BE2021C533I2 - - Google Patents

Download PDF

Info

Publication number
BE2021C533I2
BE2021C533I2 BE2021C533C BE2021C533C BE2021C533I2 BE 2021C533 I2 BE2021C533 I2 BE 2021C533I2 BE 2021C533 C BE2021C533 C BE 2021C533C BE 2021C533 C BE2021C533 C BE 2021C533C BE 2021C533 I2 BE2021C533 I2 BE 2021C533I2
Authority
BE
Belgium
Application number
BE2021C533C
Other languages
French (fr)
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=34426044&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BE2021C533(I2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of BE2021C533I2 publication Critical patent/BE2021C533I2/fr

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/644Coagulation factor IXa (3.4.21.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21022Coagulation factor IXa (3.4.21.22)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
BE2021C533C 2003-09-30 2021-08-30 BE2021C533I2 (enrdf_load_html_response)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US50822603P 2003-09-30 2003-09-30
US56654604P 2004-04-29 2004-04-29
EP17153156.9A EP3211085B1 (en) 2003-09-30 2004-09-30 Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor

Publications (1)

Publication Number Publication Date
BE2021C533I2 true BE2021C533I2 (enrdf_load_html_response) 2024-01-12

Family

ID=34426044

Family Applications (1)

Application Number Title Priority Date Filing Date
BE2021C533C BE2021C533I2 (enrdf_load_html_response) 2003-09-30 2021-08-30

Country Status (17)

Country Link
US (13) US7906111B2 (enrdf_load_html_response)
EP (7) EP2292779B1 (enrdf_load_html_response)
JP (9) JP5054975B2 (enrdf_load_html_response)
CN (4) CN102199626B (enrdf_load_html_response)
AU (2) AU2004278684B2 (enrdf_load_html_response)
BE (1) BE2021C533I2 (enrdf_load_html_response)
CA (3) CA3072423A1 (enrdf_load_html_response)
CY (3) CY1119833T1 (enrdf_load_html_response)
DK (4) DK2292779T3 (enrdf_load_html_response)
ES (5) ES2648241T3 (enrdf_load_html_response)
FR (1) FR21C1037I1 (enrdf_load_html_response)
HU (4) HUE035567T2 (enrdf_load_html_response)
NZ (1) NZ545628A (enrdf_load_html_response)
PL (2) PL2292780T3 (enrdf_load_html_response)
PT (2) PT2292780T (enrdf_load_html_response)
SI (2) SI2292780T1 (enrdf_load_html_response)
WO (1) WO2005033321A2 (enrdf_load_html_response)

Families Citing this family (613)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX359371B (es) * 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
WO2003052051A2 (en) * 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
HUE035567T2 (en) * 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
ATE497785T1 (de) 2004-04-28 2011-02-15 Univ Pennsylvania Sequenzielle freisetzung immunogener moleküle über adenoviren und adeno-assoziierte viren vermittelte abgaben
CA2563500C (en) 2004-04-28 2016-06-28 The Trustees Of The University Of Pennsylvania Immunization regimen with e4-deleted adenovirus prime and e1-deleted adenovirus boost
CN102994549B (zh) 2005-04-07 2015-02-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2007120542A2 (en) * 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
WO2007118245A2 (en) 2006-04-07 2007-10-18 The Board Of Regents Of The University Of Texas System Methods and compositions related to adenoassociated virus-phage particles
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
WO2008027084A2 (en) * 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
LT2191001T (lt) * 2007-04-09 2016-10-10 University Of Florida Research Foundation, Inc. Raav vektoriaus kompozicijos, turinčios tirozin-modifikuotų kapsidės baltymų ir jų panaudojimo būdai
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009108274A2 (en) * 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
PL2425000T3 (pl) * 2009-04-30 2019-08-30 The Trustees Of The University Of Pennsylvania Kompozycje do kierowania komórek przewodzących do dróg oddechowych zawierające konstrukty wirusów związanych z adenowirusami
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9133479B2 (en) * 2009-06-03 2015-09-15 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
KR20130040844A (ko) 2010-03-29 2013-04-24 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 약학적으로 유발된 전이유전자 제거 시스템
EP3536781A1 (en) 2010-04-23 2019-09-11 University of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2011133901A2 (en) 2010-04-23 2011-10-27 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
EP2673289B1 (en) * 2011-02-10 2023-05-03 The University of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
WO2012112832A1 (en) 2011-02-17 2012-08-23 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
US20140031418A1 (en) 2011-04-20 2014-01-30 The Trustees Of The University Of Pennsylvania Regimens and Compositions for AAV-Mediated Passive Immunization of Airborne Pathogens
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
HUE034510T2 (en) 2011-04-22 2018-02-28 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
US20130039888A1 (en) * 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
EP3147295B2 (en) * 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
US8859256B2 (en) 2011-10-05 2014-10-14 Genelux Corporation Method for detecting replication or colonization of a biological therapeutic
WO2013063019A1 (en) 2011-10-28 2013-05-02 The Wistar Institute Of Anatomy And Biology Methods and compositions for enhancing the therapeutic effect of anti-tumor t cells
US10238755B2 (en) 2011-11-30 2019-03-26 The Wistar Institute Of Anatomy And Biology Methods and compositions for regulation of cell aging, carcinogenesis and reprogramming
EP2601968A1 (en) * 2011-12-06 2013-06-12 Deutsches Krebsforschungszentrum HPV derived polynucleic acids for therapy
US9611305B2 (en) 2012-01-06 2017-04-04 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
US20140087362A1 (en) 2012-03-16 2014-03-27 Aladar A. Szalay Methods for assessing effectiveness and monitoring oncolytic virus treatment
US20130280170A1 (en) 2012-04-20 2013-10-24 Aladar A. Szalay Imaging methods for oncolytic virus therapy
JP6385920B2 (ja) * 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
WO2014011210A1 (en) 2012-07-11 2014-01-16 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
BR112015002168A2 (pt) 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
CN104797593B (zh) * 2012-09-28 2020-05-08 北卡罗来纳-查佩尔山大学 靶向少突胶质细胞的aav载体
US20140140959A1 (en) 2012-10-05 2014-05-22 Aladar A. Szalay Energy Absorbing-Based Diagnostic and Therapeutic Methods Employing Nucleic Acid Molecules Encoding Chromophore-Producing Enzymes
EP2749569A1 (en) 2012-12-28 2014-07-02 Annibale Alessandro Puca Variant of BPIFB4 protein
US8957044B2 (en) 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
CA2905952A1 (en) * 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
AU2014241078B2 (en) 2013-03-14 2020-03-12 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with ALS
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
PL2968605T3 (pl) * 2013-03-15 2022-11-07 The University Of North Carolina At Chapel Hill Metody i kompozycje dla wektorów aav podwójnie wiążących glikan
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
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
AU2014324717B2 (en) 2013-09-26 2020-10-08 University Of Florida Research Foundation, Inc. Synthetic combinatorial AAV capsid library for targeted gene therapy
EP3054995A1 (en) * 2013-10-07 2016-08-17 Kiromic, LLC Compositions and methods for treating cardiovascular diseases using disease-specific promoter
NZ758023A (en) * 2013-10-11 2021-12-24 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
US10238700B2 (en) 2014-01-02 2019-03-26 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
HRP20201544T1 (hr) 2014-03-09 2021-02-19 The Trustees Of The University Of Pennsylvania Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc)
ES2990174T3 (es) 2014-03-10 2024-11-29 Uniqure Ip Bv Vectores de AAV mejorados producidos en células de insecto
CN112626125A (zh) 2014-03-17 2021-04-09 阿德夫拉姆生物技术股份有限公司 用于视锥细胞中增强的基因表达的组合物和方法
AU2015231294B2 (en) 2014-03-18 2020-10-29 University Of Massachusetts rAAV-based compositions and methods for treating amyotrophic lateral sclerosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
EP3134115A4 (en) 2014-04-25 2017-10-25 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
WO2015164778A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennysylvania Ldlr variants and their use in compositions for reducing cholesterol levels
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
PT3142750T (pt) 2014-05-13 2020-09-22 Univ Pennsylvania Composições que compreendem aav expressando construções de anticorpos duplos e suas utilizações
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
SG10201913259QA (en) 2014-09-24 2020-02-27 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
EP3795580A1 (en) 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
DK3207130T3 (da) 2014-10-14 2019-11-11 Halozyme Inc Sammensætninger af Adenosin Deaminase-2 (ADA2), varianter deraf og fremgangsmåder til anvendelse af samme
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
US10907130B2 (en) * 2014-11-05 2021-02-02 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
AU2015346162B2 (en) 2014-11-14 2022-02-10 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
EP3221453A4 (en) * 2014-11-21 2018-09-12 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
WO2016100575A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US11033638B2 (en) 2015-01-07 2021-06-15 Universität Autonoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
CA2972811A1 (en) 2015-01-16 2016-07-21 University Of Washington Novel micro-dystrophins and related methods of use
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP2018509164A (ja) 2015-03-10 2018-04-05 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 組換えGlut1アデノ随伴ウイルスベクターコンストラクトおよびGlut1発現を回復させる方法
CN107532177A (zh) 2015-03-24 2018-01-02 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
CA3021949C (en) 2015-04-24 2023-10-17 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
WO2016179496A1 (en) 2015-05-07 2016-11-10 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
WO2016183297A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
CN108135994B (zh) 2015-05-13 2022-01-25 宾夕法尼亚州大学信托人 Aav-介导的抗-流感抗体的表达及其使用方法
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
EP3325018B1 (en) 2015-07-22 2025-01-15 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
CA2994160C (en) * 2015-07-30 2021-08-10 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
IL295616A (en) 2015-07-31 2022-10-01 Us Health Adapted cells and treatment methods
CA2994552A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glucagon-like peptide 1 and use thereof in compositions for treating metabolic diseases
EP4177346A3 (en) 2015-08-25 2023-07-26 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
US11117942B2 (en) 2015-08-31 2021-09-14 The Trustees Of The University Of Pennsylvania AAV-EPO for treating companion animals
RU2727411C2 (ru) 2015-09-24 2020-07-21 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Композиция и способ для лечения заболевания, опосредованного комплементом
BR112018003665A2 (pt) 2015-09-28 2018-09-25 Univ North Carolina Chapel Hill métodos e composições para vetores virais de evasão de anticorpo
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
EP3362571A4 (en) 2015-10-13 2019-07-10 Duke University GENOM 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
DK3364997T5 (da) 2015-10-22 2024-09-30 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
WO2017070516A1 (en) * 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070491A1 (en) * 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
CN108779167A (zh) 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US10548947B2 (en) 2015-11-05 2020-02-04 Bamboo Therapeutics, Inc. Modified Friedreich ataxia genes and vectors for gene therapy
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR DETECTION OF NEUTRALIZING ANTIBODIES OF VAA
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
EP3386537B1 (en) 2015-12-11 2021-10-20 Massachusetts Eye and Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017100671A1 (en) * 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
US10889832B2 (en) 2015-12-11 2021-01-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
EP4215605A1 (en) 2015-12-11 2023-07-26 The Trustees of The University of Pennsylvania Scalable purification method for aav8
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
MX2018007230A (es) 2015-12-14 2018-11-09 Univ Pennsylvania Terapia genica para trastornos oculares.
CA3008280A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
EP3389723A1 (en) 2015-12-14 2018-10-24 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
BR112018015751A2 (pt) 2016-02-03 2019-02-05 Univ Pennsylvania terapia gênica para tratamento de mucopolissacaridose tipo i
LT3411484T (lt) 2016-02-05 2023-11-27 Emory University Viengrandžio arba sau pačiam komplementaraus adeno-asocijuoto viruso 9 injekcija į smegenų skystį
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
CA3019847A1 (en) 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
JP7171439B2 (ja) 2016-04-15 2022-11-15 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ii型を処置するための遺伝子療法
AU2017248656B2 (en) * 2016-04-15 2023-07-27 The Trustees Of The University Of Pennsylvania Novel AAV8 mutant capsids and compositions containing same
PE20181922A1 (es) 2016-04-15 2018-12-11 Univ Pennsylvania Terapia genica para tratar la hemofilia a
EP3442999A2 (en) 2016-04-15 2019-02-20 Alpine Immune Sciences, Inc. Icos ligand variant immunomodulatory proteins and uses thereof
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
AU2017248830B2 (en) 2016-04-15 2023-03-09 Alpine Immune Sciences, Inc. CD80 variant immunomodulatory proteins and uses thereof
KR102574810B1 (ko) 2016-04-15 2023-09-08 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 습성 연령 관련 황반 변성의 치료를 위한 조성물
WO2017184463A1 (en) 2016-04-17 2017-10-26 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3452495B1 (en) * 2016-05-03 2021-06-23 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
SG11201809684YA (en) 2016-05-13 2018-11-29 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
MX2018014154A (es) 2016-05-18 2019-05-06 Voyager Therapeutics Inc Polinucleotidos moduladores.
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
AU2017284198B2 (en) 2016-06-13 2023-09-28 The University Of North Carolina At Chapel Hill Optimized CLN1 genes and expression cassettes and their use
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
IL297095B1 (en) 2016-07-08 2025-06-01 Univ Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
KR102604510B1 (ko) * 2016-07-26 2023-11-23 바이오마린 파머수티컬 인크. 신규 아데노-관련 바이러스 캡시드 단백질
US11834490B2 (en) 2016-07-28 2023-12-05 Alpine Immune Sciences, Inc. CD112 variant immunomodulatory proteins and uses thereof
WO2018022946A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd155 variant immunomodulatory proteins and uses thereof
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
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
EP3506817A4 (en) 2016-08-30 2020-07-22 The Regents of The University of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
EP3526333A4 (en) 2016-10-13 2020-07-29 University of Massachusetts AAV CAPSIDE DESIGNS
JP7181862B2 (ja) 2016-10-18 2022-12-01 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 腫瘍浸潤リンパ球および治療の方法
WO2018075798A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CA3040296A1 (en) 2016-10-20 2018-04-26 Alpine Immune Sciences, Inc. Secretable variant immunomodulatory proteins and engineered cell therapy
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
WO2018100054A1 (en) 2016-12-01 2018-06-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Pharmaceutical compositions for the treatment of retinal degenerative diseases
US11382941B2 (en) 2016-12-30 2022-07-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating Phenylketonuria
CN110290814A (zh) 2016-12-30 2019-09-27 宾夕法尼亚州立大学托管会 用于治疗威尔逊病的基因疗法
WO2018134168A1 (en) 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
JP2020506705A (ja) 2017-02-01 2020-03-05 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア シトルリン血症を処置するための遺伝子療法
WO2018148462A1 (en) 2017-02-09 2018-08-16 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and compositions and methods thereof
EP3595688A4 (en) 2017-02-20 2020-12-30 The Trustees Of The University Of Pennsylvania GENE THERAPY TO TREAT FAMILY HYPERCHOLESTEROLEMIA
CA3114549A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
WO2018154096A1 (en) 2017-02-24 2018-08-30 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
US10786568B2 (en) * 2017-02-28 2020-09-29 The Trustees Of The University Of Pennsylvania AAV mediated influenza vaccines
SG11201907714UA (en) * 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JP7211960B2 (ja) 2017-03-01 2023-01-24 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患の遺伝子治療
US11376321B2 (en) 2017-03-02 2022-07-05 Genethon Method for removing anti-AAV antibodies from a blood-derived composition
CA3053812A1 (en) 2017-03-16 2018-09-20 Alpine Immune Sciences, Inc. Pd-l2 variant immunomodulatory proteins and uses thereof
EP4306537A3 (en) 2017-03-16 2024-03-06 Alpine Immune Sciences, Inc. Pd-l1 variant immunomodulatory proteins and uses thereof
CA3054068A1 (en) 2017-03-16 2018-09-20 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
US11946074B2 (en) 2017-03-24 2024-04-02 Lankenau Institute For Medical Research Methods and compositions for inducible extracellular membrane capture of monoclonal immunoglobulins secreted by hybridomas
EP3613856A4 (en) 2017-03-31 2020-12-23 Staidson(Beijing) Biopharmaceuticals Co., Ltd. SHORT RNA EXPRESSION CASSETTE IN HAIRPIN, POLYNUCLEOTIDIAL SEQUENCE WEARING IT AND CORRESPONDING APPLICATION
US11499154B2 (en) 2017-04-10 2022-11-15 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
TW201903146A (zh) 2017-04-14 2019-01-16 美商銳進科斯生物股份有限公司 使用由人類神經或膠細胞產生的重組人類艾杜糖醛酸鹽 (iduronate)-2-硫酸酯酶 (ids) 之黏多醣病ii之治療
EP3612190A4 (en) * 2017-04-17 2021-01-20 University of Florida Research Foundation, Incorporated REGULATION OF RAN TRANSLATION THROUGH PKR AND EIF2A-P
EP3634986A4 (en) 2017-04-24 2021-09-08 The Trustees of The University of Pennsylvania GENE THERAPY FOR EYE DISORDERS
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
AU2018265414B2 (en) 2017-05-10 2022-01-20 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (APP)-dependence of viruses
SG10201912401QA (en) 2017-05-11 2020-02-27 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
EP3630986A4 (en) 2017-05-31 2021-08-11 The Trustees of The University of Pennsylvania GENE THERAPY FOR THE TREATMENT OF PEROXYSOMA DISORDERS
JP2020527335A (ja) 2017-06-14 2020-09-10 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患のための遺伝子療法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3645052A4 (en) 2017-06-30 2021-07-28 Regents of the University of California ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE
JP2020530307A (ja) 2017-06-30 2020-10-22 インティマ・バイオサイエンス,インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
BR112020000063A2 (pt) 2017-07-06 2020-07-14 The Trustees Of The University Of Pennsylvania terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i
CA3069045A1 (en) 2017-07-07 2019-01-10 Genethon Novel polynucleotides encoding a human fkrp protein
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
CA3067509A1 (en) 2017-07-27 2019-01-31 Novartis Ag Sheddase resistant trem2 variants
AU2018311069A1 (en) 2017-08-03 2020-02-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
BR112020003571A2 (pt) 2017-08-28 2020-08-25 The Regents Of The University Of California variantes capsidiais de vírus adenoassociado e métodos de uso das mesmas
CR20200165A (es) 2017-09-20 2020-06-05 4D Molecular Therapeutics Inc Variantes de cápsides de virus adenoasociados y métodos de uso de estas
US11739330B2 (en) 2017-09-22 2023-08-29 University Of Massachusetts SOD1 dual expression vectors and uses thereof
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ii型を治療するための遺伝子療法
CA3076214A1 (en) 2017-09-26 2019-04-04 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders
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.
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
SG11202003479TA (en) 2017-10-18 2020-05-28 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
WO2019079494A1 (en) 2017-10-18 2019-04-25 Regenxbio, Inc. TREATMENT OF OCULAR DISEASES AND METASTATIC COLON CANCER WITH A VEGF TRAP WITH HUMAN POST-TRANSLATIONAL MODIFICATION
KR102776394B1 (ko) 2017-10-18 2025-03-10 알파인 이뮨 사이언시즈, 인코포레이티드 변이체 icos 리간드 면역조절 단백질 및 관련 조성물 및 방법
EP3697448A1 (en) 2017-10-20 2020-08-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant aav9-derived vector
FI3717636T3 (fi) 2017-11-27 2023-06-01 4D Molecular Therapeutics Inc Adenoassosioidun viruksen kapsidivariantteja ja käyttö angiogeneesin estämisessä
EP3717652A4 (en) 2017-11-30 2021-11-24 The Trustees of the University of Pennsylvania GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS TYPE IIIB
BR112020010735A2 (pt) 2017-11-30 2020-11-10 The Trustees Of The University Of Pennsylvania terapia genética para mucopolissacaridose iii a
CN111936171A (zh) 2017-12-19 2020-11-13 阿库斯股份有限公司 Aav介导的治疗性抗体到内耳的递送
BR112020013236A2 (pt) 2018-01-03 2020-12-01 Alpine Immune Sciences, Inc. proteínas imunomoduladoras de múltiplos domínios e métodos de seu uso
US12195767B2 (en) 2018-01-17 2025-01-14 Adrenas Therapeutics, Inc. Adeno-associated virus gene therapy for 21-hydroxylase deficiency
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
MX2020008133A (es) 2018-02-01 2020-10-14 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas.
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
IL276859B1 (en) * 2018-02-27 2025-08-01 Univ Pennsylvania Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses therefor
JP7498665B2 (ja) * 2018-02-27 2024-06-12 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 新規アデノ随伴ウイルス(aav)ベクター、低減されたカプシド脱アミド化を有するaavベクター、およびその使用
SG11202008687XA (en) * 2018-03-16 2020-10-29 Res Inst Nationwide Childrens Hospital Increasing tissue specific gene delivery by capsid modification
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
MX2020010464A (es) 2018-04-03 2021-01-29 Vectores de virus que evitan anticuerpos.
KR20210006358A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 안 조직을 표적으로 하기 위한 바이러스 벡터
EP3775266A4 (en) 2018-04-05 2021-06-30 Massachusetts Eye and Ear Infirmary METHOD OF MANUFACTURING AND USING COMBINATIONAL BAR-CODED NUCLEIC ACID LIBRARIES WITH DEFINED VARIATION
US12037362B2 (en) 2018-04-05 2024-07-16 Genethon Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism
EP3781671A4 (en) 2018-04-18 2022-01-26 The Trustees of Columbia University in the City of New York Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
WO2019212921A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Scalable clarification process for recombinant aav production
WO2019212922A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
IL278393B2 (en) 2018-05-08 2023-11-01 Univ Rutgers AAV compatible polymerization proteins bind to laminin
JP2021522811A (ja) 2018-05-09 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド フェニルケトン尿症の治療方法
WO2019217911A1 (en) 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Liver-specific tropism of adeno-associated viruses
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
WO2019221992A1 (en) 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒(aav)之組成物和方法
UY38247A (es) 2018-05-30 2019-12-31 Novartis Ag Anticuerpos frente a entpd2, terapias de combinación y métodos de uso de los anticuerpos y las terapias de combinación
US11505782B2 (en) 2018-06-04 2022-11-22 Calidi Biotherapeutics, Inc. Cell-based vehicles for potentiation of viral therapy
EP3801584B1 (en) 2018-06-04 2023-09-27 Calidi Biotherapeutics, Inc. Cell-based vehicles for potentiation of viral therapy
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2019241315A1 (en) 2018-06-12 2019-12-19 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CN112469822A (zh) 2018-06-14 2021-03-09 再生生物股份有限公司 用于重组aav生产的阴离子交换色谱
US12065476B2 (en) 2018-06-15 2024-08-20 Alpine Immune Sciences, Inc. PD-1 variant immunomodulatory proteins and uses thereof
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
MX2021000443A (es) 2018-07-12 2021-05-28 Spacecraft Seven Llc Vectores de terapia génica para el tratamiento de la enfermedad de danon.
EP3823981A4 (en) 2018-07-17 2022-04-06 Neuromyon Inc. Treatment of neuropathy with dna constructs expressing igf-1 isoforms
WO2020018691A1 (en) * 2018-07-18 2020-01-23 The General Hospital Corporation Modified t cells and methods of their use
EP3826719A1 (en) 2018-07-24 2021-06-02 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
CA3108113A1 (en) 2018-08-10 2020-02-13 Michael GILLMEISTER Scalable method for recombinant aav production
WO2020047161A2 (en) 2018-08-28 2020-03-05 Actym Therapeutics, Inc. Engineered immunostimulatory bacterial strains and uses thereof
EP3843774A4 (en) 2018-08-30 2022-08-31 Tenaya Therapeutics, Inc. REPROGRAMMING HEART CELLS WITH MYOCARDINE AND ASCL1
SG11202103102WA (en) 2018-09-26 2021-04-29 California Inst Of Techn Adeno-associated virus compositions for targeted gene therapy
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP7289033B2 (ja) 2018-10-01 2023-06-09 徹 宮崎 神経変性疾患治療剤
SG11202102942VA (en) 2018-10-01 2021-04-29 Univ Pennsylvania Compositions useful for treating gm1 gangliosidosis
EP3861113A1 (en) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
TW202028458A (zh) * 2018-10-05 2020-08-01 美商航海家醫療公司 編碼腺相關病毒(aav)生產蛋白之經基因工程化核酸構築體
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2019360937A1 (en) 2018-10-15 2021-05-20 Voyager Therapeutics, Inc. Expression vectors for large-scale production of rAAV in the baculovirus/Sf9 system
WO2020081415A1 (en) 2018-10-15 2020-04-23 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
KR20210082205A (ko) 2018-10-22 2021-07-02 유니버시티 오브 로체스터 레트로바이러스 인테그라제-Cas9 융합 단백질을 이용한 유도된 비상동 DNA 삽입에 의한 게놈 편집
WO2020086742A1 (en) 2018-10-24 2020-04-30 Obsidian Therapeutics, Inc. Er tunable protein regulation
WO2020097269A1 (en) 2018-11-06 2020-05-14 Calidi Biotherapeutics, Inc. Enhanced systems for cell-mediated oncolytic viral therapy
CA3119618A1 (en) 2018-11-16 2020-05-22 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
JP7541516B2 (ja) 2018-11-21 2024-08-28 インダプタ セラピューティクス インコーポレイテッド ナチュラルキラー(nk)細胞サブセットの増大のための方法ならびに関連する組成物および方法
AU2019389151B2 (en) 2018-11-30 2025-07-24 Alpine Immune Sciences, Inc. CD86 variant immunomodulatory proteins and uses thereof
MX2021006359A (es) * 2018-11-30 2021-08-11 Novartis Ag Vectores virales de vaa y sus usos.
US20220089670A1 (en) 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
PL3906066T3 (pl) 2019-01-04 2024-09-02 Ultragenyx Pharmaceutical Inc. Konstrukty do terapii genowej do leczenia choroby wilsona
JP7416451B2 (ja) 2019-01-14 2024-01-17 ユニバーシティ オブ ロチェスター CRISPR-Casによる標的化された核内RNA切断及びポリアデニル化
MX2021008542A (es) 2019-01-18 2021-11-12 Voyager Therapeutics Inc Métodos y sistemas para la producción de partículas de aav.
US20230193315A1 (en) 2019-01-31 2023-06-22 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
KR20210131370A (ko) 2019-02-22 2021-11-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
CN113677801A (zh) 2019-02-25 2021-11-19 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
EP3931335A2 (en) 2019-02-25 2022-01-05 Novartis AG Compositions and methods to treat bietti crystalline dystrophy
KR20210132095A (ko) 2019-02-26 2021-11-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 크라베병의 치료에 유용한 조성물
EP3931210A1 (en) 2019-02-27 2022-01-05 Actym Therapeutics, Inc. Immunostimulatory bacteria engineered to colonize tumors, tumor-resident immune cells, and the tumor microenvironment
US12024709B2 (en) 2019-02-27 2024-07-02 Actym Therapeutics, Inc. Immunostimulatory bacteria engineered to colonize tumors, tumor-resident immune cells, and the tumor microenvironment
EP3941929A1 (en) 2019-03-21 2022-01-26 Stridebio, Inc. Recombinant adeno-associated virus vectors
CA3130196A1 (en) 2019-03-25 2020-10-01 Isabelle Richard Production of large-sized quasidystrophins using overlapping aav vectors
AU2020253435A1 (en) 2019-04-01 2021-11-18 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
SG11202110789VA (en) 2019-04-03 2021-10-28 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
WO2020210600A1 (en) 2019-04-11 2020-10-15 Regenxbio Inc. Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
TW202332458A (zh) 2019-04-19 2023-08-16 美商銳進科斯生物股份有限公司 腺相關病毒載體調配物及方法
AU2020262416A1 (en) 2019-04-24 2021-12-16 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
CA3134468A1 (en) * 2019-04-29 2020-11-05 Kalyani NAMBIAR Novel aav capsids and compositions containing same
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
PE20220930A1 (es) 2019-05-03 2022-05-31 Univ Pennsylvania Composiciones utiles en el tratamiento de leucodistrofia metacromatica
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP3969061A1 (en) 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
CA3135271A1 (en) 2019-05-28 2020-12-03 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
EP3976806A1 (en) 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Recombinant herpesvirales vector
WO2020261178A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
US20220380800A1 (en) 2019-07-02 2022-12-01 M6P Therapeutics (Switzerland) Llc Vector compositions and methods of using same for treatment of lysosomal storage disorders
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
FI3997214T3 (fi) 2019-07-11 2023-11-02 Centre Nat Rech Scient Kemiallisesti modifioitu adeno-associated virus
EP3997226A1 (en) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
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
WO2021016453A1 (en) 2019-07-23 2021-01-28 University Of Rochester Targeted rna cleavage with crispr-cas
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
US20230042103A1 (en) 2019-07-26 2023-02-09 Regenxbio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
US20220290182A1 (en) 2019-08-09 2022-09-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
AU2020334462A1 (en) 2019-08-16 2022-02-24 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting LAMA1 gene
WO2021034920A1 (en) * 2019-08-20 2021-02-25 St. Jude Children's Research Hospital, Inc. SCHIZOPHRENIA-RELATED MICRODELETION GENE 2510002D24Rik IS ESSENTIAL FOR SOCIAL MEMORY
TW202122419A (zh) 2019-08-26 2021-06-16 美商銳進科斯生物股份有限公司 以全人類轉譯後修飾之抗VEGF Fab治療糖尿病性視網膜病變
WO2021040736A1 (en) 2019-08-30 2021-03-04 Obsidian Therapeutics, Inc. Tandem cd19 car-based compositions and methods for immunotherapy
US20220307013A1 (en) 2019-08-30 2022-09-29 The Regents Of The University Of California Gene fragment overexpression screening methodologies, and uses thereof
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
US20220348937A1 (en) 2019-09-06 2022-11-03 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
CN114555814A (zh) 2019-09-13 2022-05-27 罗特格斯新泽西州立大学 Aav相容的层粘连蛋白-连接子聚合蛋白
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與entpd2抗體之組合療法
WO2021053559A1 (en) 2019-09-18 2021-03-25 Novartis Ag Entpd2 antibodies, combination therapies, and methods of using the antibodies and combination therapies
CN114521143B (zh) 2019-09-19 2025-03-14 吉尼松公司 减轻fkrp心脏毒性的基因治疗表达系统
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
CN114728049A (zh) 2019-10-07 2022-07-08 再生生物股份有限公司 腺相关病毒载体药物组合物和方法
WO2021071976A1 (en) 2019-10-08 2021-04-15 Exhaura, Ltd. Compositions and methods for ocular therapy
AU2020361533A1 (en) 2019-10-08 2022-04-28 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
EP4045525A1 (en) 2019-10-17 2022-08-24 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
WO2021092298A1 (en) * 2019-11-08 2021-05-14 President And Fellows Of Harvard College Viral capsid polypeptides
US20220396808A1 (en) * 2019-11-08 2022-12-15 President And Fellows Of Harvard College Viral capsid polypeptides
EP4061943A1 (en) 2019-11-19 2022-09-28 Institut National de la Santé et de la Recherche Médicale (INSERM) Antisense oligonucleotides and their use for the treatment of cancer
TW202134260A (zh) 2019-11-28 2021-09-16 美商銳進科斯生物股份有限公司 微小肌縮蛋白基因療法之構築體及其用途
EP4073257A1 (en) 2019-12-09 2022-10-19 UCL Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
IL293684A (en) 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
KR20220128632A (ko) 2019-12-31 2022-09-21 스완바이오 테라퓨틱스 리미티드 개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
JP2023511382A (ja) 2020-01-22 2023-03-17 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα-L-イズロニダーゼ(IDUA)を用いたムコ多糖症I型の治療
BR112022015183A2 (pt) 2020-01-29 2022-12-20 Regenxbio Inc Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose tipo ii (mps ii)
IL294638A (en) 2020-01-29 2022-09-01 Regenxbio Inc Treatment of mucopolysaccharidosis iva
IL294924A (en) 2020-02-02 2022-09-01 Univ Pennsylvania Compositions useful for treating gm1 gangliosidosis
JP2023514149A (ja) 2020-02-07 2023-04-05 ユニバーシティ オブ ロチェスター リボザイムにより媒介されるrnaアセンブリ及び発現
WO2021158982A2 (en) 2020-02-07 2021-08-12 University Of Rochester Targeted translation of rna with crispr-cas13 to enhance protein synthesis
KR20220140537A (ko) 2020-02-14 2022-10-18 울트라제닉스 파마수티컬 인코포레이티드 Cdkl5 결핍 장애를 치료하기 위한 유전자 요법
JP2023515820A (ja) 2020-02-28 2023-04-14 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル メープルシロップ尿症の遺伝子治療
JP2023515672A (ja) 2020-03-02 2023-04-13 テナヤ セラピューティクス, インコーポレイテッド 心筋細胞発現マイクロrnaによる遺伝子ベクター制御
CA3170369A1 (en) 2020-03-05 2022-04-14 Michal Shahar Methods and compositions for treating cancer with immune cells
JP2023517222A (ja) 2020-03-11 2023-04-24 ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア 操作されたウイルス粒子を使用した遺伝子送達のための方法および組成物
WO2021188892A1 (en) 2020-03-19 2021-09-23 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
TW202202520A (zh) 2020-03-27 2022-01-16 比利時商Ucb生物製藥公司 自主旋鈕(autonomous knob)結構域肽
WO2021195519A1 (en) 2020-03-27 2021-09-30 University Of Rochester Targeted destruction of viral rna by crispr-cas13
EP4127170A1 (en) 2020-03-27 2023-02-08 University of Rochester Crispr-cas13 crrna arrays
JP2023520402A (ja) 2020-03-31 2023-05-17 ウルトラジェニックス ファーマシューティカル インコーポレイテッド プロピオン酸血症を処置するための遺伝子治療
US20230131352A1 (en) 2020-04-01 2023-04-27 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
MX2022012525A (es) 2020-04-10 2023-02-22 Sola Biosciences Llc Composiciones y metodos para el tratamiento de trastornos de agregacion de proteinas.
JP7713956B2 (ja) 2020-04-15 2025-07-28 ボイジャー セラピューティクス インコーポレイテッド タウ結合化合物
BR112022021134A2 (pt) * 2020-04-20 2023-02-14 Tenaya Therapeutics Inc Vírus adeno-associado com capsídeo projetado
CN116368221A (zh) 2020-04-22 2023-06-30 因达普塔治疗公司 自然杀伤(nk)细胞组合物及其生成方法
CN115836129A (zh) 2020-04-28 2023-03-21 索拉生物科学有限公司 用于治疗tdp-43蛋白病的组合物和方法
IL297577A (en) * 2020-05-05 2022-12-01 Univ Duke Cross-species compatible adeno-associated virus compositions and methods of use thereof
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
TW202208622A (zh) 2020-05-12 2022-03-01 賓州大學委員會 用於治療克拉培氏病之組成物
TW202208406A (zh) 2020-05-13 2022-03-01 美商阿科奧斯公司 用於治療kcnq4相關性聽力損失之組成物及方法
US20230203102A1 (en) 2020-05-13 2023-06-29 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
MX2022014204A (es) 2020-05-13 2023-04-14 Akouos Inc Composiciones y metodos para tratar la perdida auditiva asociada al gen slc26a4.
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
CA3183251A1 (en) 2020-06-05 2021-12-09 Sola Biosciences Llc Compositions and methods for the treatment of synucleinopathies
IL299167A (en) 2020-06-17 2023-02-01 Univ Pennsylvania Compositions and methods for treating patients with gene therapy
WO2021255245A2 (en) 2020-06-19 2021-12-23 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
CA3187635A1 (en) * 2020-07-03 2022-01-06 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
EP4179083A4 (en) * 2020-07-07 2024-08-28 The Board Of Regents Of The University Of Texas System Adeno-associated virus vector for dwarf open reading frame
WO2022011262A1 (en) 2020-07-10 2022-01-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
JP2023532806A (ja) 2020-07-10 2023-07-31 ジェネトン 新規の筋肉特異的プロモーター
TW202216244A (zh) 2020-07-13 2022-05-01 賓州大學委員會 有用於治療夏馬杜三氏病之組成物
AU2021307447A1 (en) 2020-07-15 2023-03-09 University Of Rochester Targeted RNA cleavage with dCas13-RNase fusion proteins
KR20230093241A (ko) 2020-07-27 2023-06-27 보이저 테라퓨틱스, 인크. 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CN116121274B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
US20230295656A1 (en) 2020-08-06 2023-09-21 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
MX2023001998A (es) 2020-08-17 2023-05-04 Massachusetts Inst Technology Enfoques de terapia con el gen shank3.
BR112023003145A2 (pt) 2020-08-19 2023-05-09 Sarepta Therapeutics Inc Vetores de vírus adenoassociado para tratamento da síndrome de rett
JP2023543125A (ja) 2020-08-24 2023-10-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Glp-1受容体アゴニスト融合物をコードするウイルスベクター及び代謝性疾患の治療におけるその使用
BR112023002904A2 (pt) 2020-08-26 2023-04-25 Univ Pennsylvania Vírus adenoassociado recombinante para tratar neurodegeneração com início na idade adulta associada a grn
WO2022050413A1 (ja) 2020-09-04 2022-03-10 国立大学法人神戸大学 小型化シチジンデアミナーゼを含む二本鎖dnaの改変用複合体
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
WO2022061002A1 (en) 2020-09-18 2022-03-24 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
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
EP4225270A1 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
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
MX2023003988A (es) 2020-10-07 2023-06-22 Regenxbio Inc Formulaciones para administración supracoroidea tales como formulaciones con formación de agregados.
EP4225777A2 (en) 2020-10-07 2023-08-16 RegenxBio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2022076582A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Gene therapy for ocular manifestations of cln2 disease
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
IL302127A (en) 2020-10-18 2023-06-01 Univ Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
EP4236999A1 (en) 2020-10-28 2023-09-06 RegenxBio Inc. Vectorized anti-tnf-alfa antibodies for ocular indications
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2022094255A2 (en) 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
MX2023004843A (es) 2020-10-29 2023-05-10 Regenxbio Inc Antagonistas de tnf-alfa con vectorizacion para indicaciones oculares.
IL302128A (en) 2020-11-02 2023-06-01 Biomarin Pharm Inc Process for enriching adeno-associated virus
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
EP4256065A2 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
EP4255570A1 (en) 2020-12-01 2023-10-11 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
KR20230120128A (ko) 2020-12-16 2023-08-16 리젠엑스바이오 인크. 재조합 바이러스 입자의 생산 방법
AU2021404944A1 (en) 2020-12-23 2023-07-06 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
US20240309076A1 (en) 2020-12-29 2024-09-19 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
JP2024500786A (ja) 2020-12-29 2024-01-10 アコーオス インコーポレイテッド Clrn1関連難聴及び/または視力低下を治療するための組成物及び方法
WO2022147480A1 (en) 2020-12-30 2022-07-07 Ansun Biopharma, Inc. Oncolytic virus encoding sialidase and multispecific immune cell engager
CN116981697A (zh) 2021-01-14 2023-10-31 森迪生物科学公司 可分泌有效载荷调节
CA3205209A1 (en) 2021-01-21 2022-07-28 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
KR20230149299A (ko) * 2021-01-25 2023-10-26 트라메스 바이오 인코포레이티드 초점 간질 및 신경병성 통증 치료를 위한 아데노-연관 바이러스 캡시드 및 조작된 리간드 개폐 이온 채널
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
US11926842B2 (en) * 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
EP4288555A1 (en) 2021-02-05 2023-12-13 Maze Therapeutics, Inc. Vectors comprising stuffer polynucleotide sequences
US20240115734A1 (en) * 2021-02-09 2024-04-11 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
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
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CA3212876A1 (en) 2021-03-19 2022-09-22 Adrenas Therapeutics, Inc. Gene therapies for 21-hydroxylase deficiency
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
EP4320139A1 (en) 2021-04-05 2024-02-14 Solid Biosciences Inc. Recombinant herpesvirales vector
MX2023012052A (es) 2021-04-12 2024-03-15 Univ Pennsylvania Composiciones útiles para tratar la atrofia muscular espinal y bulbar (sbma).
US20240181084A1 (en) 2021-04-23 2024-06-06 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas Fusion Protein and Methods of Treatment
US20240207452A1 (en) 2021-04-23 2024-06-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022229807A1 (en) 2021-04-26 2022-11-03 Pfizer Inc. Adeno-associated viral vector capsids with improved tissue tropism
TW202309293A (zh) 2021-04-26 2023-03-01 美商銳進科斯生物股份有限公司 用於治療肌縮蛋白症(dystrophinopathy)之微小肌縮蛋白基因療法給藥
WO2022235614A2 (en) 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
EP4337267A1 (en) 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
WO2022261209A1 (en) * 2021-06-08 2022-12-15 Nf2 Therapeutics, Inc. Compositions and methods for treating neurofibromatic disorders
CA3222911A1 (en) * 2021-06-18 2022-12-22 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
JP2023002483A (ja) 2021-06-22 2023-01-10 ファイザー・インク 昆虫細胞におけるアデノ随伴ウイルスベクターの産生
US20240287493A1 (en) * 2021-06-24 2024-08-29 University Of Utah Research Foundation Compositions and methods for treating pgm1 deficiency
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
KR20240032971A (ko) * 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 유전자 요법을 위한 최적화된 발현 카세트
WO2023004331A1 (en) 2021-07-19 2023-01-26 New York University Auf1 combination therapies for treatment of muscle degenerative disease
WO2023004407A2 (en) * 2021-07-23 2023-01-26 Duke University Adeno-associated virus compositions and methods of use thereof
US20250108090A1 (en) 2021-07-30 2025-04-03 Toru Miyazaki Therapeutic agent for ischemic disease
AU2022326565A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
US20250127820A1 (en) 2021-08-11 2025-04-24 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
EP4384598A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
AU2022325232A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified primary cells for allogeneic cell therapy
WO2023028566A2 (en) * 2021-08-25 2023-03-02 Canbridge Pharmaceuticals, Inc. Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease
EP4392570A4 (en) * 2021-08-25 2025-07-16 Canbridge Pharmaceuticals Inc AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND AN ACID ALPHA-GLUCOSIDASE (GAA) AND THEIR USE FOR TREATING POMPE DISEASE
KR20240107201A (ko) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 항원-결합 분자 및 이의 용도
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
KR20240067112A (ko) 2021-09-30 2024-05-16 아카우오스, 인크. Kcnq4-연관 청력손실을 치료하기 위한 조성물 및 방법
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
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
JP2024537178A (ja) 2021-10-08 2024-10-10 ソラ・バイオサイエンシズ・エルエルシー p53媒介性癌の処置のための組成物および方法
EP4413024A2 (en) 2021-10-08 2024-08-14 SOLA Biosciences LLC Compositions and methods for the treatment of proteopathies
CA3234664A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
EP4415746A1 (en) 2021-10-12 2024-08-21 Bridgebio Gene Therapy LLC Methods and compositions for treating leukodystrophies
WO2023069987A1 (en) 2021-10-20 2023-04-27 University Of Rochester Rejuvenation treatment of age-related white matter loss cross reference to related application
US20250025574A1 (en) 2021-10-28 2025-01-23 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
US20230270818A1 (en) 2021-11-02 2023-08-31 University Of Rochester Tcf7l2 mediated remyelination in the brain
IL311861A (en) 2021-11-02 2024-06-01 Voyager Therapeutics Inc Aav capsid variants and uses thereof
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
EP4433490A2 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
AU2022397707A1 (en) 2021-11-29 2024-06-13 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
WO2023114897A2 (en) * 2021-12-15 2023-06-22 Homology Medicines, Inc. Methods and compositions for the production of adeno-associated virus
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
US20250177495A1 (en) 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
EP4466028A1 (en) 2022-01-17 2024-11-27 Dinaqor AG Gene therapy composition and treatment for dystrophin-related cardiomyopathy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
AU2023211652A1 (en) 2022-01-25 2024-08-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2023150142A1 (en) 2022-02-02 2023-08-10 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Aav衣殼變異體及其用途
WO2023158836A1 (en) 2022-02-17 2023-08-24 Sana Biotechnology, Inc. Engineered cd47 proteins and uses thereof
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
WO2023164545A1 (en) 2022-02-23 2023-08-31 Massachusetts Institute Of Technology Methods for upregulating shank3 expression
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
WO2023178053A1 (en) 2022-03-13 2023-09-21 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
US20250213730A1 (en) 2022-04-01 2025-07-03 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
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
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
KR20250007064A (ko) 2022-04-06 2025-01-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Her2 양성 전이성 유방암 및 기타 암을 치료하기 위한 조성물 및 방법
JP2025512962A (ja) 2022-04-06 2025-04-22 リジェネックスバイオ インコーポレイテッド 上脈絡膜投与のための導入遺伝子をコードする発現カセットを有する組換えアデノ随伴ウイルスベクターを含む医薬組成物
WO2023198652A1 (en) 2022-04-11 2023-10-19 Centre National De La Recherche Scientifique Chemically-modified adeno-associated viruses
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
US20250277004A1 (en) 2022-04-18 2025-09-04 Regenxbio Inc. Hybrid aav capsids
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
EP4518973A1 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
EP4518972A2 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
EP4519463A1 (en) 2022-05-06 2025-03-12 Novartis AG Novel recombinant aav vp2 fusion polypeptides
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023230409A1 (en) * 2022-05-24 2023-11-30 Kate Therapeutics, Inc. Compositions for treating xlmtm
IL316719A (en) 2022-06-02 2024-12-01 Voyager Therapeutics Inc Adeno-associated virus capsid variants and their uses
WO2023240158A2 (en) * 2022-06-07 2023-12-14 Oregon Health & Science University Mutant adeno-associated virus (aav) capsids
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024006741A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4547269A1 (en) 2022-06-30 2025-05-07 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
AR129843A1 (es) 2022-07-06 2024-10-02 Voyager Therapeutics Inc Variantes de la cápside de aav y usos de estas
EP4558149A1 (en) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
AU2023320453A1 (en) 2022-08-03 2025-02-06 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
AU2023331251A1 (en) 2022-08-25 2025-02-27 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024064856A1 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of cardiomyopathy with aav gene therapy vectors
TW202421788A (zh) 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用aav基因療法載體治療致心律不整性心肌病
IL319873A (en) 2022-09-30 2025-05-01 Regenxbio Inc Treatment of eye diseases with recombinant viral vectors containing anti-VEGF FAB
CN120225686A (zh) * 2022-10-04 2025-06-27 赛仁生物技术公司 用于重组病毒载体生产的修饰的CpG二核苷酸
EP4601710A2 (en) 2022-10-11 2025-08-20 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
TW202440939A (zh) 2022-12-17 2024-10-16 賓州大學委員會 具心臟及骨骼肌特異性靶向模體的重組aav突變載體及含有其之組成物
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
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
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
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
WO2024149844A1 (en) 2023-01-12 2024-07-18 Nantes Université Chemically-modified adeno-associated virus
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024163747A2 (en) 2023-02-02 2024-08-08 University Of Rochester Competitive replacement of glial cells
WO2024163979A2 (en) * 2023-02-02 2024-08-08 Codexis, Inc. Engineered aav polypeptides
TW202449165A (zh) 2023-02-17 2024-12-16 美商渤健麻塞諸塞州股份有限公司 用於遞送有效載荷之含rgd之肽
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024196855A2 (en) 2023-03-17 2024-09-26 University Of Rochester Ribozyme-mediated rna assembly and expression
WO2024206226A1 (en) * 2023-03-25 2024-10-03 Duke University Compositions comprising kidney-tropic aavs and methods of use thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024220389A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav2 variants and uses thereof
WO2024220463A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav5 capsid with non-canonical amino acid incorporation and uses thereof
WO2024218192A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
WO2024218190A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Generation of adeno-associated virus capsid libraries for insect cells
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229167A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2024229302A1 (en) 2023-05-03 2024-11-07 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
WO2024229125A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2024229163A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
TW202444917A (zh) 2023-05-04 2024-11-16 美商航海家醫療公司 Aav衣殼變異體及其用途
WO2024233422A1 (en) 2023-05-05 2024-11-14 Massachusetts Institute Of Technology Shank3 gene therapy approaches
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
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
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
WO2024241176A1 (en) 2023-05-24 2024-11-28 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
WO2025017168A1 (en) 2023-07-19 2025-01-23 Genethon Novel optimized utrophin micro-genes
WO2025017169A1 (en) 2023-07-20 2025-01-23 Genethon Novel mididystrophins
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025042711A1 (en) 2023-08-18 2025-02-27 Eli Lilly And Company Engineered transferrin receptor binding peptides as well as methods of making and using the same
WO2025072604A1 (en) 2023-09-28 2025-04-03 University Of Rochester Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1)
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025080780A1 (en) 2023-10-10 2025-04-17 University Of Rochester Delivery and expression of prime editing crispr systems
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025096967A1 (en) 2023-11-02 2025-05-08 Biogen Ma Inc. Redirection of aav capsids for central nervous system targeting
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025122543A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025122536A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025171001A1 (en) 2024-02-06 2025-08-14 Regeneron Pharmaceuticals, Inc. Compositions and methods for improved production of aav viral vectors

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
EP0162067B1 (en) 1983-10-28 1992-07-15 Genetics Institute, Inc. Production of factor viii and related products
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
IL74909A (en) 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
US5171844A (en) 1987-06-12 1992-12-15 Gist-Brocades N.W. Proteins with factor viii activity: process for their preparation using genetically-engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
HUT64591A (en) 1989-11-17 1994-01-28 Novo Nordisk As Protein complexes with viii/c factor activity and process for their production
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
ES2226029T3 (es) 1993-06-10 2005-03-16 Bayer Corporation Vector y linea celular de mamifero con productividad mejorada.
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
WO1997003195A1 (en) 1995-07-11 1997-01-30 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
US20020088014A1 (en) * 1996-05-31 2002-07-04 Xiangming Fang Minimal adenovirus mediated recombinant vaccine
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
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
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
EP0932694A2 (en) 1996-09-11 1999-08-04 THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
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
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
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
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2002248297A1 (en) * 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
CN1236057C (zh) * 2001-10-18 2006-01-11 华中科技大学同济医学院附属同济医院 表达人类激肽释放酶的重组腺相关病毒及其制法和用途
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
US7198951B2 (en) 2001-12-17 2007-04-03 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
WO2003052051A2 (en) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
WO2003089612A2 (en) * 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. IMPROVED rAAV VECTORS
CA2526120A1 (en) * 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
HUE035567T2 (en) * 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
CN102994549B (zh) * 2005-04-07 2015-02-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
WO2012112832A1 (en) * 2011-02-17 2012-08-23 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
SG10201913259QA (en) * 2014-09-24 2020-02-27 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
SG11201907714UA (en) 2017-02-28 2019-09-27 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
US10786568B2 (en) * 2017-02-28 2020-09-29 The Trustees Of The University Of Pennsylvania AAV mediated influenza vaccines

Also Published As

Publication number Publication date
US20200155704A1 (en) 2020-05-21
US20190015527A1 (en) 2019-01-17
SI3211085T1 (sl) 2021-12-31
US10973928B2 (en) 2021-04-13
EP3211085A1 (en) 2017-08-30
US20070036760A1 (en) 2007-02-15
ES2616016T3 (es) 2017-06-09
JP2012175974A (ja) 2012-09-13
WO2005033321A2 (en) 2005-04-14
US7906111B2 (en) 2011-03-15
JP2017060483A (ja) 2017-03-30
CN102199626A (zh) 2011-09-28
CA2939103A1 (en) 2005-04-14
PT2292780T (pt) 2017-11-28
JP2014012022A (ja) 2014-01-23
EP2292780B1 (en) 2017-08-23
HUS2100033I1 (hu) 2021-09-28
EP2292780A3 (en) 2011-03-30
US9737618B2 (en) 2017-08-22
HUE035567T2 (en) 2018-05-28
DK2292779T3 (en) 2017-02-27
US20210299274A1 (en) 2021-09-30
DK2345731T3 (en) 2016-01-25
CY2021024I2 (el) 2021-12-31
HUE054805T2 (hu) 2021-09-28
CY2021024I1 (el) 2021-12-31
CA3072423A1 (en) 2005-04-14
US11357867B2 (en) 2022-06-14
ES2648241T3 (es) 2017-12-29
US11357868B2 (en) 2022-06-14
CN102220376A (zh) 2011-10-19
EP2345731B1 (en) 2015-10-21
JP5911069B2 (ja) 2016-04-27
PL2292780T3 (pl) 2018-05-30
DK2292780T3 (en) 2017-12-04
US20210170050A1 (en) 2021-06-10
ES2555612T3 (es) 2016-01-05
FR21C1037I1 (fr) 2021-10-15
US10265417B2 (en) 2019-04-23
EP2292779A2 (en) 2011-03-09
ES2411479T3 (es) 2013-07-05
ES2874298T3 (es) 2021-11-04
CA2537793C (en) 2013-07-23
CN1856576B (zh) 2011-05-04
CN102174574B (zh) 2016-08-03
JP2007507223A (ja) 2007-03-29
US20190054188A1 (en) 2019-02-21
EP2298926A1 (en) 2011-03-23
JP2015163083A (ja) 2015-09-10
JP6673963B2 (ja) 2020-04-01
US20170028082A1 (en) 2017-02-02
CN102212558A (zh) 2011-10-12
EP2345731A1 (en) 2011-07-20
CY1124586T1 (el) 2021-12-31
JP2020054373A (ja) 2020-04-09
JP6335141B2 (ja) 2018-05-30
US20220025400A1 (en) 2022-01-27
US10485883B2 (en) 2019-11-26
AU2004278684A1 (en) 2005-04-14
AU2008203421B2 (en) 2011-08-25
JP6516713B2 (ja) 2019-05-22
EP1668143A2 (en) 2006-06-14
US10695441B2 (en) 2020-06-30
JP5431516B2 (ja) 2014-03-05
CN1856576A (zh) 2006-11-01
AU2008203421A1 (en) 2008-08-21
AU2004278684B2 (en) 2011-05-12
JP2011101646A (ja) 2011-05-26
EP2292779A3 (en) 2011-03-30
PT3211085T (pt) 2021-06-17
JP2018108113A (ja) 2018-07-12
SI2292780T1 (sl) 2017-12-29
JP5054975B2 (ja) 2012-10-24
CN102199626B (zh) 2015-06-24
EP1668143B1 (en) 2013-03-20
EP3211085B1 (en) 2021-03-31
US20110236353A1 (en) 2011-09-29
EP2292780A2 (en) 2011-03-09
EP2292779B1 (en) 2016-11-16
US20220023441A1 (en) 2022-01-27
PL3211085T3 (pl) 2021-11-15
JP5797397B2 (ja) 2015-10-21
JP2022173573A (ja) 2022-11-18
CA2939103C (en) 2020-03-24
WO2005033321A3 (en) 2005-06-30
US20190022249A1 (en) 2019-01-24
US10722598B2 (en) 2020-07-28
CN102174574A (zh) 2011-09-07
US20150315612A1 (en) 2015-11-05
EP3910063A1 (en) 2021-11-17
US20170159027A1 (en) 2017-06-08
NZ545628A (en) 2009-04-30
DK3211085T3 (da) 2021-06-21
HUE033158T2 (en) 2017-11-28
CA2537793A1 (en) 2005-04-14
CN102212558B (zh) 2016-08-03
CY1119833T1 (el) 2018-06-27

Similar Documents

Publication Publication Date Title
BE2021C533I2 (enrdf_load_html_response)
BE2013C075I2 (enrdf_load_html_response)
BE2013C070I2 (enrdf_load_html_response)
BE2013C067I2 (enrdf_load_html_response)
BE2013C038I2 (enrdf_load_html_response)
BE2013C036I2 (enrdf_load_html_response)
BE2011C030I2 (enrdf_load_html_response)
BE2013C034I2 (enrdf_load_html_response)
BE2012C053I2 (enrdf_load_html_response)
AU2002331433A1 (enrdf_load_html_response)
AU2002341898A1 (enrdf_load_html_response)
AU2002256398A1 (enrdf_load_html_response)
AU2002310561A1 (enrdf_load_html_response)
AU2002311957A1 (enrdf_load_html_response)
AU2002316235A1 (enrdf_load_html_response)
AU2002316511A1 (enrdf_load_html_response)
AU2002318342A1 (enrdf_load_html_response)
AU2002321535A1 (enrdf_load_html_response)
AU2002322913A1 (enrdf_load_html_response)
AU2002324323A1 (enrdf_load_html_response)
AU2002327042A1 (enrdf_load_html_response)
AU2002327736A1 (enrdf_load_html_response)
AU2002329412A1 (enrdf_load_html_response)
AU2002254171A1 (enrdf_load_html_response)
AU2002333044A1 (enrdf_load_html_response)