B2 (en) * 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
US20140140959A1 (en) 2012-10-05 2014-05-22 Aladar A. Szalay Energy Absorbing-Based Diagnostic and Therapeutic Methods Employing Nucleic Acid Molecules Encoding Chromophore-Producing Enzymes
EP2749569A1 (en) 2012-12-28 2014-07-02 Annibale Alessandro Puca Variant of BPIFB4 protein
US8957044B2 (en) 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
WO2014160092A1 (en) * 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
CA2904960A1 (en) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
KR102346455B1 (ko) 2013-03-15 2022-01-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ｍｐｓｉ 치료를 위한 조성물 및 방법
CN110885854A (zh) * 2013-03-15 2020-03-17 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
EP3561062A1 (en) * 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
EP3633041A3 (en) 2013-09-26 2020-07-29 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CN106029107A (zh) * 2013-10-07 2016-10-12 基罗米克有限责任公司 使用疾病特异性启动子治疗心血管疾病的组合物和方法
WO2015054653A2 (en) * 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
US10238700B2 (en) 2014-01-02 2019-03-26 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity
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
DK3116900T3 (da) 2014-03-09 2020-09-28 Univ Pennsylvania Sammensætninger som kan anvendes til behandling af ornithintranscarbamylase (otc) defekt
MX385339B (es) 2014-03-10 2025-03-18 Uniqure Ip Bv Vectores aav mejorados adicionales producidos en células de insecto.
CN106414474B (zh) 2014-03-17 2021-01-15 阿德夫拉姆生物技术股份有限公司 用于视锥细胞中增强的基因表达的组合物和方法
EP4410805A3 (en) 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
JP6741590B2 (ja) 2014-04-25 2020-08-19 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用
US10780182B2 (en) 2014-04-25 2020-09-22 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
JP6974943B2 (ja) 2014-05-13 2021-12-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ２種の抗体構築物を発現するａａｖを含む組成物およびこの使用
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
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
PL3198018T3 (pl) 2014-09-24 2021-07-19 City Of Hope Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
JP6625627B2 (ja) 2014-10-14 2019-12-25 ハロザイム インコーポレイテッド アデノシンデアミナーゼ−２（ａｄａ２）、その変異体の組成物およびそれを使用する方法
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
EP3215602B1 (en) 2014-11-05 2019-12-25 The Research Institute at Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CN107532173A (zh) * 2014-11-21 2018-01-02 北卡罗来纳-查佩尔山大学 靶向中枢神经系统的aav载体
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
CA2967468A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
EP4599851A3 (en) 2015-01-16 2025-12-31 University of Washington NEW MICRO-DYSTROPHINS AND ASSOCIATED METHODS OF USE
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
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
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
CA2978068C (en) 2015-03-02 2024-05-28 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
CN107635575A (zh) 2015-03-10 2018-01-26 纽约市哥伦比亚大学理事会 重组glut1腺相关病毒载体构建体以及用于恢复glut1表达的相关方法
JP6836999B2 (ja) * 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions 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
HK1252512A1 (zh) 2015-05-07 2019-05-31 Massachusetts Eye & Ear Infirmary 向眼部遞送藥劑的方法
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EP3294894B8 (en) * 2015-05-12 2019-09-25 The U.S.A. as represented by the Secretary, Department of Health and Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US11535665B2 (en) 2015-05-13 2022-12-27 The Trustees Of The University Of Pennsylvania AAV-mediated expression of anti-influenza antibodies and methods of use thereof
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
PT3872085T (pt) * 2015-07-30 2023-04-13 Massachusetts Eye & Ear Infirmary Sequências de aav ancestrais e utilizações destas
WO2017023803A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Modified cells and methods of therapy
KR102745604B1 (ko) 2015-08-06 2024-12-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Glp-1 및 대사성 질환 치료용 조성물에서 이의 용도
EA201890565A1 (ru) 2015-08-25 2019-04-30 Дьюк Юниверсити Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз
US11117942B2 (en) 2015-08-31 2021-09-14 The Trustees Of The University Of Pennsylvania AAV-EPO for treating companion animals
CN108291216B (zh) 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
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
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US12188037B2 (en) * 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
EP3364996B1 (en) * 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070491A1 (en) * 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
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
KR20210005994A (ko) 2015-11-05 2021-01-15 뱀부 테라퓨틱스 인코포레이티드 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
ES2869284T3 (es) 2015-12-11 2021-10-25 California Inst Of Techn Péptidos de diana para dirigir virus adenoasociados (AAV)
EP3386537B1 (en) 2015-12-11 2021-10-20 Massachusetts Eye and Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3394270A1 (en) * 2015-12-14 2018-10-31 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
CA3007330A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
MX2018007230A (es) 2015-12-14 2018-11-09 Univ Pennsylvania Terapia genica para trastornos oculares.
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
KR20180121899A (ko) 2016-02-03 2018-11-09 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 I형 점액다당류증을 치료하기 위한 유전자 요법
SI3411484T1 (sl) 2016-02-05 2023-12-29 Emory University Injiciranje enojnega ali samokomplementarnega adeno-povezanega virusa 9 v cerebrospinalno tekočino
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
CA3019847A1 (en) 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
AU2017248830B2 (en) 2016-04-15 2023-03-09 Alpine Immune Sciences, Inc. CD80 variant immunomodulatory proteins and uses thereof
KR102574810B1 (ko) 2016-04-15 2023-09-08 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 습성 연령 관련 황반 변성의 치료를 위한 조성물
AU2017248656B2 (en) 2016-04-15 2023-07-27 The Trustees Of The University Of Pennsylvania Novel AAV8 mutant capsids and compositions containing same
IL262211B2 (en) 2016-04-15 2024-01-01 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
SG11201808422QA (en) 2016-04-15 2018-10-30 Univ Pennsylvania Gene therapy for treating hemophilia a
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
CA3019199A1 (en) 2016-04-15 2017-10-19 Alpine Immune Sciences, Inc. Icos ligand variant immunomodulatory proteins and uses thereof
WO2017184463A1 (en) 2016-04-17 2017-10-26 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US10882886B2 (en) * 2016-05-03 2021-01-05 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
EP4209501A1 (en) 2016-05-13 2023-07-12 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN109415730A (zh) 2016-06-13 2019-03-01 北卡罗来纳大学教堂山分校 优化的cln1基因和表达盒以及它们的应用
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
KR102526506B1 (ko) 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
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
US11584780B2 (en) * 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
EP3491013A1 (en) 2016-07-28 2019-06-05 Alpine Immune Sciences, Inc. Cd155 variant immunomodulatory proteins and uses thereof
US11834490B2 (en) 2016-07-28 2023-12-05 Alpine Immune Sciences, Inc. CD112 variant immunomodulatory proteins and uses thereof
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
US20200340012A1 (en) 2016-08-18 2020-10-29 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CA3041068A1 (en) 2016-10-18 2018-04-26 Regents Of The University Of Minnesota Tumor infiltrating lymphocytes and methods of therapy
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
AU2017345764A1 (en) 2016-10-20 2019-05-02 Alpine Immune Sciences, Inc. Secretable variant immunomodulatory proteins and engineered cell therapy
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
EP3548065B1 (en) 2016-12-01 2022-11-09 INSERM - Institut National de la Santé et de la Recherche Médicale Pharmaceutical compositions for the treatment of retinal degenerative diseases
CN110325199A (zh) 2016-12-30 2019-10-11 宾夕法尼亚州立大学托管会 用于治疗苯丙酮尿症的基因疗法
CN110290814A (zh) 2016-12-30 2019-09-27 宾夕法尼亚州立大学托管会 用于治疗威尔逊病的基因疗法
WO2018134168A1 (en) 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
JP2020506705A (ja) 2017-02-01 2020-03-05 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 シトルリン血症を処置するための遺伝子療法
CA3052473A1 (en) 2017-02-09 2018-08-16 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and compositions and methods thereof
CA3051011A1 (en) 2017-02-20 2018-08-23 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3114549A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
EP3655533A1 (en) 2017-02-24 2020-05-27 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
WO2018160582A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
WO2018160573A1 (en) * 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
BR112019017327A2 (pt) 2017-03-01 2020-04-14 Univ Pennsylvania terapia gênica para distúrbios oculares
EP3589311A1 (en) 2017-03-02 2020-01-08 Genethon Method for removing anti-aav antibodies from a blood-derived composition
IL320149A (en) 2017-03-16 2025-06-01 Alpine Immune Sciences Inc Pd-l1 variant immunomodulatory proteins and uses thereof
IL319966A (en) 2017-03-16 2025-05-01 Alpine Immune Sciences Inc CD80 Variant Immune Modulator Proteins and Uses Thereof
CA3053812A1 (en) 2017-03-16 2018-09-20 Alpine Immune Sciences, Inc. Pd-l2 variant immunomodulatory proteins and uses thereof
WO2018175917A1 (en) 2017-03-24 2018-09-27 Lankenau Institute For Medical Research Methods and compositions for inducible extracellular membrane capture of monoclonal immunoglobulins secreted by hybridomas
EP3613856A4 (en) 2017-03-31 2020-12-23 Staidson(Beijing) Biopharmaceuticals Co., Ltd. SHORT RNA EXPRESSION CASSETTE IN HAIRPIN, POLYNUCLEOTIDIAL SEQUENCE WEARING IT AND CORRESPONDING APPLICATION
US11499154B2 (en) 2017-04-10 2022-11-15 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
UY37679A (es) 2017-04-14 2018-11-30 Regenxbio Inc Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas
AU2018255293B2 (en) 2017-04-17 2024-03-07 University Of Florida Research Foundation, Incorporated Regulation of RAN translation by PKR and eIF2a-P pathways
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
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
CA3061655A1 (en) 2017-05-11 2018-11-15 The Trustees Of The University Of Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
EP3630986A4 (en) 2017-05-31 2021-08-11 The Trustees of The University of Pennsylvania GENE THERAPY FOR TREATMENT OF PEROXISOMAL DISEASES
WO2018232149A1 (en) 2017-06-14 2018-12-20 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
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
BR112019019015A2 (pt) 2017-06-30 2020-04-14 Univ California vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso
WO2019010335A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
WO2019008157A1 (en) 2017-07-07 2019-01-10 Genethon NOVEL POLYNUCLEOTIDES ENCODING HUMAN FKRP PROTEIN
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
AU2018307877A1 (en) 2017-07-27 2020-01-02 Novartis Ag Sheddase resistant TREM2 variants
JP7221275B2 (ja) 2017-08-03 2023-02-13 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を送達するための組成物および方法
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
UA127455C2 (uk) 2017-09-20 2023-08-30 4Д Молекьюлар Терапьютикс Інк. Капсид аденоасоційованого вірусу і спосіб його застосування
WO2019060686A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
AU2018338451A1 (en) 2017-09-25 2020-04-02 University Of Florida Research Foundation, Incorporated Immunoassays for detection of RAN proteins
JP7350337B2 (ja) 2017-09-26 2023-09-26 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 神経障害の処置においてｒａｎタンパク質レベルを低下させるためのメトホルミンおよびそのアナログの使用
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202500579A (zh) 2017-10-18 2025-01-01 美商艾爾潘免疫科學有限公司 變異型icos 配位體免疫調節蛋白及相關組合物及方法
EP3697449A1 (en) 2017-10-18 2020-08-26 REGENXBIO Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap
CA3079568A1 (en) 2017-10-18 2019-04-25 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
US11723988B2 (en) 2017-10-20 2023-08-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
WO2019104279A1 (en) 2017-11-27 2019-05-31 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
US11723989B2 (en) 2017-11-30 2023-08-15 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIB
KR102823833B1 (ko) 2017-11-30 2025-06-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 뮤코다당류증 iiia형에 대한 유전자 요법
EA202091509A1 (ru) 2017-12-19 2020-09-22 Акуос, Инк. Aav-опосредованная доставка терапевтических антител во внутреннее ухо
US12297253B2 (en) 2018-01-03 2025-05-13 Alpine Immune Sciences, Inc. Multi-domain immunomodulatory proteins and methods of use thereof
KR102892243B1 (ko) 2018-01-17 2025-11-27 아드레나스 테라퓨틱스, 인코포레이티드 21-하이드록실라제 결핍을 위한 아데노-관련 바이러스 유전자 요법
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
EP3758724A4 (en) * 2018-02-27 2022-07-06 The Trustees of The University of Pennsylvania NOVEL VECTORS OF ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
US12416016B2 (en) * 2018-02-27 2025-09-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor
BR112020018354A2 (pt) * 2018-03-16 2020-12-29 Research Institute At Nationwide Children's Hospital Aumento de fornecimento gênico específico a tecido através de modificação de capsídeo
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，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
WO2019195701A1 (en) 2018-04-05 2019-10-10 Massachusetts Eye And Ear Infirmary Methods of making and using combinatorial barcoded nucleic acid libraries having defined variation
MA51353B1 (fr) 2018-04-05 2022-09-30 Univ Sorbonne Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit
US12460226B2 (en) 2018-04-16 2025-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
MX2020010994A (es) 2018-04-18 2021-01-08 Univ Columbia Terapia de gen para enfermedades causadas por combinaciones desequilibradas de nucleótidos que incluyen síndromes de agotamiento de adn mitocondrial.
RU2020134965A (ru) 2018-04-27 2022-04-27 Спейскрафт Севен, Ллк Генная терапия при дегенерации цнс
EP3787771A1 (en) 2018-04-29 2021-03-10 REGENXBIO Inc. Scalable clarification process for recombinant aav production
US20210231560A1 (en) 2018-04-29 2021-07-29 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
CA3098871A1 (en) 2018-05-08 2019-11-14 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
BR112020022858A2 (pt) 2018-05-11 2021-02-23 Massachusetts Eye And Ear Infirmary tropismo específico hepático de vírus adeno-associado
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
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
JP2021522840A (ja) 2018-05-15 2021-09-02 プレジデント アンド フェローズ オブ ハーバード カレッジ 改善された遺伝子送達特性を示すウイルスベクター
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38247A (es) 2018-05-30 2019-12-31 Novartis Ag Anticuerpos frente a entpd2, terapias de combinación y métodos de uso de los anticuerpos y las terapias de combinación
US11505782B2 (en) 2018-06-04 2022-11-22 Calidi Biotherapeutics, Inc. Cell-based vehicles for potentiation of viral therapy
ES2966045T3 (es) 2018-06-04 2024-04-18 Calidi Biotherapeutics Inc Vehículos basados en células para la potenciación de la terapia viral
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2019241315A1 (en) 2018-06-12 2019-12-19 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CN112469822A (zh) 2018-06-14 2021-03-09 再生生物股份有限公司 用于重组aav生产的阴离子交换色谱
WO2019241758A1 (en) 2018-06-15 2019-12-19 Alpine Immune Sciences, Inc. Pd-1 variant immunomodulatory proteins and uses thereof
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
AU2019299970B2 (en) 2018-07-12 2025-12-11 Spacecraft Seven, Llc Gene therapy vectors for treatment of Danon disease
CN113302202B (zh) 2018-07-17 2024-01-30 赫利世弥斯株式会社 利用表达胰岛素样生长因子1异构体的脱氧核糖核酸构建体的神经病变的治疗
WO2020018691A1 (en) * 2018-07-18 2020-01-23 The General Hospital Corporation Modified t cells and methods of their use
EP3826719A1 (en) 2018-07-24 2021-06-02 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2020033842A1 (en) 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production
WO2020037303A1 (en) 2018-08-16 2020-02-20 The Regents Of The University Of California Chemically and photochemically initiated cell membrane blebbing to induce cell vesicle production, modifications thereof, and uses thereof
US11242528B2 (en) 2018-08-28 2022-02-08 Actym Therapeutics, Inc. Engineered immunostimulatory bacterial strains and uses thereof
SG11202102058UA (en) 2018-08-30 2021-03-30 Tenaya Therapeutics Inc Cardiac cell reprogramming with myocardin and ascl1
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
AU2019346655B2 (en) 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020072451A1 (en) 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
JP7289033B2 (ja) 2018-10-01 2023-06-09 徹 宮崎 神経変性疾患治療剤
EP3860659A1 (en) 2018-10-01 2021-08-11 The Trustees of the University of Pennsylvania Compositions useful for treating gm1 gangliosidosis
CN119684417A (zh) 2018-10-02 2025-03-25 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
EP3861107A1 (en) 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
US20210324483A1 (en) 2018-10-15 2021-10-21 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
AU2019360937A1 (en) 2018-10-15 2021-05-20 Voyager Therapeutics, Inc. Expression vectors for large-scale production of rAAV in the baculovirus/Sf9 system
MX2021004602A (es) 2018-10-22 2021-09-08 Univ Rochester Edición del genoma por inserción no homóloga de adn dirigida utilizando una proteína de fusión retroviral integrasacas9.
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
CA3116192C (en) 2018-11-06 2025-09-23 Calidi Biotherapeutics (Nevada), Inc. IMPROVED SYSTEMS FOR CELL-MEDIATED ONCOLYTIC VIRAL THERAPY
KR20210093954A (ko) 2018-11-16 2021-07-28 아스텔라스세이야쿠 가부시키가이샤 유트로핀 유전자를 표적으로 하여 근이영양증을 치료하는 방법
EP3884041A2 (en) 2018-11-21 2021-09-29 Indapta Therapeutics, Inc. Methods for expansion of natural killer (nk) cell subset and related compositions and methods
WO2020113141A2 (en) 2018-11-30 2020-06-04 Alpine Immune Sciences, Inc. Cd86 variant immunomodulatory proteins and uses thereof
AU2019389047A1 (en) * 2018-11-30 2021-05-20 Novartis Ag AAV viral vectors and uses thereof
WO2020117898A1 (en) 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
WO2020140007A1 (en) 2018-12-28 2020-07-02 University Of Rochester Gene therapy for best1 dominant mutations
JP7531495B2 (ja) 2019-01-04 2024-08-09 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
EP3911741A1 (en) 2019-01-14 2021-11-24 University of Rochester Targeted nuclear rna cleavage and polyadenylation with crispr-cas
CN113631225A (zh) 2019-01-18 2021-11-09 沃雅戈治疗公司 用于生产aav颗粒的方法和系统
JP2022523719A (ja) 2019-01-31 2022-04-26 オレゴン・ヘルス・アンド・サイエンス・ユニバーシティ Ａａｖキャプシドの転写依存性定方向進化を使用するための方法
SG11202108504YA (en) 2019-02-22 2021-09-29 Univ Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
MX2021010149A (es) 2019-02-25 2021-09-14 Novartis Ag Composiciones y metodos para tratar la distrofia cristalina de bietti.
EP3931337A1 (en) 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Compositions useful in treatment of krabbe disease
MX2021010273A (es) 2019-02-27 2021-09-23 Actym Therapeutics Inc Bacterias inmunoestimuladoras modificadas para colonizar tumores, celulas inmunitarias residentes de tumor y el microentorno tumoral.
US12024709B2 (en) 2019-02-27 2024-07-02 Actym Therapeutics, Inc. Immunostimulatory bacteria engineered to colonize tumors, tumor-resident immune cells, and the tumor microenvironment
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CN113677697B (zh) 2019-03-25 2024-06-04 吉尼松公司 使用重叠的aav载体生产大型拟抗肌萎缩蛋白
WO2020205889A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP3946467A1 (en) 2019-04-03 2022-02-09 REGENXBIO Inc. Gene therapy for eye pathologies
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
FI3953483T3 (fi) 2019-04-11 2023-11-30 Regenxbio Inc Kokoekskluusiokromatografiamenetelmiä rekombinanttien adenoassosioituneiden viruskoostumusten karakterisoimiseksi
MX2021012564A (es) 2019-04-19 2022-01-18 Regenxbio Inc Formulaciones y métodos de vectores de virus adenoasociados.
CA3137284A1 (en) 2019-04-24 2020-10-29 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
SG11202111102SA (en) * 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP3962536A1 (en) 2019-04-29 2022-03-09 Voyager Therapeutics, Inc. Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors
US12338451B2 (en) 2019-05-03 2025-06-24 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
EP3969061A1 (en) 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
EP3976785A1 (en) 2019-05-28 2022-04-06 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
EP3976806A1 (en) 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Recombinant herpesvirales vector
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
TW202122579A (zh) 2019-07-02 2021-06-16 美商Ｍ６Ｐ生物醫藥公司 用於治療溶酶體儲積症之載體組合物及其使用方法
EP3997225A1 (en) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
FI3997214T3 (fi) 2019-07-11 2023-11-02 Centre Nat Rech Scient Kemiallisesti modifioitu adeno-associated virus
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
EP4004206A1 (en) 2019-07-23 2022-06-01 University of Rochester Targeted rna cleavage with crispr-cas
US20230042103A1 (en) 2019-07-26 2023-02-09 Regenxbio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
US20220396806A1 (en) 2019-07-26 2022-12-15 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
US20230173036A1 (en) 2019-08-16 2023-06-08 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting lama1 gene
WO2021034920A1 (en) * 2019-08-20 2021-02-25 St. Jude Children's Research Hospital, Inc. SCHIZOPHRENIA-RELATED MICRODELETION GENE 2510002D24Rik IS ESSENTIAL FOR SOCIAL MEMORY
CN114502197A (zh) 2019-08-26 2022-05-13 再生生物股份有限公司 用全人经翻译后修饰的抗VEGF Fab治疗糖尿病性视网膜病变
US20220307013A1 (en) 2019-08-30 2022-09-29 The Regents Of The University Of California Gene fragment overexpression screening methodologies, and uses thereof
US20230092895A1 (en) 2019-08-30 2023-03-23 Obsidian Therapeutics, Inc. Tandem cd19 car-based compositions and methods for immunotherapy
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021046451A1 (en) 2019-09-06 2021-03-11 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
EP4028532A1 (en) 2019-09-13 2022-07-20 Rutgers, the State University of New Jersey Aav-compatible laminin-linker polymerization proteins
EP4031578A1 (en) 2019-09-18 2022-07-27 Novartis AG Entpd2 antibodies, combination therapies, and methods of using the antibodies and combination therapies
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與ｅｎｔｐｄ２抗體之組合療法
JP7651562B2 (ja) 2019-09-19 2025-03-26 ジェネトン Ｆｋｒｐの心毒性を軽減する遺伝子治療発現系
CA3154741A1 (en) 2019-09-20 2021-03-25 University Of Florida Research Foundation, Incorporated Detection of antibodies against ran proteins from serum and tissue lysates
JP2022550435A (ja) 2019-10-04 2022-12-01 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 組換えａａｖの改善された治療的使用のための方法
JP2022552262A (ja) 2019-10-07 2022-12-15 リジェネックスバイオ インコーポレイテッド アデノ随伴ウイルスベクター医薬組成物および方法
CN114761546A (zh) 2019-10-08 2022-07-15 波士顿学院董事会 含有多个不同非天然氨基酸的蛋白质以及制备和使用此类蛋白质的方法
CN115003820A (zh) 2019-10-08 2022-09-02 爱科霍拉有限公司 用于眼部治疗的组合物和方法
WO2021076600A1 (en) 2019-10-14 2021-04-22 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
US20230002451A1 (en) * 2019-11-08 2023-01-05 President And Fellows Of Harvard College Viral capsid polypeptides
WO2021092300A1 (en) * 2019-11-08 2021-05-14 President And Fellows Of Harvard College Viral capsid polypeptides
WO2021099394A1 (en) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides and their use for the treatment of cancer
US20230270886A1 (en) 2019-11-28 2023-08-31 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
CN114981442A (zh) * 2019-12-04 2022-08-30 桑格摩生物治疗股份有限公司 用于生产重组aav的新颖组合物及方法
EP4073257A1 (en) 2019-12-09 2022-10-19 UCL Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
IL293684A (en) 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
CA3166374A1 (en) 2019-12-31 2021-07-08 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
JP2023511382A (ja) 2020-01-22 2023-03-17 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
WO2021154956A1 (en) 2020-01-29 2021-08-05 Regenxbio Inc. Treatment of mucopolysaccharidosis iva
WO2021154963A1 (en) 2020-01-29 2021-08-05 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
CN115867323A (zh) 2020-02-02 2023-03-28 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂症的组成物
WO2021158982A2 (en) 2020-02-07 2021-08-12 University Of Rochester Targeted translation of rna with crispr-cas13 to enhance protein synthesis
JP2023514149A (ja) 2020-02-07 2023-04-05 ユニバーシティ オブ ロチェスター リボザイムにより媒介されるｒｎａアセンブリ及び発現
IL294781A (en) 2020-02-14 2022-09-01 Ultragenyx Pharmaceutical Inc Gene therapy for treating cdkl5 deficiency disorder
US20230136699A1 (en) 2020-02-28 2023-05-04 INSERM (Institut National de la Santé et de la Recherche Médicale) Gene therapy for maple syrup urine disease
JP2023515672A (ja) 2020-03-02 2023-04-13 テナヤ セラピューティクス， インコーポレイテッド 心筋細胞発現マイクロｒｎａによる遺伝子ベクター制御
EP4114449A2 (en) 2020-03-05 2023-01-11 Neotx Therapeutics Ltd. Methods and compositions for treating cancer with immune cells
CN115485289A (zh) 2020-03-11 2022-12-16 宾夕法尼亚大学董事会 使用工程化的病毒颗粒进行基因递送的方法和组合物
EP4121544A1 (en) * 2020-03-19 2023-01-25 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
EP4127169A1 (en) 2020-03-27 2023-02-08 University of Rochester Targeted destruction of viral rna by crispr-cas13
CA3175034A1 (en) 2020-03-27 2021-09-30 UCB Biopharma SRL Autonomous knob domain peptides
EP4127170A1 (en) 2020-03-27 2023-02-08 University of Rochester Crispr-cas13 crrna arrays
JP2023520402A (ja) 2020-03-31 2023-05-17 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 プロピオン酸血症を処置するための遺伝子治療
EP4126910A1 (en) 2020-04-01 2023-02-08 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
CN115667289A (zh) 2020-04-10 2023-01-31 索拉生物科学有限公司 用于治疗蛋白聚集障碍的组合物和方法
CN116249712A (zh) 2020-04-15 2023-06-09 沃雅戈治疗公司 Tau结合化合物
CN115997011A (zh) * 2020-04-20 2023-04-21 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
JP2023523429A (ja) 2020-04-22 2023-06-05 インダプタ セラピューティクス インコーポレイテッド ナチュラルキラー（ｎｋ）細胞組成物およびそれを生成させるための方法
BR112022021604A2 (pt) 2020-04-28 2022-12-06 Sola Biosciences Llc Composições e métodos para tratamento de proteinopatias associadas à tdp-43
US20230151389A1 (en) 2020-05-05 2023-05-18 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
BR112022021786A2 (pt) 2020-05-12 2023-01-17 Univ Pennsylvania Composições úteis em tratamento de doença de krabbe
TW202208406A (zh) 2020-05-13 2022-03-01 美商阿科奧斯公司 用於治療ｋｃｎｑ４相關性聽力損失之組成物及方法
AU2021273447A1 (en) 2020-05-13 2022-12-08 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
KR20230041965A (ko) 2020-05-13 2023-03-27 아카우오스, 인크. Slc26a4-연관 청력손실을 치료하기 위한 조성물 및 방법
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
CN116096737A (zh) 2020-06-05 2023-05-09 索拉生物科学有限公司 用于治疗突触核蛋白病的组合物和方法
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
EP4168052A2 (en) 2020-06-19 2023-04-26 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
EP4176064A1 (en) * 2020-07-03 2023-05-10 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
EP4179083A4 (en) * 2020-07-07 2024-08-28 The Board Of Regents Of The University Of Texas System Adeno-associated virus vector for dwarf open reading frame
CA3184923A1 (en) 2020-07-10 2022-01-13 William LOSTAL A novel muscle-specific promoter
JP2023540429A (ja) 2020-07-10 2023-09-25 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） てんかんを治療するための方法及び組成物
JP2023534037A (ja) 2020-07-13 2023-08-07 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア シャルコー・マリー・トゥース病の治療に有用な組成物
WO2022015916A1 (en) 2020-07-15 2022-01-20 University Of Rochester Targeted rna cleavage with dcasl3-rnase fusion proteins
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
KR20230093241A (ko) 2020-07-27 2023-06-27 보이저 테라퓨틱스, 인크. 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법
CN116121274B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
MX2023001998A (es) 2020-08-17 2023-05-04 Massachusetts Inst Technology Enfoques de terapia con el gen shank3.
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
EP4200429A1 (en) 2020-08-24 2023-06-28 The Trustees of The University of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
EP4204014A1 (en) 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
CA3194019A1 (en) 2020-09-04 2022-03-10 National University Corporation Kobe University Miniaturized cytidine deaminase-containing complex for modifying double-stranded dna
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
WO2022060915A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized lanadelumab and administration thereof
US20230383278A1 (en) 2020-09-18 2023-11-30 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
CA3193697A1 (en) 2020-10-07 2022-04-14 Joseph Bruder Adeno-associated viruses for ocular delivery of gene therapy
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
MX2023004005A (es) 2020-10-07 2023-06-06 Regenxbio Inc Formulaciones para administración supracoroidea tales como formulaciones de gel.
EP4225270A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
JP2023544803A (ja) 2020-10-07 2023-10-25 レジェンクスバイオ インコーポレーテッド Ｃｌｎ２疾患の眼症状に対する遺伝子療法
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
US20230391864A1 (en) 2020-10-28 2023-12-07 Regenxbio, Inc. Vectorized anti-tnf-alpha antibodies for ocular indications
TW202233844A (zh) * 2020-10-29 2022-09-01 賓州大學委員會 Aav衣殼及含有其之組成物
CN116457373A (zh) 2020-10-29 2023-07-18 再生生物股份有限公司 用于眼部适应症的载体化TNF-α拮抗剂
EP4236974A2 (en) 2020-10-29 2023-09-06 RegenxBio Inc. Vectorized factor xii antibodies and administration thereof
MX2023005041A (es) 2020-11-02 2023-05-17 Biomarin Pharm Inc Proceso para enriquecer virus adenoasociado.
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
JP2023551903A (ja) 2020-12-01 2023-12-13 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 組織特異的標的化モチーフを有する新規組成物及びそれを含有する組成物
AU2021392642A1 (en) 2020-12-01 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
PH12023551518A1 (en) 2020-12-01 2024-05-13 Akouos Inc Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
US20240167054A1 (en) 2020-12-16 2024-05-23 Regenxbio Inc. Method of producing a recombinant virus particle
JP2024503234A (ja) 2020-12-23 2024-01-25 ファイザー・インク 親和性クロマトグラフィーによるａａｖベクターの精製方法
EP4271419A1 (en) 2020-12-29 2023-11-08 Akouos, Inc. Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
TW202241943A (zh) 2020-12-29 2022-11-01 美商銳進科斯生物股份有限公司 Tau特異性抗體基因療法組合物、方法及其用途
WO2022147481A1 (en) 2020-12-30 2022-07-07 Ansun Biopharma Inc. Combination therapy of an oncolytic virus delivering a foreign antigen and an engineered immune cell expressing a chimeric receptor targeting the foreign antigen
EP4277933A4 (en) 2021-01-14 2024-12-11 Senti Biosciences, Inc. SECRETABLE PAYLOAD REGULATION
JP2024503895A (ja) 2021-01-21 2024-01-29 リジェネクスバイオ インコーポレイテッド 組換えポリペプチド及びウイルスの生成の改善
EP4281551A4 (en) * 2021-01-25 2025-06-04 Trames Bio, Inc. ADENO-ASSOCIATED VIRUS CAPSIDS AND MANIPULATED LIGAND-GATED ION CHANNELS FOR THE TREATMENT OF FOCAL EPILEPSY AND NEUROPATHIC PAIN
BR112023015303A2 (pt) 2021-02-01 2023-11-14 Regenxbio Inc Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito
WO2022169774A1 (en) * 2021-02-02 2022-08-11 University Of Massachusetts Inverted terminal repeats from aav serotypes 8 and rh.39 in gene therapy vectors
EP4288555A1 (en) 2021-02-05 2023-12-13 Maze Therapeutics, Inc. Vectors comprising stuffer polynucleotide sequences
AU2022218706A1 (en) * 2021-02-09 2023-09-28 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
US20240117328A1 (en) 2021-02-10 2024-04-11 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
KR20230150836A (ko) 2021-02-26 2023-10-31 다케다 야쿠힌 고교 가부시키가이샤 파브리병 치료를 위한 조성물 및 방법
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4301768A2 (en) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240191213A1 (en) 2021-03-19 2024-06-13 Adrenas Therapeutics, Inc. Gene therapies for 21-hydroxylase deficiency
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
CA3218333A1 (en) 2021-04-05 2022-10-13 Solid Biosciences Inc. Recombinant herpesvirales vector
KR20230170022A (ko) 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
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
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
EP4329820A1 (en) 2021-04-26 2024-03-06 Alexion Pharma International Operations Limited Adeno-associated viral vector capsids with improved tissue tropism
CA3216744A1 (en) 2021-04-26 2022-11-03 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2022235614A2 (en) 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
WO2022241030A1 (en) 2021-05-11 2022-11-17 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
CN117813314A (zh) * 2021-06-08 2024-04-02 Nf2治疗公司 用于治疗神经纤维瘤疾患的组合物和方法
WO2022266452A1 (en) * 2021-06-18 2022-12-22 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
KR20240023638A (ko) 2021-06-22 2024-02-22 화이자 인코포레이티드 곤충 세포에서의 아데노-연관 바이러스 벡터의 생산
US20240287493A1 (en) * 2021-06-24 2024-08-29 University Of Utah Research Foundation Compositions and methods for treating pgm1 deficiency
WO2023278811A1 (en) 2021-07-01 2023-01-05 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
JP2024523707A (ja) 2021-07-08 2024-06-28 テナヤ セラピューティクス， インコーポレイテッド 遺伝子治療のための最適化された発現カセット
US20240409955A1 (en) 2021-07-19 2024-12-12 New York University Auf1 combination therapies for treatment of muscle degenerative disease
US20240366788A1 (en) * 2021-07-23 2024-11-07 Duke University Adeno-associated virus compositions and methods of use thereof
EP4378472A4 (en) 2021-07-30 2025-04-02 Toru Miyazaki THERAPEUTIC AGENT AGAINST ISCHEMIC DISEASES
AU2022325955A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
AU2022325231A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
WO2023019229A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified primary cells for allogeneic cell therapy
EP4384544A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
AR126870A1 (es) * 2021-08-25 2023-11-22 Canbridge Pharmaceuticals Inc PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GALACTOSIDASA Y SU USO PARA TRATAR LA ENFERMEDAD DE FABRY
JP2024535121A (ja) * 2021-08-25 2024-09-26 ケンブリッジ ファーマシューティカルズ，インコーポレーテッド 肝臓指向性カプシドタンパク質及び酸性α－グルコシダーゼを含むＡＡＶ粒子並びにポンペ病を治療するための使用
CA3229838A1 (en) 2021-08-31 2023-03-09 Scout Bio, Inc. Antigen-binding molecules and uses thereof
US20250223376A1 (en) 2021-09-20 2025-07-10 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
AU2022356427A1 (en) 2021-09-30 2024-05-09 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
US20240384298A1 (en) 2021-10-02 2024-11-21 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
US20230151372A1 (en) * 2021-10-07 2023-05-18 Academia Sinica Composition and Method of Treatment for Heart Protection and Regeneration
US20250179518A1 (en) 2021-10-07 2025-06-05 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
EP4413024A2 (en) 2021-10-08 2024-08-14 SOLA Biosciences LLC Compositions and methods for the treatment of proteopathies
WO2023060248A1 (en) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions and methods for the treatment of p53-mediated cancers
AU2022358779A1 (en) 2021-10-08 2024-04-18 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
AU2022366947A1 (en) 2021-10-12 2024-05-02 Bridgebio Gene Therapy Llc Methods and compositions for treating leukodystrophies
CN118660710A (zh) 2021-10-20 2024-09-17 罗切斯特大学 用于年龄相关白质损失的竞争性治疗的分离神经胶质祖细胞
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023081633A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
CA3235632A1 (en) 2021-11-02 2023-05-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
US20250011372A1 (en) 2021-11-17 2025-01-09 Voyager Thrapeutics, Inc. Aav capsid variants and uses thereof
EP4442830A4 (en) 2021-11-29 2025-04-30 Shanghai Regenelead Therapies Co., Ltd. AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
US20230279433A1 (en) * 2021-12-15 2023-09-07 Oxford Biomedica Solutions Llc Methods and compositions for the production of adeno-associated virus
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
EP4466028A1 (en) 2022-01-17 2024-11-27 Dinaqor AG Gene therapy composition and treatment for dystrophin-related cardiomyopathy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US20250144243A1 (en) 2022-01-25 2025-05-08 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
EP4473097A1 (en) 2022-02-02 2024-12-11 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
CN118679182A (zh) 2022-02-02 2024-09-20 阿库斯股份有限公司 用于治疗前庭神经鞘瘤相关症状的抗vegf抗体构建体和相关方法
IL314156A (en) 2022-02-08 2024-09-01 Voyager Therapeutics Inc Adeno-associated virus capsid variants and their uses
CA3244101A1 (en) 2022-02-17 2023-08-24 Sana Biotechnology, Inc. MODIFIED CD47 PROTEINS AND THEIR USES
CN118660913A (zh) 2022-02-21 2024-09-17 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
EP4482970A1 (en) 2022-02-23 2025-01-01 Massachusetts Institute of Technology Methods for upregulating shank3 expression
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
TW202346590A (zh) 2022-03-13 2023-12-01 美商銳進科斯生物股份有限公司 經修飾之肌肉特異性啟動子
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
US20250213730A1 (en) 2022-04-01 2025-07-03 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
AU2023250660A1 (en) 2022-04-06 2024-10-24 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
CN119317645A (zh) 2022-04-06 2025-01-14 宾夕法尼亚州大学信托人 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
TW202404993A (zh) 2022-04-11 2024-02-01 美商特納亞治療股份有限公司 具經工程化蛋白殼之腺相關病毒
JP2025513835A (ja) 2022-04-11 2025-04-30 サントル ナショナル ドゥ ラ ルシェルシュ シアンティフィック 化学修飾型アデノ随伴ウイルス
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
EP4507741A1 (en) 2022-04-14 2025-02-19 RegenxBio Inc. Gene therapy for treating an ocular disease
US20250277004A1 (en) 2022-04-18 2025-09-04 Regenxbio Inc. Hybrid aav capsids
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
EP4518972A2 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2023215807A1 (en) 2022-05-03 2023-11-09 Regenxbio Inc. VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
US20250295809A1 (en) 2022-05-13 2025-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
CA3256727A1 (en) * 2022-05-24 2023-11-30 Kate Therapeutics, Inc. COMPOSITIONS FOR THE TREATMENT OF XLMTM
IL316719A (en) 2022-06-02 2024-12-01 Voyager Therapeutics Inc Adeno-associated virus capsid variants and their uses
WO2023240158A2 (en) * 2022-06-07 2023-12-14 Oregon Health & Science University Mutant adeno-associated virus (aav) capsids
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
JP2025524469A (ja) 2022-06-24 2025-07-30 チューン セラピューティクス インコーポレイテッド 標的を定めた遺伝子抑制を介して低密度リポタンパク質を低減するための組成物、系、及び方法
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
TW202417467A (zh) 2022-06-28 2024-05-01 美商航海家醫療公司 Ａａｖ蛋白殼變異體及其用途
KR20250060188A (ko) 2022-06-30 2025-05-07 인답타 세라뷰틱스 인코포레이티드 조작된 자연 살해(nk) 세포 및 항체 요법의 조합 및 관련 방법
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4558149A1 (en) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
EP4565597A2 (en) 2022-08-03 2025-06-11 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
EP4577663A2 (en) 2022-08-24 2025-07-02 RegenxBio Inc. Recombinant adeno-associated viruses and uses thereof
EP4577249A1 (en) 2022-08-25 2025-07-02 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
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
IL319714A (en) 2022-09-22 2025-05-01 Dinaqor Ag Treating cardiac myopathy with AAV gene therapy vectors
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
CN120225686A (zh) * 2022-10-04 2025-06-27 赛仁生物技术公司 用于重组病毒载体生产的修饰的CpG二核苷酸
WO2024081746A2 (en) 2022-10-11 2024-04-18 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
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
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
EP4649160A1 (en) 2023-01-12 2025-11-19 Nantes Université Chemically-modified adeno-associated virus
WO2024163979A2 (en) * 2023-02-02 2024-08-08 Codexis, Inc. Engineered aav polypeptides
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
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
AU2024221161A1 (en) 2023-02-17 2025-08-28 Biogen Ma Inc. Rgd-containing peptides for delivering payloads
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
KR20250158036A (ko) 2023-03-17 2025-11-05 유니버시티 오브 로체스터 리보자임-매개 rna 조립 및 발현
WO2024206226A1 (en) * 2023-03-25 2024-10-03 Duke University Compositions comprising kidney-tropic aavs and methods of use thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024220389A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav2 variants and uses thereof
WO2024220463A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav5 capsid with non-canonical amino acid incorporation and uses thereof
AU2024258816A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Generation of adeno-associated virus capsid libraries for insect cells
AU2024258047A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CN121079310A (zh) 2023-05-03 2025-12-05 沃雅戈治疗公司 用于治疗与cdkl5缺乏相关的病症的组合物及方法
WO2024229167A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
WO2024229302A1 (en) 2023-05-03 2024-11-07 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024229125A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
TW202444917A (zh) 2023-05-04 2024-11-16 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2024233422A1 (en) 2023-05-05 2024-11-14 Massachusetts Institute Of Technology Shank3 gene therapy approaches
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
WO2024241176A1 (en) 2023-05-24 2024-11-28 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
WO2025017168A1 (en) 2023-07-19 2025-01-23 Genethon Novel optimized utrophin micro-genes
WO2025017169A1 (en) 2023-07-20 2025-01-23 Genethon Novel mididystrophins
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025042711A1 (en) 2023-08-18 2025-02-27 Eli Lilly And Company Engineered transferrin receptor binding peptides as well as methods of making and using the same
WO2025072604A1 (en) 2023-09-28 2025-04-03 University Of Rochester Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1)
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025080780A1 (en) 2023-10-10 2025-04-17 University Of Rochester Delivery and expression of prime editing crispr systems
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025096967A1 (en) 2023-11-02 2025-05-08 Biogen Ma Inc. Redirection of aav capsids for central nervous system targeting
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
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
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
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
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
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
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
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
WO2025171001A1 (en) 2024-02-06 2025-08-14 Regeneron Pharmaceuticals, Inc. Compositions and methods for improved production of aav viral vectors
WO2025188625A1 (en) 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025207948A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids for targeting human transferrin receptor
WO2025207932A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids comprising anti-transferrin antibody domains
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
GB202407038D0 (en) 2024-05-17 2024-07-03 Axovia Therapeutics Ltd Novel Gene Therapy
WO2025242657A1 (en) 2024-05-21 2025-11-27 Shape Biopharmaceuticals Ag Lipopeptide building blocks and aggregates
WO2025250457A1 (en) 2024-05-28 2025-12-04 University Of Rochester Enhanced brain transduction by gene therapeutics

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
WO1985001961A1 (en) 1983-10-28 1985-05-09 Genetics Institute Production of factor viii and related products
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
IL74909A (en) 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IE69026B1 (en) 1987-06-12 1996-08-07 Immuno Ag Novel proteins with factor VIII activity process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
JP2865861B2 (ja) 1989-11-17 1999-03-08 ノボ ノルディスク アクティーゼルスカブ 第▲ｖｉｉｉ▼：ｃ因子活性を有するタンパク質複合体およびその製法
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
DE69433925T2 (de) 1993-06-10 2005-07-28 Bayer Corp. Vektoren und Zelllinien von Säugetieren mit erhöhter Produktivität
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
AU6486196A (en) 1995-07-11 1997-02-10 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
US20020088014A1 (en) * 1996-05-31 2002-07-04 Xiangming Fang Minimal adenovirus mediated recombinant vaccine
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
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
CA2264483C (en) 1996-09-06 2011-03-22 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
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
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
CA2304168A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
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
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
US6962815B2 (en) * 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
CN1236057C (zh) * 2001-10-18 2006-01-11 华中科技大学同济医学院附属同济医院 表达人类激肽释放酶的重组腺相关病毒及其制法和用途
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
EP2573170B1 (en) * 2001-12-17 2017-12-20 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
US20060292117A1 (en) * 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
CA2526120A1 (en) * 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
ES2874298T3 (es) * 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
ES2428218T3 (es) * 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
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
SG10201601110VA (en) * 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
PL3198018T3 (pl) * 2014-09-24 2021-07-19 City Of Hope Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby
WO2018160582A1 (en) 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
WO2018160573A1 (en) * 2017-02-28 2018-09-07 The Trustees Of The University Of Pennsylvania Influenza vaccines based on aav vectors

Also Published As

Publication number Publication date
JP5797397B2 (ja) 2015-10-21
US20170028082A1 (en) 2017-02-02
US9737618B2 (en) 2017-08-22
EP2292780B1 (en) 2017-08-23
AU2008203421A1 (en) 2008-08-21
CN102199626A (zh) 2011-09-28
EP3211085A1 (en) 2017-08-30
US20170159027A1 (en) 2017-06-08
CA2939103A1 (en) 2005-04-14
JP6335141B2 (ja) 2018-05-30
CY2021024I2 (el) 2021-12-31
CY1119833T1 (el) 2018-06-27
DK2292780T3 (en) 2017-12-04
US20220023441A1 (en) 2022-01-27
US10695441B2 (en) 2020-06-30
JP5911069B2 (ja) 2016-04-27
JP5431516B2 (ja) 2014-03-05
CA3072423A1 (en) 2005-04-14
CN102212558B (zh) 2016-08-03
EP2298926A1 (en) 2011-03-23
JP6516713B2 (ja) 2019-05-22
CN102220376A (zh) 2011-10-19
CA2939103C (en) 2020-03-24
CN1856576B (zh) 2011-05-04
US20190054188A1 (en) 2019-02-21
US20190015527A1 (en) 2019-01-17
EP2292779A3 (en) 2011-03-30
EP1668143A2 (en) 2006-06-14
HUS2100033I1 (hu) 2021-09-28
CN1856576A (zh) 2006-11-01
JP2012175974A (ja) 2012-09-13
US20210299274A1 (en) 2021-09-30
FR21C1037I1 (fr) 2021-10-15
EP3910063A1 (en) 2021-11-17
HUE054805T2 (hu) 2021-09-28
US7906111B2 (en) 2011-03-15
US20110236353A1 (en) 2011-09-29
JP5054975B2 (ja) 2012-10-24
JP2007507223A (ja) 2007-03-29
CY1124586T1 (el) 2021-12-31
AU2004278684A1 (en) 2005-04-14
EP2292780A2 (en) 2011-03-09
US20210170050A1 (en) 2021-06-10
PT3211085T (pt) 2021-06-17
ES2648241T3 (es) 2017-12-29
EP2292779A2 (en) 2011-03-09
ES2411479T3 (es) 2013-07-05
JP6673963B2 (ja) 2020-04-01
AU2004278684B2 (en) 2011-05-12
US10265417B2 (en) 2019-04-23
PT2292780T (pt) 2017-11-28
CN102212558A (zh) 2011-10-12
BE2021C533I2 (enExample) 2024-01-12
HUE033158T2 (en) 2017-11-28
EP3211085B1 (en) 2021-03-31
WO2005033321A3 (en) 2005-06-30
EP2292779B1 (en) 2016-11-16
AU2008203421B2 (en) 2011-08-25
SI2292780T1 (sl) 2017-12-29
SI3211085T1 (sl) 2021-12-31
CA2537793A1 (en) 2005-04-14
DK2345731T3 (en) 2016-01-25
US20070036760A1 (en) 2007-02-15
CN102174574B (zh) 2016-08-03
US10722598B2 (en) 2020-07-28
PL2292780T3 (pl) 2018-05-30
US11357868B2 (en) 2022-06-14
WO2005033321A2 (en) 2005-04-14
DK2292779T3 (en) 2017-02-27
US11357867B2 (en) 2022-06-14
ES2616016T3 (es) 2017-06-09
CN102174574A (zh) 2011-09-07
US20200155704A1 (en) 2020-05-21
DK3211085T3 (da) 2021-06-21
CA2537793C (en) 2013-07-23
JP2011101646A (ja) 2011-05-26
US20150315612A1 (en) 2015-11-05
NZ545628A (en) 2009-04-30
EP2292780A3 (en) 2011-03-30
JP2015163083A (ja) 2015-09-10
JP2018108113A (ja) 2018-07-12
JP2020054373A (ja) 2020-04-09
HUE035567T2 (en) 2018-05-28
CN102199626B (zh) 2015-06-24
PL3211085T3 (pl) 2021-11-15
JP2014012022A (ja) 2014-01-23
EP2345731B1 (en) 2015-10-21
US10485883B2 (en) 2019-11-26
US20190022249A1 (en) 2019-01-24
US10973928B2 (en) 2021-04-13
US20220025400A1 (en) 2022-01-27
JP2017060483A (ja) 2017-03-30
CY2021024I1 (el) 2021-12-31
EP1668143B1 (en) 2013-03-20
ES2555612T3 (es) 2016-01-05
EP2345731A1 (en) 2011-07-20
JP2022173573A (ja) 2022-11-18

Similar Documents

Publication Publication Date Title
US11357867B2 (en) Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
HK40064515A (en) Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor