PT1453547T - Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas - Google Patents

Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas

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Publication number
PT1453547T
PT1453547T PT2795539T PT02795539T PT1453547T PT 1453547 T PT1453547 T PT 1453547T PT 2795539 T PT2795539 T PT 2795539T PT 02795539 T PT02795539 T PT 02795539T PT 1453547 T PT1453547 T PT 1453547T
Authority
PT
Portugal
Prior art keywords
serotype
aav
adeno
sequences
associated virus
Prior art date
Application number
PT2795539T
Other languages
English (en)
Inventor
M Wilson James
Gao Guangping
Alvira Mauricio
Original Assignee
Univ Pennsylvania
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=27407443&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PT1453547(T) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of PT1453547T publication Critical patent/PT1453547T/pt

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • 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)
    • C12Y304/21021—Coagulation factor VIIa (3.4.21.21)
    • 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)
    • 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
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    • 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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • 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
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    • 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
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    • 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
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    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
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  • Dermatology (AREA)
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PT2795539T 2001-12-17 2002-11-12 Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas PT1453547T (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US34115101P 2001-12-17 2001-12-17
US37713302P 2002-05-01 2002-05-01
US38612202P 2002-06-05 2002-06-05

Publications (1)

Publication Number Publication Date
PT1453547T true PT1453547T (pt) 2016-12-28

Family

ID=27407443

Family Applications (2)

Application Number Title Priority Date Filing Date
PT10189725T PT2359869T (pt) 2001-12-17 2002-11-12 Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
PT2795539T PT1453547T (pt) 2001-12-17 2002-11-12 Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PT10189725T PT2359869T (pt) 2001-12-17 2002-11-12 Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas

Country Status (11)

Country Link
US (15) US7282199B2 (pt)
EP (3) EP2359869B1 (pt)
JP (2) JP4810062B2 (pt)
AU (1) AU2002360291A1 (pt)
CA (1) CA2469785C (pt)
CY (2) CY1120162T1 (pt)
DK (2) DK2359869T3 (pt)
ES (3) ES2717377T3 (pt)
FI (1) FI3517134T3 (pt)
PT (2) PT2359869T (pt)
WO (1) WO2003052051A2 (pt)

Families Citing this family (506)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0932694A2 (en) * 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
DE69939169D1 (de) * 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
CA2455499C (en) 2001-08-08 2011-06-21 The Trustees Of The University Of Pennsylvania Method for purification of viral vectors having proteins which bind sialic acid
AU2013202568B2 (en) * 2001-11-13 2015-09-17 The Trustees Of The University Of Pennsylvania Adeno-associated virus rh.8 (AAVrh.8) sequences and recombinant AAVs comprising same
CN103555677B (zh) 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
PT2573170T (pt) * 2001-12-17 2018-03-26 Univ Pennsylvania Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações
WO2003052051A2 (en) * 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
US7419817B2 (en) * 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
AU2003304396A1 (en) * 2002-06-28 2005-02-25 University Of Florida Research Foundation, Inc. Raav compositions and methods for delivery of human factor vii polypeptides and treatment of hemophilia a
WO2004108922A2 (en) * 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
WO2004110483A1 (en) * 2003-05-31 2004-12-23 Yiyou Chen Method and composition of a novel vaccine design for the prevention and treatment of sars
US7186699B2 (en) * 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
DK3211085T3 (da) 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
WO2005049850A2 (en) 2003-11-14 2005-06-02 University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
WO2005077333A2 (en) * 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
ES2361000T3 (es) 2004-04-28 2011-06-13 The Trustees Of The University Of Pennsylvania Suministro secuencial de moléculas inmunogénicas mediante administraciones de un adenovirus y de un virus adeno-asociado.
EP1742657B1 (en) 2004-04-28 2013-11-06 The Trustees of The University of Pennsylvania Immunization regimen with e4-deleted adenovirus prime and e1-deleted adenovirus boost
CA2567741A1 (en) * 2004-05-25 2006-03-30 Chimeracore, Inc. Self-assembling nanoparticle drug delivery system
US20080188431A1 (en) * 2004-09-08 2008-08-07 Chiorini John A Transcytosis of Adeno-Associated Viruses
WO2006119432A2 (en) * 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
BRPI0611540A2 (pt) * 2005-05-02 2010-09-21 Genzyme Corp terapia de gene para distúrbios de espinha dorsal
PT2420256T (pt) 2005-05-02 2016-09-13 Genzyme Corp Terapia genética para distúrbios neurometabólicos
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2018156B1 (en) * 2006-04-07 2010-03-17 Chimeros, Inc. Compositions and methods for treating b- cell malignancies
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
CA2683063A1 (en) * 2007-04-09 2008-10-16 Chimeros, Inc. Self-assembling nanoparticle drug delivery system
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8263601B2 (en) 2009-02-27 2012-09-11 Concert Pharmaceuticals, Inc. Deuterium substituted xanthine derivatives
WO2010120874A2 (en) 2009-04-14 2010-10-21 Chimeros, Inc. Chimeric therapeutics, compositions, and methods for using same
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011032100A1 (en) 2009-09-11 2011-03-17 Government Of The U.S.A., As Represented By The Secretary, Department Of Health And Human Services Inhibitors of kshv vil6 and human il6
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
SG10201908848RA (en) 2010-03-29 2019-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US10392632B2 (en) 2011-02-14 2019-08-27 The Children's Hospital Of Philadelphia AAV8 vector with enhanced functional activity and methods of use thereof
EP2699688A1 (en) 2011-04-20 2014-02-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
US9987354B2 (en) 2011-04-29 2018-06-05 Selecta Biosciences, Inc. Tolerogenic synthetic nanocarriers for antigen-specific deletion of T effector cells
JP2014527072A (ja) 2011-09-09 2014-10-09 バイオメド リアルティー， エル．ピー． ウイルスタンパク質の集合を制御するための方法および組成物
MX354516B (es) 2011-10-27 2018-03-08 Wellstat Ophthalmics Corp Vectores que codifican el factor de viabilidad de conos derivado de bastones.
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
JP6562632B2 (ja) * 2012-02-14 2019-08-21 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 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現
JP5926580B2 (ja) 2012-03-01 2016-05-25 ユー・ディー・シー アイルランド リミテッド 有機電界発光素子、有機電界発光素子用材料、並びに、該素子を用いた発光装置、表示装置、照明装置及び該素子に用いられる化合物
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
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
EP2900686B1 (en) * 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
DK2956477T4 (da) 2013-02-15 2024-04-15 Bioverativ Therapeutics Inc Optimeret faktor viii-gen
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)
SG10201707319UA (en) * 2013-03-15 2017-10-30 Univ Pennsylvania Compositions and methods for treating mpsi
EP2986632B1 (en) 2013-04-20 2018-09-05 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
CN110251675A (zh) 2013-05-03 2019-09-20 西莱克塔生物科技公司 降低或预防响应于非变应原性抗原的过敏反应的致耐受性合成纳米载体
CA3209883A1 (en) * 2013-07-22 2015-01-29 The Children's Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
DE212015000061U1 (de) 2014-02-11 2017-09-03 The Regents Of The University Of Colorado, A Body Corporate CRISPR-ermöglichtes Multiplex Genom Engineering
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015138357A2 (en) 2014-03-09 2015-09-17 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of otc deficency
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
US11555059B2 (en) 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
DK3142750T3 (da) 2014-05-13 2020-09-14 Univ Pennsylvania Sammensætninger omfattende aav, som udtrykker dobbelt-antistofkonstrukter og anvendelser deraf
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
EP2975116B1 (en) * 2014-07-16 2019-08-21 Max-Delbrück-Centrum für Molekulare Medizin Tgif2-induced reprogramming of hepatic cells to pancreatic progenitor cells and medical uses thereof
JP6986444B2 (ja) 2014-08-09 2021-12-22 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｄｍｄ遺伝子のエクソン５内の内部リボソーム進入部位を活性化するための方法及び材料
WO2016037161A2 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
CA2960912A1 (en) * 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
EP3795580A1 (en) * 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CN107249646B (zh) 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
WO2016115503A1 (en) 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
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
EP3626274B1 (en) 2015-04-16 2025-01-22 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
CA3021949C (en) 2015-04-24 2023-10-17 University Of Massachusetts Modified aav constructs and uses thereof
EP3288594B1 (en) 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
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
HUE068603T2 (hu) 2015-06-23 2025-01-28 Childrens Hospital Philadelphia Módosított IX-es faktor, valamint készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
AU2016302335B2 (en) 2015-08-06 2022-08-04 The Trustees Of The University Of Pennsylvania GLP-1 and use thereof in compositions for treating metabolic diseases
CN108351350B (zh) 2015-08-25 2022-02-18 杜克大学 使用rna指导型内切核酸酶改善基因组工程特异性的组合物和方法
CN108137664B (zh) 2015-08-31 2021-11-26 宾夕法尼亚州大学信托人 用于治疗伴侣动物的aav-epo
US10988519B2 (en) 2015-09-24 2021-04-27 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
US11970710B2 (en) 2015-10-13 2024-04-30 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
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
CA3003435A1 (en) 2015-10-28 2017-05-04 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
EP3374388A1 (en) 2015-11-13 2018-09-19 Baxalta Incorporated Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11201804070XA (en) 2015-11-13 2018-06-28 Baxalta Inc Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
US11617783B2 (en) 2015-11-16 2023-04-04 Research Institute At Nationwide Children's Hospital Repairing a mutant human titin gene using CRISPR technology
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
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
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
EP3387118B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
JP7061067B2 (ja) 2015-12-14 2022-04-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア クリグラー・ナジャー症候群の処置のための組成物
JP7057281B2 (ja) 2015-12-14 2022-04-19 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 眼疾患のための遺伝子療法
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
WO2017136500A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
WO2017143100A1 (en) 2016-02-16 2017-08-24 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
KR102453187B1 (ko) * 2016-04-02 2022-10-07 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 조직 특이적 발현을 위한 변형된 u6 프로모터 시스템
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
AU2017248656B2 (en) * 2016-04-15 2023-07-27 The Trustees Of The University Of Pennsylvania Novel AAV8 mutant capsids and compositions containing same
WO2017180857A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of 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
JP7153562B2 (ja) 2016-04-15 2022-10-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するためのマイクロｒｎａ－２９のアデノ随伴ウイルスベクター送達
IL322403A (en) 2016-04-15 2025-09-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN116333057A (zh) 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics, Inc. composições e métodos de tratamento da doença de huntington
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
EP3474849B1 (en) * 2016-06-27 2025-05-21 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
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
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
US20210283223A1 (en) * 2016-08-15 2021-09-16 Beth Israel Deaconess Medical Center, Inc. Novel therapy to achieve glycemic control
AU2017313917B2 (en) 2016-08-18 2023-12-21 The Regents Of The University Of California CRISPR-Cas genome engineering via a modular AAV delivery system
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
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
MX2019007873A (es) * 2016-12-30 2019-11-18 Univ Pennsylvania Terapia génica para tratar la enfermedad de wilson.
EP3562494A4 (en) * 2016-12-30 2020-08-19 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS
MX2019008143A (es) 2017-01-07 2020-01-13 Selecta Biosciences Inc Dosificación sistemática de inmunosupresores acoplados a nanoportadores sintéticos.
KR20250022877A (ko) 2017-01-31 2025-02-17 리젠엑스바이오 인크. 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료
EP3576760A2 (en) 2017-02-01 2019-12-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
KR20190118163A (ko) 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
SI3589730T1 (sl) 2017-02-28 2024-04-30 The Trustees Of The University Of Pennsylvania Adeno-povezan virus (AAV) clade F vektor in njihova uporaba
US10786568B2 (en) 2017-02-28 2020-09-29 The Trustees Of The University Of Pennsylvania AAV mediated influenza vaccines
CN119752819A (zh) 2017-03-01 2025-04-04 宾夕法尼亚州立大学托管会 用于眼部病症的基因疗法
ES3036482T3 (en) 2017-03-11 2025-09-19 Cartesian Therapeutics Inc Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
DK3596222T5 (da) 2017-03-17 2024-09-02 Res Inst Nationwide Childrens Hospital Adenoassocieret virusvektorfremføring af muskelspecifik mikro-dystrophin til behandling af muskeldystrofi
WO2019012336A2 (en) 2017-03-17 2019-01-17 Newcastle University ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
CN110506060B (zh) 2017-03-30 2024-05-07 昆士兰大学 嵌合分子及其用途
US11499154B2 (en) 2017-04-10 2022-11-15 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
WO2018191666A1 (en) 2017-04-14 2018-10-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells
MX2019012452A (es) 2017-04-21 2020-08-03 Prec Biosciences Inc Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9.
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11633504B2 (en) 2017-05-09 2023-04-25 Emory University Nucleic acids encoding clotting factor variants and their use
IL316926A (en) 2017-05-11 2025-01-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis
JP7235676B2 (ja) 2017-05-17 2023-03-08 ザ ジェネラル ホスピタル コーポレイション 結節性硬化症の遺伝子治療
KR102386890B1 (ko) 2017-05-22 2022-04-15 다케다 야쿠힌 고교 가부시키가이샤 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
IL306098A (en) 2017-05-24 2023-11-01 Univ Barcelona Autonoma Viral expression vectors containing the coding sequence for FIBROBLAST GROWTH FACTOR 21 (FGF21)
CA3098592A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
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
AU2018298133A1 (en) 2017-07-06 2020-01-23 The Trustees Of The University Of Pennsylvania AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
PL3648783T3 (pl) 2017-07-07 2024-11-18 Genethon Nowe polinukleotydy kodujące ludzkie białko fkrp
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
EP3687561A4 (en) 2017-09-01 2021-06-09 The Australian National University "immunoregulatory molecules and uses therefor"
BR112020005436B1 (pt) 2017-09-20 2022-08-02 4D Molecular Therapeutics Inc Proteína do capsídeo de variante do vírus adenoassociado, virion do aav recombinante (raav) infeccioso, composições, composições farmacêuticas e usos de virion de raav ou de composições farmacêuticas
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
JP7213238B2 (ja) 2017-10-18 2023-01-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
CA3079565A1 (en) 2017-10-18 2019-04-25 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap
CN111727201A (zh) 2017-10-18 2020-09-29 再生生物股份有限公司 完全人源翻译后修饰的抗体治疗剂
CN111433367A (zh) 2017-10-20 2020-07-17 全国儿童医院研究所 Nt-3基因疗法的方法和材料
CA3081106A1 (en) 2017-11-01 2019-05-09 Queen's University At Kingston Von willebrand factor proteins for treating bleeding disorders
FI3717636T3 (fi) 2017-11-27 2023-06-01 4D Molecular Therapeutics Inc Adenoassosioidun viruksen kapsidivariantteja ja käyttö angiogeneesin estämisessä
CN111989396A (zh) 2017-11-30 2020-11-24 宾夕法尼亚州大学信托人 用于iiib型粘多糖贮积病的基因疗法
JP7389744B2 (ja) 2017-11-30 2023-11-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉｉａ型のための遺伝子療法
BR112020012336A2 (pt) 2017-12-19 2021-03-30 Akouos, Inc. Administração de anticorpos terapêuticos mediada por aav para o ouvido interno
CN111601620A (zh) 2018-01-17 2020-08-28 阿瑞纳斯治疗公司 用于21-羟化酶缺乏症的腺相关病毒基因疗法
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
US20190256867A1 (en) * 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
US12178908B2 (en) 2018-02-26 2024-12-31 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
KR20200135433A (ko) * 2018-03-23 2020-12-02 유니버시티 오브 매사추세츠 골 장애 치료를 위한 유전자 치료제
US11608510B2 (en) 2018-03-30 2023-03-21 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
US12091435B2 (en) 2018-04-03 2024-09-17 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
MX2020010464A (es) * 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
US12460226B2 (en) 2018-04-16 2025-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
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
CA3098565A1 (en) 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
AU2019270900B2 (en) 2018-05-15 2026-03-12 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
EP4512900A3 (en) 2018-06-05 2025-04-02 LifeEDIT Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
KR102930150B1 (ko) 2018-06-14 2026-02-25 리젠엑스바이오 인크. 재조합 aav 생성을 위한 음이온 교환 크로마토그래피
EP4591888A3 (en) 2018-06-18 2026-01-21 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
US12391928B2 (en) 2018-06-29 2025-08-19 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
CN112512596B (zh) 2018-07-12 2025-12-12 火箭制药有限公司 治疗danon病的基因治疗载体
AU2019306194A1 (en) 2018-07-16 2021-02-04 Baxalta GmbH Gene therapy of hemophilia A using viral vectors encoding recombinant FVIII variants with increased expression
MX2021000638A (es) 2018-07-16 2021-06-23 Selecta Biosciences Inc Métodos y composiciones de construcciones y vectores de acidemia metilmalónica.
CA3106639A1 (en) 2018-07-16 2020-01-23 Selecta Biosciences, Inc. Methods and compositions of otc constructs and vectors
AU2019304569B2 (en) 2018-07-17 2023-07-06 Helixmith Co., Ltd Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms
MX2021000810A (es) 2018-07-24 2021-04-28 Voyager Therapeutics Inc Sistemas y metodos para producir formulaciones de terapia genetica.
EP3833745A1 (en) 2018-08-10 2021-06-16 REGENXBIO Inc. Scalable method for recombinant aav production
WO2020041634A1 (en) 2018-08-22 2020-02-27 Research Institute At Nationwide Children's Hospital Recombinant virus products and methods for inhibiting expression of dystrophia myotonica protein kinase and/or interfering with a trinucleotide repeat expansion in the 3' untranslated region of the dmpk gene
WO2020047268A1 (en) 2018-08-29 2020-03-05 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of mutant gars protein
KR20210102870A (ko) 2018-08-30 2021-08-20 테나야 테라퓨틱스, 인코포레이티드 미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP2022513318A (ja) 2018-10-01 2022-02-07 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
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
CN113166781A (zh) 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
CN112912518A (zh) 2018-10-15 2021-06-04 再生生物股份有限公司 用于测量复制缺陷型病毒载体和病毒的感染性的方法
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
CA3116334A1 (en) 2018-10-22 2020-04-30 University Of Rochester Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas9 fusion protein
IL312708A (en) 2018-12-27 2024-07-01 Lifeedit Therapeutics Inc Polypeptides useful for gene editing and methods of use
WO2020140007A1 (en) 2018-12-28 2020-07-02 University Of Rochester Gene therapy for best1 dominant mutations
CA3124963A1 (en) 2018-12-31 2020-07-09 Research Institute At Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
PL3906066T3 (pl) 2019-01-04 2024-09-02 Ultragenyx Pharmaceutical Inc. Konstrukty do terapii genowej do leczenia choroby wilsona
BR112021013715A2 (pt) 2019-01-14 2021-09-21 University Of Rochester Proteína de fusão, molécula de ácido nucleico, métodos para modular a clivagem, a poliadenilação, ou ambas, de um transcrito de rna, para visualizar rna nuclear e para diminuir o número de rna nuclear ou para clivar rna nuclear, e, uso da proteína de fusão
TWI851647B (zh) 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
EP3911354B8 (en) 2019-01-18 2024-04-03 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CN109628490A (zh) * 2019-01-26 2019-04-16 青岛农业大学 一种预防犬瘟热的shRNA重组腺相关病毒
KR20210130158A (ko) 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
BR112021016501A2 (pt) 2019-02-25 2021-10-26 Novartis Ag Composições e métodos para tratar distrofia cristalina de bietti
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CN113766935A (zh) 2019-02-26 2021-12-07 全国儿童医院研究所 β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
JP7637060B2 (ja) 2019-03-04 2025-02-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ａｋｔ経路を標的とする神経保護遺伝子療法
JP7698583B2 (ja) 2019-03-21 2025-06-25 ギンコ バイオワークス インコーポレイテッド 組換えアデノ随伴ウイルスベクター
JP7635139B2 (ja) 2019-03-25 2025-02-25 ジェネトン オーバーラップａａｖベクターを使用する大きいサイズのクアシジストロフィンの産生
SG11202110607WA (en) 2019-04-01 2021-10-28 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
DK3953483T3 (da) 2019-04-11 2023-12-18 Regenxbio Inc Fremgangsmåder til størrelseskromatografi til karakterisering af sammensætninger af rekombinant adeno-associeret virus
JP7630443B2 (ja) 2019-04-19 2025-02-17 レジェンクスバイオ インコーポレーテッド アデノ随伴ウイルスベクター製剤及び方法
WO2020219868A1 (en) 2019-04-24 2020-10-29 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
WO2020223205A1 (en) 2019-04-28 2020-11-05 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
US12516351B2 (en) 2019-04-29 2026-01-06 The Trustees Of The University Of Pennsylvania AAV capsids and compositions containing same
WO2020243261A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
AU2020287627A1 (en) 2019-06-04 2021-11-18 Selecta Biosciences, Inc. Formulations and doses of PEGylated uricase
EP3994253A1 (en) 2019-07-02 2022-05-11 M6P Therapeutics (Switzerland) LLC Vector compositions and methods of using same for treatment of lysosomal storage disorders
CA3137622A1 (en) 2019-07-10 2021-01-14 Masonic Medical Research Institute Vgll4 with ucp-1 cis-regulatory element and method of use thereof
US20220251567A1 (en) 2019-07-10 2022-08-11 Inserm (Institut National De La Santè Et De La Recherche Médicale) Methods for the treatment of epilepsy
EP3997226A1 (en) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
WO2021009805A1 (ja) * 2019-07-12 2021-01-21 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
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
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
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
JP2022544015A (ja) 2019-07-23 2022-10-17 ユニバーシティ オブ ロチェスター ＣＲＩＳＰＲ－Ｃａｓでの標的化されたＲＮＡ切断
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
SI4017871T1 (sl) 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
RU2751592C2 (ru) * 2019-08-22 2021-07-15 Общество С Ограниченной Ответственностью "Анабион" Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
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
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
TW202126284A (zh) 2019-09-30 2021-07-16 美商百歐維拉提夫治療公司 慢病毒載體配製物
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
US20240108669A1 (en) 2019-10-07 2024-04-04 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
WO2021072129A2 (en) 2019-10-08 2021-04-15 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
WO2021076600A1 (en) 2019-10-14 2021-04-22 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
IL292297A (en) 2019-10-17 2022-06-01 Stridebio Inc Adeno-associated viral vectors for treatment of niemann-pick disease type c
EP4045662A1 (en) 2019-10-18 2022-08-24 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
WO2021092300A1 (en) * 2019-11-08 2021-05-14 President And Fellows Of Harvard College Viral capsid polypeptides
US20230016983A1 (en) 2019-11-19 2023-01-19 lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) Antisense oligonucleotides and thier use for the treatment of cancer
CN115023242B (zh) * 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体
US20230211018A1 (en) 2019-11-22 2023-07-06 Research Institute At Nationwide Children's Hospital Materials and methods for treatment of disorders associated with the ighmbp2 gene
EP4065596A2 (en) 2019-11-28 2022-10-05 RegenxBio Inc. Microdystrophin gene therapy constructs and uses thereof
IL293684A (en) 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
CA3165469A1 (en) 2019-12-20 2021-06-24 Research Institute At Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
KR20220128632A (ko) 2019-12-31 2022-09-21 스완바이오 테라퓨틱스 리미티드 개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도
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
BR112022014563A2 (pt) 2020-01-22 2022-09-13 Regenxbio Inc Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i)
EP4097238A1 (en) 2020-01-29 2022-12-07 REGENXBIO Inc. Treatment of mucopolysaccharidosis iva
EP4096631A1 (en) 2020-01-29 2022-12-07 RegenxBio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
US20230078498A1 (en) 2020-02-07 2023-03-16 University Of Rochester Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis
US20230073250A1 (en) 2020-02-07 2023-03-09 University Of Rochester Ribozyme-mediated RNA Assembly and Expression
EP4103724A1 (en) 2020-02-14 2022-12-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
CN115552022A (zh) 2020-03-02 2022-12-30 特纳亚治疗股份有限公司 由心肌细胞表达的微rna控制的基因载体
US20230085724A1 (en) 2020-03-05 2023-03-23 Neotx Therapeutics Ltd. Methods and compositions for treating cancer with immune cells
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
US20240218367A1 (en) 2020-03-27 2024-07-04 University Of Rochester Targeted Destruction of Viral RNA by CRISPR-Cas13
WO2021195525A1 (en) 2020-03-27 2021-09-30 University Of Rochester Crispr-cas13 crrna arrays
CA3175034A1 (en) 2020-03-27 2021-09-30 UCB Biopharma SRL Autonomous knob domain peptides
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
US20230226223A1 (en) 2020-04-10 2023-07-20 Sola Biosciences Llc Compositions and Methods for the Treatment of Protein Aggregation Disorders
BR112022021134A2 (pt) 2020-04-20 2023-02-14 Tenaya Therapeutics Inc Vírus adeno-associado com capsídeo projetado
US20230190956A1 (en) 2020-04-24 2023-06-22 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy
TW202208626A (zh) 2020-04-24 2022-03-01 美商生命編輯公司 Rna引導核酸酶及其活性片段與變體，以及使用方法
AU2021265088A1 (en) 2020-04-28 2022-11-03 Sola Biosciences Llc Compositions and methods for the treatment of TDP-43 proteinopathies
WO2021222332A1 (en) 2020-04-29 2021-11-04 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
EP4162059A1 (en) 2020-05-12 2023-04-12 The Trustees of The University of Pennsylvania Compositions for drg-specific reduction of transgene expression
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
IL298128A (en) 2020-05-13 2023-01-01 Akouos Inc Compositions and methods for treating slc26a4-associated hearing loss
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
CN115806596B (zh) * 2020-05-22 2026-03-06 中国医学科学院血液病医院(中国医学科学院血液学研究所) 腺相关病毒突变体及其应用
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
CA3183251A1 (en) 2020-06-05 2021-12-09 Sola Biosciences Llc Compositions and methods for the treatment of synucleinopathies
JP2023530974A (ja) 2020-06-15 2023-07-20 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋ジストロフィーのためのアデノ随伴ウイルスベクター送達
IL299167A (en) 2020-06-17 2023-02-01 Univ Pennsylvania Compositions and methods for treating patients with gene therapy
WO2021255245A2 (en) 2020-06-19 2021-12-23 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
WO2021262963A1 (en) 2020-06-24 2021-12-30 Bioverativ Therapeutics Inc. Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein
WO2022011262A1 (en) 2020-07-10 2022-01-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
CN116194587A (zh) 2020-07-10 2023-05-30 吉尼松公司 一种新的肌肉特异性启动子
BR112023000578A2 (pt) 2020-07-13 2023-05-09 Univ Pennsylvania Composições úteis para tratamento de doença de charcot-marie-tooth
EP4182455A1 (en) 2020-07-15 2023-05-24 University of Rochester Targeted rna cleavage with dcasl3-rnase fusion proteins
JP2023536148A (ja) 2020-07-30 2023-08-23 シェイプ セラピューティクス インク． Ｒａａｖウイルス粒子の誘導産生のための安定な細胞株
CA3188956A1 (en) 2020-08-14 2022-02-17 James M. Wilson Novel aav capsids and compositions containing same
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
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
EP4214317A2 (en) 2020-09-15 2023-07-26 Research Institute at Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
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
AU2021349277A1 (en) 2020-09-28 2023-05-11 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
WO2022073041A2 (en) 2020-09-30 2022-04-07 NGGT, Inc. Dual functional expression vectors and methods of use thereof
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
WO2022076591A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
US20240024508A1 (en) 2020-10-07 2024-01-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
IL301947A (en) 2020-10-07 2023-06-01 Regenxbio Inc Formulations for suprachoroidal administration such as gel formulations
KR20230083287A (ko) 2020-10-07 2023-06-09 리젠엑스바이오 인크. Cln2 질환의 안구 징후에 대한 유전자 요법
MX2023003699A (es) 2020-10-07 2023-04-21 Regenxbio Inc Virus adenoasociados para el suministro ocular de genoterapia.
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CN115698304A (zh) 2020-10-09 2023-02-03 特纳亚治疗股份有限公司 亲斑蛋白-2基因疗法的方法和组合物
TW202233841A (zh) 2020-10-28 2022-09-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗ＴＮＦ－α抗體
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
CN117042787A (zh) 2020-10-29 2023-11-10 宾夕法尼亚州大学信托人 Aav衣壳和含有aav衣壳的组合物
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
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
US20240026356A1 (en) 2020-11-30 2024-01-25 Research Institute At Nationwide Children's Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
AU2021391433A1 (en) 2020-12-01 2023-06-22 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
KR20230128001A (ko) 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
EP4256065A2 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
WO2022122733A1 (en) 2020-12-08 2022-06-16 Genethon New gene therapy for the treatment of duchenne muscular dystrophy
KR20230120128A (ko) 2020-12-16 2023-08-16 리젠엑스바이오 인크. 재조합 바이러스 입자의 생산 방법
JP2024500786A (ja) 2020-12-29 2024-01-10 アコーオス インコーポレイテッド Ｃｌｒｎ１関連難聴及び／または視力低下を治療するための組成物及び方法
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
WO2022150335A1 (en) 2021-01-05 2022-07-14 Selecta Biosciences, Inc. Viral vector dosing protocols
CA3205209A1 (en) 2021-01-21 2022-07-28 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
AU2022212922A1 (en) 2021-01-27 2023-08-17 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CA3210662A1 (en) 2021-02-03 2022-08-11 Research Institute At Nationwide Children's Hospital Compositions and methods for treating disease associated with dux4 overexpression
WO2022173605A2 (en) 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
AR124981A1 (es) 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co Composición y métodos para el tratamiento de la enfermedad de fabry
EP4301462A1 (en) 2021-03-04 2024-01-10 Research Institute at Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
CA3212876A1 (en) 2021-03-19 2022-09-22 Adrenas Therapeutics, Inc. Gene therapies for 21-hydroxylase deficiency
MX2023012052A (es) 2021-04-12 2024-03-15 Univ Pennsylvania Composiciones útiles para tratar la atrofia muscular espinal y bulbar (sbma).
WO2022221424A1 (en) 2021-04-13 2022-10-20 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus encoding methyl-cpg binding protein 2 for treating pitt hopkins syndrome via intrathecal delivery
EP4334334A1 (en) 2021-04-23 2024-03-13 The Trustees of The University of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
AU2022261125A1 (en) 2021-04-23 2023-11-23 University Of Rochester Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
EP4326752A1 (en) 2021-04-23 2024-02-28 Research Institute at Nationwide Children's Hospital Products and methods for treating muscular dystrophy
EP4329821A1 (en) 2021-04-26 2024-03-06 RegenxBio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2022232267A1 (en) 2021-04-27 2022-11-03 The Trustees Of The University Of Pennsylvania Porcine-derived adeno-associated virus capsids and uses thereof
WO2022229702A2 (en) 2021-04-30 2022-11-03 Takeda Pharmaceutical Company, Ltd. Aav8 capsid variants with enhanced liver targeting
WO2022229703A2 (en) 2021-04-30 2022-11-03 Takeda Pharmaceutical Company, Ltd. New aav8 based immune escaping variants
US20240218397A1 (en) 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
US20240358857A1 (en) 2021-05-11 2024-10-31 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
KR20240021765A (ko) 2021-05-17 2024-02-19 사렙타 쎄러퓨틱스 인코퍼레이티드 근이영양증 치료를 위한 재조합 aav 벡터의 제조
EP4108263A3 (en) 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
WO2022272296A2 (en) * 2021-06-25 2022-12-29 Homology Medicines, Inc. Adeno-associated virus packaging systems
WO2023280926A1 (en) 2021-07-07 2023-01-12 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies
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
WO2023004331A1 (en) 2021-07-19 2023-01-26 New York University Auf1 combination therapies for treatment of muscle degenerative disease
IL310725A (en) 2021-08-11 2024-04-01 Solid Biosciences Inc Treatment of muscular dystrophy
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
AR126840A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
EP4396237A4 (en) 2021-08-31 2025-11-19 Scout Bio Inc Antigen-binding molecules and their uses
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
CA3234702A1 (en) 2021-10-07 2023-04-13 Research Institute At Nationwide Children's Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250042958A1 (en) 2021-10-08 2025-02-06 Sola Biosciences Llc Compositions and methods for the treatment of proteopathies
EP4413023A1 (en) 2021-10-08 2024-08-14 SOLA Biosciences LLC Compositions and methods for the treatment of p53-mediated cancers
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
JP2024539012A (ja) 2021-10-12 2024-10-28 ブリッジバイオ ジーン セラピー エルエルシー 白質ジストロフィーを治療するための方法及び組成物
EP4415760A1 (en) 2021-10-12 2024-08-21 Cartesian Therapeutics, Inc. Viral vector dosing protocols
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
WO2023077092A1 (en) 2021-10-28 2023-05-04 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
EP4429662A1 (en) 2021-11-14 2024-09-18 Cartesian Therapeutics, Inc. Multiple dosing with viral vectors
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20230279431A1 (en) 2021-11-30 2023-09-07 Research Institute At Nationwide Children's Hospital Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
US20250152740A1 (en) 2021-12-21 2025-05-15 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
AU2022423978A1 (en) 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
AU2023208961A1 (en) 2022-01-24 2024-09-12 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
CA3243517A1 (en) 2022-02-02 2023-08-10 Akouos, Inc. Anti-VEGF Antibody Constructions and Related Methods for the Treatment of Symptoms Associated with Acoustic Neuroma
CA3253405A1 (en) 2022-03-03 2023-09-07 University Of Utah Research Foundation MATERIALS AND METHODS FOR THE TREATMENT OF MUTATIONS IN EIF2B5 AND DISEASES RESULTING THEREIN
WO2023172624A1 (en) 2022-03-09 2023-09-14 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
EP4493226A1 (en) 2022-03-13 2025-01-22 RegenxBio Inc. Modified muscle-specific promoters
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
EP4502166A4 (en) * 2022-03-25 2025-09-24 Univ Jichi Medical EFFICIENT ADENO-ASSOCIATED VIRAL (AAV) VECTOR PRODUCTION SYSTEM
EP4504950A1 (en) 2022-04-01 2025-02-12 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
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
CA3247507A1 (en) 2022-04-06 2023-10-12 Regenxbio Inc. PHARMACEUTICAL COMPOSITION COMPRISING A RECOMMENDED ADENO-ASSOCIATED VIRUS (AAV) VECTOR WITH AN EXPRESSION CASSETTE ENCODED BY A TRANSGENE FOR SUPRACHOROID ADMINISTRATION
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
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
EP4508062A1 (en) 2022-04-11 2025-02-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
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
US20250288697A1 (en) 2022-05-03 2025-09-18 Regenxbio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
TW202400803A (zh) 2022-05-03 2024-01-01 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
CA3255627A1 (en) 2022-05-06 2023-11-09 Novartis Ag NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV
WO2023240177A1 (en) 2022-06-08 2023-12-14 Research Instiitute At Nationwide Children's Hospital Products and methods for treating diseases or conditions associated with mutant or pathogenic kcnq3 expression
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
EP4544051A2 (en) 2022-06-24 2025-04-30 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
US20260021205A1 (en) 2022-07-15 2026-01-22 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
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
WO2024036324A1 (en) 2022-08-11 2024-02-15 Selecta Biosciences, Inc. Compositions and methods related to immunoglobulin proteases and fusions thereof
IL318914A (en) 2022-08-12 2025-04-01 Life Edit Therapeutics Inc RNA-guided nucleases and active fragments and variants thereof and methods of use
US20260055144A1 (en) 2022-08-24 2026-02-26 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
TW202413648A (zh) 2022-08-25 2024-04-01 日商武田藥品工業股份有限公司 用於治療法布立氏病（ｆａｂｒｙ ｄｉｓｅａｓｅ）之組合物及方法
US20250108127A1 (en) * 2022-08-29 2025-04-03 Shanghai Ophthal-Bright Biomedicine Technology Modified vector, construction method, and application of modified aav-8 serotype for gene targeting and expression
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
TW202417631A (zh) 2022-09-22 2024-05-01 美商拜奧馬林製藥公司 用ａａｖ基因治療載體治療心肌病
CN120225688A (zh) 2022-09-23 2025-06-27 萨雷普塔治疗公司 用于治疗肌营养不良症的重组aav载体
US20260034250A1 (en) 2022-09-30 2026-02-05 Regenxbio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
JP2025534651A (ja) 2022-10-11 2025-10-17 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 呼吸困難を伴う脊髄性筋萎縮１型（ｓｍａｒｄ１）及びシャルコー・マリー・トゥース２ｓ型（ｃｍｔ２ｓ）を治療するためのアデノ随伴ウイルス送達
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
KR20250135916A (ko) 2022-12-17 2025-09-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물
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
WO2024146935A1 (en) 2023-01-06 2024-07-11 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
EP4658796A1 (en) 2023-02-01 2025-12-10 Sarepta Therapeutics, Inc. Raav production methods
WO2024168089A2 (en) * 2023-02-07 2024-08-15 Duke University Compositions comprising aavs and methods for treating gastrointestinal diseases
WO2024168358A1 (en) 2023-02-10 2024-08-15 Expression Therapeutics, Llc Lentiviral system
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024196855A2 (en) 2023-03-17 2024-09-26 University Of Rochester Ribozyme-mediated rna assembly and expression
KR20260008735A (ko) 2023-03-30 2026-01-16 파마 싱크, 엘엘씨 간상체-유래 원뿔세포 생존인자 및 인간 IgK 신호 서열을 인코딩하는 벡터
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
EP4698558A2 (en) 2023-04-18 2026-02-25 Research Institute at Nationwide Children's Hospital, Inc. Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c
WO2024229259A1 (en) 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases
AU2024265701A1 (en) 2023-05-02 2025-12-04 Research Institute At Nationwide Children's Hospital A modular system to convert therapeutic microrna expression cassettes from polymerase iii-based to polymerase ii-based promoters
CN121399470A (zh) 2023-05-03 2026-01-23 马尼福尔德生物技术有限公司 用于高通量蛋白质递送、筛选和检测的方法和组合物
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
EP4709406A2 (en) 2023-05-11 2026-03-18 University Hospitals Cleveland Medical Center Anxiolytic therapy
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
AU2024271004A1 (en) 2023-05-16 2026-01-15 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
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
AU2024283898A1 (en) 2023-06-07 2026-01-22 Research Institute At Nationwide Children's Hospital Gene therapy for lysosomal acid lipase deficiency (lal-d)
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2024259064A1 (en) 2023-06-13 2024-12-19 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
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
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
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
WO2025096498A1 (en) 2023-10-30 2025-05-08 Research Institute At Nationwide Children's Hospital Compositions and methods for treating diseases or conditions associated with progerin expression
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
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025188993A2 (en) 2024-03-07 2025-09-12 Research Institute At Nationwide Children's Hospital Gene therapy for treating gne-related disorders
WO2025212838A1 (en) 2024-04-03 2025-10-09 Research Institute At Nationwide Children's Hospital Products and methods for treating diseases or disorders associated with dux4 overexpression
TW202548025A (zh) 2024-04-08 2025-12-16 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors
WO2025235425A1 (en) 2024-05-06 2025-11-13 Research Institute At Nationwide Children's Hospital Improved proviral plasmids
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
WO2025240690A2 (en) 2024-05-15 2025-11-20 Research Institute At Nationwide Children's Hospital Products and methods for treating diseases or conditions associated with progerin expression from an aberrant lmna gene
WO2025244671A1 (en) 2024-05-22 2025-11-27 The Scripps Research Institute Compositions for use in treating haploinsufficiency diseases
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
WO2026035687A1 (en) 2024-08-05 2026-02-12 University Of Rochester Compositions and methods for stitchr-mediated full-length scn5a expression in vivo
WO2026044087A1 (en) 2024-08-21 2026-02-26 Shape Therapeutics Inc. Increased cellular stability for aav production

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US20020037867A1 (en) 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
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
EP0950111A1 (en) 1996-09-06 1999-10-20 The Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
CN1233291A (zh) 1996-09-06 1999-10-27 宾西法尼亚大学托管会 重组腺伴随病毒定向基因治疗的方法
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
EP0938578B1 (en) 1996-12-05 2004-02-04 Crucell Holland B.V. Genetic modification of primate hemopoietic repopulating stem cells
US6399358B1 (en) * 1997-03-31 2002-06-04 Thomas Jefferson University Human gene encoding human chondroitin 6-sulfotransferase
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) * 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
AU734514B2 (en) 1997-09-18 2001-06-14 Eaton Electric N.V. Electromagnetic actuator
CA2303768C (en) * 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
EP1015619A1 (en) * 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
WO1999015677A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Method for gene transfer using bcl2 and compositions useful therein
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
DE69922934T2 (de) 1998-03-20 2005-12-08 The Trustees Of The University Of Pennsylvania Zusammensetzungen und Verfahren zur helfer-freien Herstellung von rekombinanten Adeno-assoziierten Viren
US6475988B1 (en) * 1998-05-11 2002-11-05 University Of Southern California Methods to increase white blood cell survival after chemotherapy
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
PT1127150E (pt) 1998-11-05 2007-08-22 Univ Pennsylvania ''sequências de ácido nucleico do vírus adeno associado do serotipo 1, vectores e células hospedeiras que as contêm''
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
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
AU2409200A (en) 1999-06-02 2000-12-28 Trustees Of The University Of Pennsylvania, The Compositions and methods useful for production of recombinant viruses which require helper viruses
EP1218035A2 (en) * 1999-09-29 2002-07-03 The Trustees Of The University Of Pennsylvania Rapid peg-modification of viral vectors
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
JP2003511037A (ja) * 1999-10-01 2003-03-25 ジェノボ， インコーポレイテッド ＡＡＶｒｅｐ／ｃａｐ遺伝子を含むアデノウイルスを使用する組換えＡＡＶの産生
US7115391B1 (en) * 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
WO2001040455A2 (en) 1999-12-03 2001-06-07 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yields of recombinant adenoviruses using multiple packaging signals
US6821512B1 (en) * 1999-12-03 2004-11-23 The Trustees Of The University Of Pennsylvania Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals
US20020045264A1 (en) * 2000-03-14 2002-04-18 During Matthew J. Production of chimeric capsid vectors
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
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
JP2004520812A (ja) 2000-08-30 2004-07-15 ハプロゲン・エルエルシー 対立遺伝子を決定するための方法
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
CN103555677B (zh) * 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
PT2573170T (pt) * 2001-12-17 2018-03-26 Univ Pennsylvania Sequências de um vírus adenoassociado (aav) de serotipo 9, vetor contendo as mesmas, e suas utilizações
WO2003052051A2 (en) * 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
AU2002367943A1 (en) * 2001-12-18 2003-12-22 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
DK3211085T3 (da) 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
US9819463B2 (en) 2016-02-18 2017-11-14 Huawei Technologies Co., Ltd. Method and apparatus for transmitting data in a wireless communication system

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