DK2359869T3 - Sekvenser af adeno-associeret virus (AAV) serotype 8, vektorer indeholdende disse og anvendelser deraf - Google Patents

Sekvenser af adeno-associeret virus (AAV) serotype 8, vektorer indeholdende disse og anvendelser deraf Download PDF

Info

Publication number
DK2359869T3
DK2359869T3 DK10189725.4T DK10189725T DK2359869T3 DK 2359869 T3 DK2359869 T3 DK 2359869T3 DK 10189725 T DK10189725 T DK 10189725T DK 2359869 T3 DK2359869 T3 DK 2359869T3
Authority
DK
Denmark
Prior art keywords
gly
pro
ala
ser
asn
Prior art date
Application number
DK10189725.4T
Other languages
English (en)
Inventor
Guangping Gao
Mauricio Alvira
James M Wilson
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=DK2359869(T3) "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
Application granted granted Critical
Publication of DK2359869T3 publication Critical patent/DK2359869T3/da

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • C12N9/10—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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • 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)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Diabetes (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)

Claims (10)

1. Rekombinant nukleinsyremolekyle: (a) der koder for et AAV8 vpl kapsidprotein med en sekvens omfattende aminosyrer 1 til 738 af SEQ ID NO: 2 eller (b) omfattende nukleotider 2121 til 4337 af SEQ ID NO: 1, eller en nukleotidsekvens der er mindst 99% identisk med nukleotider 2121 til 4337 af SEQ ID NO: 1.
2. Rekombinant nukleinsyremolekyle ifølge krav 1, (a) der koder for aminosyrer 138 til 738 af SEQ ID NO: 2 eller (b) der omfatter nukleotider 2532 til 4337 af SEQ ID NO: 1, eller en nukleotidsekvens der er mindst 99% identisk med nukleotider 2532 til 4337 af SEQ ID NO: 1.
3. Rekombinant nukleinsyremolekyle ifølge krav 2, (a) der koder for aminosyrer 204 til 738 af SEQ ID NO: 2 eller (b) der omfatter nukleotider 2730 til 4337 af SEQ ID NO: 1, eller en nukleotidsekvens der er mindst 99% identisk med nukleotider 2730 til 4337 af SEQ ID NO: 1.
4. Rekombinant nukleinsyremolekyle ifølge krav 1, hvor det rekombinante nukleinsyremolekyle omfatter en sekvens valgt fra: vpl, nt 2121 til 4337; vp2, nt 2532 til 4337; eller vp3, nt 2730 til 4337 af SEQ ID NO: 1.
5. Rekombinant nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 4, hvor det rekombinante nukleinsyremolekyle er et plasmid.
6. Værtscelle transfekteret med et rekombinant nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 5.
7. Værtscelle ifølge krav 6, der endvidere omfatter et funktionelt rep gen, et minigen omfattende AAV-terminalgentagelser (ITR'er) og et transgen, og tilstrækkelige hjælperfunktioner til at tillade pakning af minigenet i AAV-kapsidet.
8. Rekombinant AAV8 fremstillet af værtscellen ifølge krav 6 eller 7.
9. Adeno-associeret virus (AAV) omfattende et AAV8-kapsidprotein kodet af et rekombinant nukleinsyremolekyle ifølge et hvilket som helst af kravene 1 til 5.
10. Sammensætning omfattende AAV'et ifølge krav 8 eller 9 og en fysiologisk kompatibel bærer.
DK10189725.4T 2001-12-17 2002-11-12 Sekvenser af adeno-associeret virus (AAV) serotype 8, vektorer indeholdende disse og anvendelser deraf DK2359869T3 (da)

Applications Claiming Priority (4)

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
EP02795539.2A EP1453547B1 (en) 2001-12-17 2002-11-12 Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor

Publications (1)

Publication Number Publication Date
DK2359869T3 true DK2359869T3 (da) 2019-04-15

Family

ID=27407443

Family Applications (2)

Application Number Title Priority Date Filing Date
DK10189725.4T DK2359869T3 (da) 2001-12-17 2002-11-12 Sekvenser af adeno-associeret virus (AAV) serotype 8, vektorer indeholdende disse og anvendelser deraf
DK02795539.2T DK1453547T3 (da) 2001-12-17 2002-11-12 Adenoassocieret virus (aav)-serotype 8-sekvenser, vektorer indeholdende disse og anvendelser deraf

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK02795539.2T DK1453547T3 (da) 2001-12-17 2002-11-12 Adenoassocieret virus (aav)-serotype 8-sekvenser, vektorer indeholdende disse og anvendelser deraf

Country Status (11)

Country Link
US (15) US7282199B2 (da)
EP (3) EP2359869B1 (da)
JP (2) JP4810062B2 (da)
AU (1) AU2002360291A1 (da)
CA (1) CA2469785C (da)
CY (2) CY1120162T1 (da)
DK (2) DK2359869T3 (da)
ES (3) ES2975413T3 (da)
FI (1) FI3517134T3 (da)
PT (2) PT1453547T (da)
WO (1) WO2003052051A2 (da)

Families Citing this family (524)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
DE69939169D1 (de) * 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
EP1427835B1 (en) 2001-08-08 2016-01-20 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
CN103589692B (zh) * 2001-11-13 2018-07-24 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
EP1463805B1 (en) * 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
DK2359869T3 (da) 2001-12-17 2019-04-15 Univ Pennsylvania Sekvenser af adeno-associeret virus (AAV) serotype 8, vektorer indeholdende disse og anvendelser deraf
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
WO2005014775A2 (en) * 2002-06-28 2005-02-17 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
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
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
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
US9592284B2 (en) 2004-04-28 2017-03-14 The Trustees Of The University Of Pennsylvania Immunization regimen with E4-deleted adenovirus prime and E1-deleted adenovirus boost
US7964196B2 (en) * 2004-05-25 2011-06-21 Chimeros, Inc. Self-assembling nanoparticle drug delivery system
US20080188431A1 (en) * 2004-09-08 2008-08-07 Chiorini John A Transcytosis of Adeno-Associated Viruses
US8283151B2 (en) * 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
AU2006243776A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for neurometabolic disorders
AU2006242129A1 (en) * 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for spinal cord disorders
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
JP2009533350A (ja) * 2006-04-07 2009-09-17 キメロス， インコーポレイテッド Ｂ細胞悪性疾患を処置するための組成物および方法
JP2009535339A (ja) * 2006-04-28 2009-10-01 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 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
US20090226525A1 (en) * 2007-04-09 2009-09-10 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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts 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
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
DK2561073T3 (da) 2010-04-23 2016-12-12 Univ Massachusetts Aav-vektorer målrettet mod centralnervesystemet og fremgangsmåder til anvendelse deraf
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
ES2685611T3 (es) 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
CN107320734A (zh) 2011-04-29 2017-11-07 西莱克塔生物科技公司 从合成纳米载体中控制释放免疫抑制剂
CA2847888A1 (en) 2011-09-09 2013-03-14 Biomed Realty, L.P. Methods and compositions for controlling assembly of viral proteins
CN110777124A (zh) 2011-10-27 2020-02-11 威尔斯达眼科制剂公司 编码视杆细胞来源的视锥细胞活力因子的载体
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
CN105120901A (zh) 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
WO2014124282A1 (en) 2013-02-08 2014-08-14 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
RS61387B2 (sr) 2013-02-15 2024-06-28 Bioverativ Therapeutics Inc Gen optimizovanog faktora viii
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)
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
US9862945B2 (en) 2013-04-20 2018-01-09 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
CN105307641A (zh) 2013-05-03 2016-02-03 西莱克塔生物科技公司 用于降低不期望体液免疫应答的给药组合
NZ716102A (en) * 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
LT3105328T (lt) 2014-02-11 2020-07-10 The Regents Of The University Of Colorado, A Body Corporate Daugiakomponentis genomo konstravimas, sudarantis sąlygas crispr
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
BR112016020688B1 (pt) 2014-03-09 2024-01-30 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
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
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
CR20160519A (es) 2014-05-13 2017-01-27 The Trustees Of The Univ Of Pennysylvania Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos
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
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
CA2957661A1 (en) 2014-08-09 2016-02-18 Kevin FLANIGAN Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
EA201790551A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
WO2016054557A1 (en) * 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
JP6754361B2 (ja) 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
EP3632923A1 (en) 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
CN115094062A (zh) 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
JP6851319B2 (ja) 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
WO2016200543A2 (en) 2015-05-13 2016-12-15 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-inluenza antibodies and methods of use thereof
EP4378487A3 (en) 2015-06-23 2024-09-04 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
HK1257024A1 (zh) 2015-08-31 2019-10-11 宾夕法尼亚大学 用於治疗伴侣动物的aav-epo
EP3359663A4 (en) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
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
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US11135313B2 (en) 2015-10-28 2021-10-05 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
KR102404550B1 (ko) 2015-11-13 2022-05-31 다케다 야쿠힌 고교 가부시키가이샤 A형 혈우병의 유전자 요법을 위한 증가된 발현을 갖는 재조합 fviii 변이체를 인코딩하는 바이러스 벡터
CN108884145B (zh) 2015-11-13 2023-08-22 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
CN108463211B (zh) 2015-11-16 2022-09-27 全国儿童医院研究所 用于治疗肌联蛋白类肌病和其它肌联蛋白病变的材料和方法
WO2017096164A1 (en) 2015-12-02 2017-06-08 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
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP4085934A1 (en) * 2015-12-11 2022-11-09 The Trustees of The University of Pennsylvania Gene therapy for treating familial hypercholesterolemia
EP3387138B2 (en) 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
CN109069668B (zh) 2015-12-14 2023-04-18 宾夕法尼亚州大学信托人 用于眼病的基因疗法
AU2016370590B2 (en) * 2015-12-14 2023-11-02 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
CA3011939A1 (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
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
CA3014637A1 (en) 2016-02-16 2017-08-24 Mark A. Kay Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
BR112018070249A2 (pt) 2016-04-02 2019-01-29 Research Institute At Nationwide Children's Hospital sistema promotor de u6 modificada para expressão específica de tecido
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
IL262207B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
PE20181922A1 (es) 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
CN109661470A (zh) * 2016-04-15 2019-04-19 宾夕法尼亚州大学信托人 新型aav8突变衣壳和含有其的组合物
US11253612B2 (en) 2016-04-15 2022-02-22 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
WO2017181011A1 (en) 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
WO2017184463A1 (en) 2016-04-17 2017-10-26 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
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
US11471462B2 (en) 2016-06-27 2022-10-18 The Broad Institute, Inc. Compositions and methods for detecting and treating diabetes
US11197936B2 (en) 2016-07-08 2021-12-14 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving RDH12
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
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
CA3031688A1 (en) * 2016-08-15 2018-02-22 Beth Israel Deaconess Medical Center, Inc. Therapy to achieve glycemic control
CA3034089A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
ES2931960T3 (es) 2016-12-01 2023-01-05 Inst Nat Sante Rech Med Composiciones farmacéuticas para el tratamiento de enfermedades degenerativas retinianas
EP3562494A4 (en) 2016-12-30 2020-08-19 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS
KR102863181B1 (ko) 2016-12-30 2025-09-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 윌슨병을 치료하기 위한 유전자 치료
JP2020506890A (ja) 2017-01-07 2020-03-05 セレクタ バイオサイエンシーズ インコーポレーテッドＳｅｌｅｃｔａ Ｂｉｏｓｃｉｅｎｃｅｓ，Ｉｎｃ． 合成ナノキャリアにカップリングした免疫抑制剤のパターン化された投与
JP2020508289A (ja) 2017-01-31 2020-03-19 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
WO2018144709A2 (en) 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
JP2020510648A (ja) 2017-02-20 2020-04-09 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 家族性高コレステロール血症を処置するための遺伝子治療
TW201837170A (zh) 2017-02-28 2018-10-16 賓州大學委員會 新穎ａａｖ媒介的流感疫苗
RS65241B1 (sr) 2017-02-28 2024-03-29 Univ Pennsylvania Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018160849A1 (en) 2017-03-01 2018-09-07 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EP4599848A3 (en) 2017-03-11 2025-12-03 Cartesian Therapeutics, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
CN110997923B (zh) 2017-03-17 2024-01-02 全国儿童医院研究所 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
CN110506060B (zh) 2017-03-30 2024-05-07 昆士兰大学 嵌合分子及其用途
EP3610016A1 (en) 2017-04-10 2020-02-19 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
KR20240093919A (ko) 2017-04-14 2024-06-24 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
EP4269596A3 (en) 2017-04-21 2024-02-14 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the pcsk9 gene
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
JP2020519272A (ja) 2017-05-09 2020-07-02 エモリー ユニバーシティー 凝固因子変異体及びその使用
BR112019023303A2 (pt) 2017-05-11 2020-06-16 The Trustees Of The University Of Pennsylvania Terapia de gene para lipofuscinoses ceroides neuronais
US11958887B2 (en) 2017-05-17 2024-04-16 The General Hospital Corporation Gene therapy for tuberous sclerosis
TWI753168B (zh) 2017-05-22 2022-01-21 日商武田藥品工業股份有限公司 用於血友病ｂ基因療法之編碼具有增強表現的重組ｆｉｘ變異體之病毒載體
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (ｆｇｆ21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
US11793887B2 (en) 2017-05-31 2023-10-24 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
US11827898B2 (en) 2017-06-14 2023-11-28 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3068328A1 (en) 2017-07-06 2019-01-10 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
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
CA3071769A1 (en) 2017-07-31 2019-02-07 Reflection Biotechnologies Limited Cellular models of and therapies for ocular diseases
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
WO2019040994A1 (en) 2017-09-01 2019-03-07 The Australian National University IMMUNOREGULATOR MOLECULES AND USES THEREOF
DK3684423T5 (da) 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
WO2019075360A1 (en) 2017-10-13 2019-04-18 Selecta Biosciences, Inc. METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
MX2020003945A (es) 2017-10-18 2020-11-09 Regenxbio Inc Tratamiento de enfermedades oculares y cancer de colon metastasico con vegf-trap humano modificado post-traduccionalmente .
WO2019078916A1 (en) 2017-10-18 2019-04-25 Research Institute At Nationwide Children's Hospital ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
KR20200067195A (ko) 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
MX2020004035A (es) 2017-10-20 2020-11-09 Res Inst Nationwide Childrens Hospital Métodos y materiales para terapia génica con nt-3.
CA3081106A1 (en) 2017-11-01 2019-05-09 Queen's University At Kingston Von willebrand factor proteins for treating bleeding disorders
US11766489B2 (en) 2017-11-27 2023-09-26 4D Molecular Therapeutics, Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
EA202091352A1 (ru) 2017-11-30 2020-08-27 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Генотерапия мукополисахаридоза iiib
US11555206B2 (en) 2017-11-30 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIA
CA3086045A1 (en) 2017-12-19 2019-06-27 Akouos, Inc. Aav-mediated delivery of therapeutic antibodies to the inner ear
JP7569216B2 (ja) 2018-01-17 2024-10-17 アドリーナス セラピューティクス， インコーポレイテッド ２１－ヒドロキシラーゼ欠損症のためのアデノ随伴ウイルス遺伝子療法
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
AU2019215152A1 (en) * 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
WO2019152841A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス，インコーポレーテッド 寛容原性リポソーム及びその使用方法
EP4169576A1 (en) 2018-03-23 2023-04-26 University of Massachusetts Gene therapeutics for treating bone disorders
EP3774853A1 (en) 2018-03-30 2021-02-17 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
BR112020020266A2 (pt) * 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3099958A1 (en) 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
CA3098566A1 (en) 2018-04-29 2019-11-07 Zhuchun WU 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
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019221992A1 (en) 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
AU2019282149B2 (en) 2018-06-05 2025-08-21 Life Edit Therapeutics, Inc. RNA-guided nucleases and active fragments and variants thereof and methods of use
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
IL319002A (en) 2018-06-14 2025-04-01 Regenxbio Inc Anion exchange chromatography for recombinant AAV production
IL324506A (en) 2018-06-18 2026-01-01 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy
IL279789B2 (en) 2018-06-29 2026-04-01 Res Inst Nationwide Childrens 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
EP3820537A4 (en) 2018-07-12 2022-04-13 Spacecraft Seven, LLC GENE THERAPY VECTORS FOR TREATMENT OF DANON DISEASE
US12091675B2 (en) 2018-07-16 2024-09-17 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression
EP3823676A1 (en) 2018-07-16 2021-05-26 Selecta Biosciences, Inc. Methods and compositions of mma constructs and vectors
CN112771070A (zh) 2018-07-16 2021-05-07 西莱克塔生物科技公司 Otc构建体和载体的方法和组合物
CA3106078A1 (en) 2018-07-17 2020-01-23 Junghun Lee Treatment of neuropathy with dna constructs expressing igf-1 isoforms
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020033842A1 (en) 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production
AU2019326551A1 (en) 2018-08-22 2021-03-18 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
AU2019328270B2 (en) 2018-08-29 2026-02-26 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
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354296A1 (en) 2018-10-01 2021-04-29 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
JP2022514112A (ja) 2018-10-15 2022-02-09 リジェネクスバイオ インコーポレイテッド 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス，インコーポレーテッド 第ｉｘ因子を発現するための組成物及び方法
US20210363509A1 (en) 2018-10-22 2021-11-25 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas9 Fusion Protein
CN113613668A (zh) 2018-10-24 2021-11-05 百时美施贵宝公司 小型化肌营养不良蛋白及其用途
CA3123841A1 (en) 2018-12-18 2020-06-25 Ultragenyx Pharmaceutical Inc. Methods and compositions for treating glycogen storage diseases
CN113631704A (zh) 2018-12-27 2021-11-09 生命编辑制药股份有限公司 可用于基因编辑的多肽及使用方法
WO2020140007A1 (en) 2018-12-28 2020-07-02 University Of Rochester Gene therapy for best1 dominant mutations
JP2022516515A (ja) 2018-12-31 2022-02-28 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 Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止
KR20210112339A (ko) 2019-01-04 2021-09-14 울트라제닉스 파마수티컬 인코포레이티드 윌슨병을 치료하기 위한 유전자 요법 구축물
CN113544267B (zh) 2019-01-14 2025-04-18 罗切斯特大学 使用CRISPR-Cas进行靶向核RNA裂解和聚腺苷酸化
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
CN113924109A (zh) 2019-01-18 2022-01-11 巴斯德研究所 Aav介导的基因疗法恢复耳畸蛋白基因
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
CN119390787A (zh) 2019-01-31 2025-02-07 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
KR20210133242A (ko) 2019-02-26 2021-11-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료
AU2020232692A1 (en) 2019-03-04 2021-09-30 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the AKT pathway
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
EP3947427A1 (en) 2019-03-25 2022-02-09 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
EP4667578A3 (en) 2019-04-03 2026-04-01 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
ES2965341T3 (es) 2019-04-11 2024-04-12 Regenxbio Inc Métodos de cromatografía de exclusión por tamaño para la caracterización de composiciones de virus adenoasociados recombinantes
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
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
US20220195462A1 (en) 2019-04-24 2022-06-23 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
CA3138525A1 (en) 2019-04-28 2020-11-05 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
WO2020223232A1 (en) 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
CA3141863A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
US12553041B2 (en) 2019-06-04 2026-02-17 Cartesian Therapeutics Inc. Formulations and doses of pegylated uricase
BR112021026866A2 (pt) 2019-07-02 2022-03-03 M6P Therapeutics Composições de vetor e métodos de seu uso para o tratamento de distúrbios do armazenamento lisossômico
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
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
EP3997226A1 (en) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
JP7371954B2 (ja) * 2019-07-12 2023-10-31 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
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
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
WO2021016453A1 (en) 2019-07-23 2021-01-28 University Of Rochester Targeted rna cleavage with crispr-cas
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
CA3150964A1 (en) 2019-08-21 2021-02-25 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
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
CA3149401A1 (en) 2019-08-26 2021-03-04 Stephen Joseph Pakola Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab
EP4031673A1 (en) 2019-09-19 2022-07-27 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
JP7803014B2 (ja) 2019-09-30 2026-01-21 バイオベラティブ セラピューティクス インコーポレイテッド レンチウイルスベクター製剤
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
WO2021071835A1 (en) 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
US12680122B2 (en) 2019-10-08 2026-07-14 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
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
JP7781057B2 (ja) 2019-10-18 2025-12-05 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 Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
EP4055030A4 (en) 2019-11-08 2024-05-22 President And Fellows Of Harvard College VIRAL CAPSID POLYPEPTIDES
EP4055034A4 (en) 2019-11-08 2024-05-29 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
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
CN115023242B (zh) * 2019-11-22 2025-03-14 费城儿童医院 腺相关病毒载体变体
AU2020392243A1 (en) 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
KR20220112283A (ko) 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Hunter 질환 치료용 아데노-연합된 바이러스 벡터
EP4077687A1 (en) 2019-12-20 2022-10-26 Research Institute at Nationwide Children's Hospital Optimized gene therapy for targeting muscle in muscle diseases
CN116096904A (zh) 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途
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
KR20220131522A (ko) 2020-01-22 2022-09-28 리젠엑스바이오 인크. 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료
US20230088992A1 (en) 2020-01-29 2023-03-23 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
BR112022015036A2 (pt) 2020-01-29 2022-10-11 Regenxbio Inc Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
EP4100533A1 (en) 2020-02-07 2022-12-14 University of Rochester Ribozyme-mediated rna assembly and expression
US20230078498A1 (en) 2020-02-07 2023-03-16 University Of Rochester Targeted Translation of RNA with CRISPR-Cas13 to Enhance Protein Synthesis
AU2021218703A1 (en) 2020-02-14 2022-07-28 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
JP2023517011A (ja) 2020-03-05 2023-04-21 ネオティーエックス セラピューティクス リミテッド 免疫細胞を用いて癌を治療するための方法および組成物
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
EP4127170A1 (en) 2020-03-27 2023-02-08 University of Rochester Crispr-cas13 crrna arrays
JP2023519307A (ja) 2020-03-27 2023-05-10 ユニバーシティ オブ ロチェスター Ｃｒｉｓｐｒ－ｃａｓ１３によるウイルスｒｎａの標的化破壊
JP2023519576A (ja) 2020-03-27 2023-05-11 ユーシービー バイオファルマ エスアールエル 自律ノブドメインペプチド
US12686874B2 (en) 2020-03-31 2026-07-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
JP2023520927A (ja) 2020-04-10 2023-05-22 ソラ・バイオサイエンシズ・エルエルシー タンパク質凝集障害の処置のための組成物および方法
AU2021259422A1 (en) 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
JP2023516226A (ja) 2020-04-24 2023-04-18 ライフエディット セラピューティクス，インコーポレイティド Ｒｎａ誘導型ヌクレアーゼとその活性な断片およびバリアント、ならびに利用の方法
EP4138929A1 (en) 2020-04-24 2023-03-01 Institut National de la Santé et de la Recherche Médicale (INSERM) Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy
JP7894321B2 (ja) 2020-04-28 2026-07-23 ソラ・バイオサイエンシズ・エルエルシー Ｔｄｐ－４３プロテイノパチーの処置のための組成物および方法
US11535868B2 (en) 2020-04-29 2022-12-27 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
IL298138A (en) 2020-05-12 2023-01-01 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression
EP4165194A2 (en) 2020-05-13 2023-04-19 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
KR20230041965A (ko) 2020-05-13 2023-03-27 아카우오스, 인크. Slc26a4-연관 청력손실을 치료하기 위한 조성물 및 방법
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
CN115925818B (zh) * 2020-05-22 2026-02-06 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
US20230234997A1 (en) 2020-06-05 2023-07-27 Sola Biosciences Llc Compositions and Methods for the Treatment of Synucleinopathies
EP4164668A1 (en) 2020-06-15 2023-04-19 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
CN115996758A (zh) 2020-06-19 2023-04-21 吉尼松公司 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统
JP2023537565A (ja) 2020-06-24 2023-09-04 バイオベラティブ セラピューティクス インコーポレイテッド タンパク質を発現するように改変されたレンチウイルスベクターの調製物から遊離第ｖｉｉｉ因子を除去する方法
BR112023000305A2 (pt) 2020-07-10 2023-03-21 Genethon Promotor, sistema de expressão e composição farmacêutica
KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
WO2022015715A1 (en) 2020-07-13 2022-01-20 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
EP4182455A1 (en) 2020-07-15 2023-05-24 University of Rochester Targeted rna cleavage with dcasl3-rnase fusion proteins
WO2022020548A2 (en) 2020-07-23 2022-01-27 President And Fellows Of Harvard College Variant txnip compositions and methods of use thereof for the treatment of degenerative ocular diseases
US12054738B2 (en) 2020-07-30 2024-08-06 Shape Therapeutics Inc. Stable cell lines for inducible production of rAAV virions
WO2022036220A1 (en) 2020-08-14 2022-02-17 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
US20230372539A1 (en) 2020-08-24 2023-11-23 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
US20250326861A1 (en) 2020-09-15 2025-10-23 Regenxbio Inc. Vectorized lanadelumab and administration thereof
JP2023541444A (ja) 2020-09-15 2023-10-02 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 筋ジストロフィー患者における多様なｄｍｄ変異の補正のためのａａｖ媒介性の相同性非依存的標的化組み込み遺伝子編集
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
US20240261434A1 (en) 2020-09-28 2024-08-08 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
EP4225777A2 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Adeno-associated viruses for ocular delivery of gene therapy
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
TW202228646A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如凝膠調配物之用於脈絡膜上投與之調配物
EP4225381A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
TW202228648A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如高黏度調配物之用於脈絡膜上投與之調配物
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
AU2021371307A1 (en) 2020-10-28 2023-06-01 Regenxbio, Inc. VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS
MX2023005113A (es) 2020-10-29 2023-08-04 Univ Pennsylvania Cápsides de aav y composiciones que las contienen.
US20230390418A1 (en) 2020-10-29 2023-12-07 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
KR20230098176A (ko) 2020-10-29 2023-07-03 리젠엑스바이오 인크. 안구 적응증을 위한 벡터화된 tnf-알파 길항제
KR20230078805A (ko) 2020-11-02 2023-06-02 바이오마린 파머수티컬 인크. 아데노-연관 바이러스의 농축 공정
CA3203585A1 (en) 2020-11-30 2022-06-02 Research Institute At Nationwide Children's Hospital Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
WO2022119890A1 (en) 2020-12-01 2022-06-09 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
AR124216A1 (es) 2020-12-01 2023-03-01 Univ Pennsylvania Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
WO2022122733A1 (en) 2020-12-08 2022-06-16 Genethon New gene therapy for the treatment of duchenne muscular dystrophy
EP4263841A1 (en) 2020-12-16 2023-10-25 REGENXBIO Inc. Method of producing a recombinant adeno-associated virus particle
US20240309076A1 (en) 2020-12-29 2024-09-19 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
CN117157108A (zh) 2020-12-29 2023-12-01 阿库斯股份有限公司 用于治疗clrn1相关听力损失和/或视力损失的组合物和方法
AU2022206197A1 (en) 2021-01-05 2023-07-13 Selecta Biosciences, Inc. Viral vector dosing protocols
AU2022210592A1 (en) 2021-01-21 2023-07-13 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
WO2022164860A1 (en) 2021-01-27 2022-08-04 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
US20240093191A1 (en) 2021-02-03 2024-03-21 Research Institute At Nationwide Children's Hospital Compositions and methods for treating disease associated with dux4 overexpression
AU2022221284A1 (en) 2021-02-10 2023-08-17 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
IL307633A (en) 2021-04-12 2023-12-01 Univ Pennsylvania Useful preparations in the treatment of spinal muscular atrophy (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
AU2022262771A1 (en) 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022226296A2 (en) 2021-04-23 2022-10-27 University Of Rochester Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
AU2022262420A1 (en) 2021-04-23 2023-11-16 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
IL307902A (en) 2021-04-26 2023-12-01 Regenxbio Inc Microdystrophin gene therapy administration for treatment of dystrophinopathies
TW202309066A (zh) 2021-04-27 2023-03-01 賓州大學委員會 衍生自豬的腺相關病毒衣殼及其用途
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
EP4334454A2 (en) 2021-05-04 2024-03-13 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
BR112023024078A2 (pt) 2021-05-17 2024-01-30 Sarepta Therapeutics Inc Produção de vetores de aav recombinantes para o tratamento de distrofia muscular
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
US20230055381A1 (en) * 2021-06-25 2023-02-23 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems
EP4366757A1 (en) 2021-07-07 2024-05-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Synergistic combination of rdcfv2 and rdcvf2l for the treatment of tauopathies
TW202317768A (zh) 2021-07-08 2023-05-01 美商特納亞治療股份有限公司 用於基因療法的優化表現卡匣
AU2022313258A1 (en) 2021-07-19 2024-02-08 New York University Auf1 combination therapies for treatment of muscle degenerative disease
CA3228365A1 (en) 2021-08-11 2023-02-16 Solid Biosciences Inc. Treatment of muscular dystrophy
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
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
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도
US20250288692A1 (en) 2021-09-30 2025-09-18 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
EP4413018A1 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for targeted delivery
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20240401046A1 (en) 2021-10-07 2024-12-05 Research Institute At Nationwide Children' S Hospital Products and methods for myelin protein zero silencing and treating cmt1b disease
WO2023060221A2 (en) 2021-10-08 2023-04-13 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
US20230140196A1 (en) 2021-10-12 2023-05-04 Selecta Biosciences, Inc. Viral vector dosing protocols
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
AU2022366947A1 (en) 2021-10-12 2024-05-02 Bridgebio Gene Therapy Llc Methods and compositions for treating leukodystrophies
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2023086615A1 (en) 2021-11-14 2023-05-19 Selecta Biosciences, Inc. Multiple dosing with viral vectors
WO2023087019A2 (en) 2021-11-15 2023-05-19 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
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
JP2025500891A (ja) 2021-12-15 2025-01-15 フラクティル ヘルス，インコーポレイテッド 代謝障害のための遺伝子治療
EP4453013A1 (en) 2021-12-21 2024-10-30 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
WO2023125481A1 (zh) 2021-12-28 2023-07-06 成都弘基生物科技有限公司 经过修饰的aav衣壳蛋白及其用途
TW202340467A (zh) 2022-01-10 2023-10-16 賓州大學委員會 有用於治療c9orf72介導之病症之組成物及方法
JP2025504898A (ja) 2022-01-24 2025-02-19 ライフエディット セラピューティクス，インコーポレイティド Ｒｎａ誘導型ヌクレアーゼ及びその活性断片及びバリアント、並びに使用方法
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
AR128422A1 (es) 2022-02-02 2024-05-08 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
EP4472657A4 (en) 2022-02-04 2026-02-18 M6P Therapeutics Inc COMPOSITIONS AND METHODS OF USE OF A TWO-PROMOTER VECTOR FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS
WO2023168400A2 (en) 2022-03-03 2023-09-07 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
US20230357437A1 (en) 2022-03-09 2023-11-09 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
JPWO2023182476A1 (da) * 2022-03-25 2023-09-28
EP4504950A1 (en) 2022-04-01 2025-02-12 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
US20250250326A1 (en) 2022-04-06 2025-08-07 The Trustees Of The University Of Pennsylvania Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
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
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
WO2023201207A1 (en) 2022-04-11 2023-10-19 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
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023205610A2 (en) 2022-04-18 2023-10-26 Regenxbio Inc. Hybrid aav capsids
TW202417633A (zh) 2022-05-03 2024-05-01 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抑制劑
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
JP2025523399A (ja) 2022-06-08 2025-07-23 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 変異型又は病原性ｋｃｎｑ３発現に関連する疾患又は状態を治療するための生成物及び方法
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
JP2025526816A (ja) 2022-08-11 2025-08-15 カーティザン セラピューティクス，インコーポレーテッド 免疫グロブリンプロテアーゼおよびその融合体に関する組成物および方法
AU2023323547A1 (en) 2022-08-12 2025-03-20 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
KR20250052387A (ko) 2022-08-25 2025-04-18 다케다 야쿠힌 고교 가부시키가이샤 파브리병의 치료에서의 사용을 위한 조성물
WO2024044892A1 (zh) * 2022-08-29 2024-03-07 上海目镜生物医药科技有限公司 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用
UY40443A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía con vectores de genoterapia con aav
UY40442A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav
EP4590838A1 (en) 2022-09-23 2025-07-30 Sarepta Therapeutics, Inc. Recombinant aav vectors for treating muscular dystrophy
EP4593955A1 (en) 2022-09-30 2025-08-06 RegenxBio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
JP2025534666A (ja) 2022-10-11 2025-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
WO2024081706A1 (en) 2022-10-11 2024-04-18 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
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
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
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
KR20250138783A (ko) 2023-02-01 2025-09-22 사렙타 쎄러퓨틱스 인코퍼레이티드 rAAV 생산 방법
WO2024168089A2 (en) * 2023-02-07 2024-08-15 Duke University Compositions comprising aavs and methods for treating gastrointestinal diseases
EP4662231A1 (en) 2023-02-10 2025-12-17 Expression Therapeutics, LLC Lentiviral system
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
CN120882872A (zh) 2023-03-17 2025-10-31 罗切斯特大学 核酶介导的rna组装和表达
CN121285568A (zh) 2023-03-30 2026-01-06 制药五有限责任公司 编码视杆细胞源性视锥细胞活力因子和人IgK信号序列的载体
JP2026513932A (ja) 2023-04-07 2026-05-01 リジェネクスバイオ インコーポレイテッド 組換えａａｖ生成のための組成物及び方法
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4695396A1 (en) 2023-04-10 2026-02-18 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
EP4694904A2 (en) 2023-04-13 2026-02-18 REGENXBIO Inc. Targeting aav capsids, methods of manufacturing and using same
JP2026516715A (ja) 2023-04-18 2026-05-26 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 肢帯型筋ジストロフィーｒ９及び先天性筋ジストロフィー１ｃを治療するための遺伝子療法
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
AU2024265705A1 (en) 2023-05-02 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases
AU2024266138A1 (en) 2023-05-03 2025-11-13 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
EP4709866A2 (en) 2023-05-07 2026-03-18 REGENXBIO Inc. Compositions and methods for recombinant aav production
KR20260010739A (ko) 2023-05-11 2026-01-21 유니버시티 하스피탈즈 클리블랜드 메디컬 센터 항불안 치료
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents 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
WO2024254319A1 (en) 2023-06-07 2024-12-12 Research Institute At Nationwide Children's Hospital Gene therapy for lysosomal acid lipase deficiency (lal-d)
JP2026522354A (ja) 2023-06-12 2026-07-07 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 ムコ多糖症ｉｉｉｂのためのａａｖ遺伝子療法
EP4727966A1 (en) 2023-06-13 2026-04-22 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom
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
IL326390A (en) 2023-08-10 2026-04-01 Univ 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
KR20260114566A (ko) 2023-11-14 2026-07-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도
AU2024386366A1 (en) 2023-11-23 2026-06-04 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
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
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
TW202613313A (zh) 2024-05-31 2026-04-01 美商薩羅塔治療公司 肌向性ｒａａｖ
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
WO2026073032A1 (en) 2024-09-27 2026-04-02 Shape Therapeutics Inc. Constructs for improved aav production
WO2026072752A1 (en) 2024-09-27 2026-04-02 Pharma Cinq, Llc Rod-derived cone viability factor fusion protein
WO2026094009A1 (en) 2024-11-04 2026-05-07 Insmed Incorporated Compositions and methods for treating duchenne muscular dystrophy
WO2026102076A1 (en) 2024-11-07 2026-05-15 Juno Therapeutics, Inc. Aav production method
WO2026114267A1 (en) 2024-11-29 2026-06-04 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating alzheimer's disease in primates
EP4763209A1 (en) 2024-12-17 2026-06-24 Institut National de la Santé et de la Recherche Médicale Use of (alpha)s-setmar agonist for preventing or treating glioblastoma
WO2026137014A1 (en) 2024-12-20 2026-06-25 Samsung Bioepis Co., Ltd. Methods of purifying recombinant adeno-associated viruses
WO2026150302A1 (en) 2025-01-09 2026-07-16 Novartis Ag Novel aav rep/cap encoding constructs
WO2026150324A1 (en) 2025-01-10 2026-07-16 Sarepta Therapeutics, Inc. Compositions and methods for treating dysferlinopathies

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
EP1696036B1 (en) 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Use of recombinant adeno-associated virus in the manufacture of a medicament for gene therapy via muscle cells
WO1998010086A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
CA2264482A1 (en) 1996-09-06 1998-03-12 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
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
CA2274146C (en) 1996-12-05 2007-02-06 Introgene 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
NZ503426A (en) 1997-09-18 2001-08-31 Holec Holland N Electromagnetic switch actuator with switch on and switch off coils
AU9774998A (en) 1997-09-19 1999-04-12 Trustees Of The University Of Pennsylvania, The Method for gene transfer using bcl2 and compositions useful therein
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
JP2001517454A (ja) * 1997-09-19 2001-10-09 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産生に有用な方法および細胞株
ATE286138T1 (de) 1998-03-20 2005-01-15 Univ Pennsylvania Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren
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
WO1999058140A1 (en) * 1998-05-11 1999-11-18 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
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
DE69936104T2 (de) * 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
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
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
JP2003501067A (ja) 1999-06-02 2003-01-14 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 ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法
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
WO2001023001A2 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Rapid peg-modification
AU7841400A (en) * 1999-10-01 2001-05-10 Genovo, Incorporated Production of recombinant aav using adenovirus comprising aav rep/cap genes
US7115391B1 (en) * 1999-10-01 2006-10-03 Genovo, Inc. Production of recombinant AAV using adenovirus comprising AAV rep/cap genes
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
JP2003523180A (ja) 1999-12-03 2003-08-05 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 多重のパッケージングシグナルを用いる組み換えアデノウイルスのパッケージングおよび収量を増加するための組成物および方法
EP1228234A2 (en) * 2000-03-14 2002-08-07 Neurologix, Inc. Production of chimeric capsid vectors
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
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
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
CN1293204C (zh) 2000-08-30 2007-01-03 戴诺生物技术有限公司 等位基因的测定方法
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
CN103589692B (zh) 2001-11-13 2018-07-24 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
DK2359869T3 (da) * 2001-12-17 2019-04-15 Univ Pennsylvania Sekvenser af adeno-associeret virus (AAV) serotype 8, vektorer indeholdende disse og anvendelser deraf
EP1463805B1 (en) * 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
AU2002367943A1 (en) * 2001-12-18 2003-12-22 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
EP1668143B1 (en) * 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US9819463B2 (en) 2016-02-18 2017-11-14 Huawei Technologies Co., Ltd. Method and apparatus for transmitting data in a wireless communication system

Also Published As

Publication number Publication date
US20090227030A1 (en) 2009-09-10
JP5649529B2 (ja) 2015-01-07
CY1121446T1 (el) 2020-05-29
US20080075737A1 (en) 2008-03-27
US20190017068A1 (en) 2019-01-17
PT2359869T (pt) 2019-04-16
EP3517134B1 (en) 2024-01-17
US20210348190A1 (en) 2021-11-11
WO2003052051A3 (en) 2004-02-19
JP4810062B2 (ja) 2011-11-09
US10301650B2 (en) 2019-05-28
AU2002360291A1 (en) 2003-06-30
EP2359869A2 (en) 2011-08-24
US8318480B2 (en) 2012-11-27
US10266846B2 (en) 2019-04-23
US8962332B2 (en) 2015-02-24
EP1453547B1 (en) 2016-09-21
US20140004143A1 (en) 2014-01-02
US20170088858A1 (en) 2017-03-30
AU2002360291A8 (en) 2003-06-30
EP3517134A1 (en) 2019-07-31
ES2717377T3 (es) 2019-06-20
JP2005512533A (ja) 2005-05-12
US9493788B2 (en) 2016-11-15
US20150139953A1 (en) 2015-05-21
US11390883B2 (en) 2022-07-19
PT1453547T (pt) 2016-12-28
US7282199B2 (en) 2007-10-16
CA2469785A1 (en) 2003-06-26
US20030228282A1 (en) 2003-12-11
US9677089B2 (en) 2017-06-13
US9587250B2 (en) 2017-03-07
EP1453547A4 (en) 2006-04-05
US20200283799A1 (en) 2020-09-10
US11396663B2 (en) 2022-07-26
US20220025399A1 (en) 2022-01-27
ES2602352T3 (es) 2017-02-20
EP2359869A3 (en) 2014-01-08
EP1453547A2 (en) 2004-09-08
US20170306354A1 (en) 2017-10-26
US7790449B2 (en) 2010-09-07
US20160201088A1 (en) 2016-07-14
DK1453547T3 (da) 2016-12-05
FI3517134T3 (fi) 2024-04-03
JP2011254821A (ja) 2011-12-22
CA2469785C (en) 2014-04-22
WO2003052051A2 (en) 2003-06-26
EP2359869B1 (en) 2018-12-26
CY1120162T1 (el) 2018-12-12
US10590435B2 (en) 2020-03-17
US8962330B2 (en) 2015-02-24
US20190292563A1 (en) 2019-09-26
US20080075740A1 (en) 2008-03-27
US20150176027A1 (en) 2015-06-25
ES2975413T3 (es) 2024-07-05

Similar Documents

Publication Publication Date Title
US11390883B2 (en) Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
US10308958B2 (en) Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
HK40012107A (en) Adeno-associated virus (aav) serotype 8 sequences, vectors containing same and uses therefor