JP7237843B2 - 修飾されたａａｖキャプシドタンパク質およびその使用 - Google Patents

修飾されたａａｖキャプシドタンパク質およびその使用 Download PDF

Info

Publication number
JP7237843B2
JP7237843B2 JP2019546041A JP2019546041A JP7237843B2 JP 7237843 B2 JP7237843 B2 JP 7237843B2 JP 2019546041 A JP2019546041 A JP 2019546041A JP 2019546041 A JP2019546041 A JP 2019546041A JP 7237843 B2 JP7237843 B2 JP 7237843B2
Authority
JP
Japan
Prior art keywords
raav
variant
cells
aav2
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2019546041A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020508667A (ja
JP2020508667A5 (enExample
Inventor
ゾロツキン，セルゲイ
ボエ，サンフォード，エル．
ボエ，シャノン，イー．
マーシク、ダミアン
ムジクツカ，ニコラス
メンデス－ゴメス，ヘクター，ルーベン
ガムリン，ポール，デー．
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Florida Research Foundation Inc
Original Assignee
University of Florida Research Foundation Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of Florida Research Foundation Inc filed Critical University of Florida Research Foundation Inc
Publication of JP2020508667A publication Critical patent/JP2020508667A/ja
Publication of JP2020508667A5 publication Critical patent/JP2020508667A5/ja
Application granted granted Critical
Publication of JP7237843B2 publication Critical patent/JP7237843B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1037—Screening libraries presented on the surface of microorganisms, e.g. phage display, E. coli display
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Peptides Or Proteins (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2019546041A 2017-02-21 2018-02-21 修飾されたａａｖキャプシドタンパク質およびその使用 Active JP7237843B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762461770P 2017-02-21 2017-02-21
US62/461,770 2017-02-21
US201862625486P 2018-02-02 2018-02-02
US62/625,486 2018-02-02
PCT/US2018/019050 WO2018156654A1 (en) 2017-02-21 2018-02-21 Modified aav capsid proteins and uses thereof

Publications (3)

Publication Number Publication Date
JP2020508667A JP2020508667A (ja) 2020-03-26
JP2020508667A5 JP2020508667A5 (enExample) 2021-04-08
JP7237843B2 true JP7237843B2 (ja) 2023-03-13

Family

ID=63253351

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019546041A Active JP7237843B2 (ja) 2017-02-21 2018-02-21 修飾されたａａｖキャプシドタンパク質およびその使用

Country Status (6)

Country Link
US (3) US10793606B2 (enExample)
EP (1) EP3585883A4 (enExample)
JP (1) JP7237843B2 (enExample)
AU (1) AU2018224044B2 (enExample)
CA (2) CA3114549A1 (enExample)
WO (1) WO2018156654A1 (enExample)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2623605B1 (en) 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. RAAV vector compositions having tyrosine-modified capsid proteins and methods for use
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
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
JP7237843B2 (ja) 2017-02-21 2023-03-13 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 修飾されたａａｖキャプシドタンパク質およびその使用
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US11723988B2 (en) 2017-10-20 2023-08-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
CN112105630A (zh) * 2018-02-15 2020-12-18 T·比约尔克隆德 经修饰的病毒衣壳
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP2022513318A (ja) 2018-10-01 2022-02-07 ウルトラジェニックス ファーマシューティカル インコーポレイテッド プロピオン酸血症を処置するための遺伝子治療
WO2020077411A1 (en) * 2018-10-17 2020-04-23 Children's Medical Research Institute Nucleic acid molecules and methods for aav vector selection
WO2020154535A1 (en) 2019-01-23 2020-07-30 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
MX2021016014A (es) 2019-06-27 2022-04-06 Univ Florida Mejora de la transducción mediada por aav en los tejidos oculares con ácido hialurónico.
CA3153133A1 (en) * 2019-09-30 2021-04-08 Chris Bartolome Adeno-associated virus (aav) systems for treatment of genetic hearing loss
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
EP4103724A1 (en) 2020-02-14 2022-12-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
US20230235353A1 (en) 2020-03-19 2023-07-27 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
WO2021211641A2 (en) * 2020-04-14 2021-10-21 University Of Florida Researchfoundation, Incorporated Enhancing aav-mediated delivery and transduction with polyvinyl alcohol
CN111729078B (zh) * 2020-07-24 2020-11-20 苏州世诺生物技术有限公司 鸡传染性贫血病毒基因工程疫苗
JP2023535956A (ja) 2020-07-29 2023-08-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 改善されたｘ連鎖性網膜分離症のａａｖ媒介療法
CA3196439A1 (en) * 2020-11-10 2022-05-19 Avirmax, Inc. Engineered viral capsids and methods of use
WO2022150409A1 (en) * 2021-01-05 2022-07-14 Mayo Foundation For Medical Education And Research Treatments for intraocular pressure related disorders
JP2024531138A (ja) 2021-08-11 2024-08-29 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 筋ジストロフィーを処置するための組成物および方法
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
EP4490278A1 (en) 2022-03-07 2025-01-15 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
EP4704917A1 (en) 2023-05-01 2026-03-11 University of Florida Research Foundation, Incorporated Dual aav vectors for treating stargardt disease
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025235555A1 (en) * 2024-05-06 2025-11-13 University Of Florida Research Foundation, Incorporated Rational design of aav vectors with limited liver targeting

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
WO2015121501A1 (en) 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
WO2015134643A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
JP2016520311A (ja) 2013-05-21 2016-07-14 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
JP2016533709A (ja) 2013-09-26 2016-11-04 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
EP1389090A2 (en) 2001-04-26 2004-02-18 Board of Regents, The University of Texas System Diagnostic imaging compositions, their methods of synthesis and use
JP4353799B2 (ja) 2001-10-09 2009-10-28 ザ・ジョーンズ・ホプキンス・ユニバーシティ 転移に関連するホスファターゼ
EP1441030A4 (en) 2001-10-11 2007-08-15 Katakura Ind Co Ltd PROCESS FOR CLEANING A RECOMBINANT FUSION PROTEIN AND METHOD FOR PRODUCING PROTEIN THEREWITH
US20030225260A1 (en) 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
US20060127358A1 (en) 2002-05-01 2006-06-15 Nicholas Muzyczka Raav expression systems and methods for enhancing transduction of mammalian neural cells
US20060093589A1 (en) 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
JP2006524035A (ja) 2002-12-26 2006-10-26 シーマインズ リミテッド ライアビリティ カンパニー 癌の診断、予後診断および治療のための方法および組成物
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP2007524386A (ja) 2003-06-19 2007-08-30 アビジェン， インコーポレイテッド 減少した免疫反応性を有するａａｖビリオン、およびその使用
DK3211085T3 (da) 2003-09-30 2021-06-21 Univ Pennsylvania Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf
US7892809B2 (en) 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US8999678B2 (en) 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
TWI422594B (zh) 2007-02-02 2014-01-11 Baylor Res Inst 經由樹狀細胞去唾液酸糖蛋白受體（ｄｃ-ａｓｇｐｒ）接合抗原呈現細胞之藥劑
CN101679508B (zh) 2007-02-02 2014-11-05 贝勒研究院 基于靶向抗原至抗原呈递细胞上表达的dcir的疫苗
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
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
EP2623605B1 (en) 2007-04-09 2018-11-28 University of Florida Research Foundation, Inc. RAAV vector compositions having tyrosine-modified capsid proteins and methods for use
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
US10415056B2 (en) 2010-11-10 2019-09-17 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
JP6042825B2 (ja) * 2011-02-10 2016-12-14 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 改変された形質導入プロファイルを有するウイルスベクターならびにその製造および使用の方法
WO2013114360A1 (en) 2012-01-31 2013-08-08 Cell Cure Neurosciences Ltd. Methods of selecting retinal pigmented epithelial cells
RU2683497C2 (ru) * 2012-04-18 2019-03-28 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
CN105163764B (zh) 2013-03-15 2019-11-12 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
US20160272687A1 (en) * 2013-11-08 2016-09-22 The Board Of Trustees Of The University Of Arkansas Adeno-associated virus "x" oncogene
WO2015126972A1 (en) 2014-02-21 2015-08-27 University Of Florida Research Foundation, Inc. Methods and compositions for gene delivery to on bipolar cells
WO2016118787A1 (en) 2015-01-21 2016-07-28 University Of Florida Research Foundation, Inc. Engineered receptor/ligand system for delivery of therapeutic agents
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
WO2016154344A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
WO2017004514A1 (en) 2015-07-02 2017-01-05 University Of Florida Research Foundation, Inc. Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
JP7064214B2 (ja) 2015-09-28 2022-05-10 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
CN108602859B (zh) * 2015-12-14 2023-05-26 北卡罗来纳大学教堂山分校 增强细小病毒载体递送的修饰衣壳蛋白
JP7237843B2 (ja) 2017-02-21 2023-03-13 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 修飾されたａａｖキャプシドタンパク質およびその使用
AU2020329751A1 (en) 2019-08-09 2022-02-24 University Of Florida Research Foundation, Incorporated AAV capsid variants for targeting human glioblastoma cells

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. Vectors for delivery of light-sensitive proteins and methods of use
JP2016520311A (ja) 2013-05-21 2016-07-14 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド キャプシド修飾ｒＡＡＶ３ベクター組成物およびヒト肝がんの遺伝子治療における使用方法
JP2016533709A (ja) 2013-09-26 2016-11-04 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 標的遺伝子治療のための合成コンビナトリアルａａｖカプシドライブラリー
WO2015121501A1 (en) 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
WO2015134643A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells

Also Published As

Publication number Publication date
EP3585883A4 (en) 2021-04-14
CA3054131C (en) 2021-05-25
US20210061863A1 (en) 2021-03-04
US10793606B2 (en) 2020-10-06
AU2018224044A1 (en) 2019-09-19
WO2018156654A1 (en) 2018-08-30
JP2020508667A (ja) 2020-03-26
US20220267383A1 (en) 2022-08-25
EP3585883A1 (en) 2020-01-01
US11767346B2 (en) 2023-09-26
AU2018224044B2 (en) 2024-01-25
CA3114549A1 (en) 2018-08-30
US20200002386A1 (en) 2020-01-02
CA3054131A1 (en) 2018-08-30
US11332502B2 (en) 2022-05-17

Similar Documents

Publication Publication Date Title
JP7237843B2 (ja) 修飾されたａａｖキャプシドタンパク質およびその使用
Castle et al. Controlling AAV tropism in the nervous system with natural and engineered capsids
EP3393522B1 (en) Improved hybrid dual recombinant aav vector systems for gene therapy
WO2023284879A1 (en) Modified aav capsid for gene therapy and methods thereof
JP2018121653A (ja) ウイルスベクター精製における拡張可能な製造プラットフォームおよび遺伝子治療における使用のための高純度ウイルスベクター
CN111876432B (zh) 一组肝靶向新型腺相关病毒的获得及其应用
US20260001917A1 (en) Modified adeno-associated virus capsid proteins and methods thereof
TW202246516A (zh) 病毒蛋白之控制表現
CA3088079A1 (en) Compositions and methods for treating retinal disorders
JP2026500351A (ja) 心筋および骨格筋特異的標的化モチーフを有する組換えａａｖ変異体ベクターおよびそれを含む組成物
CN121358754A (zh) 具有外周器官去靶向性的新型嗜神经性腺相关病毒衣壳
RU2820088C1 (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
US20260042803A1 (en) Variant aav capsid polypeptides targeting the eye
WO2025212745A1 (en) Engineered adeno-associated virus (aav) capsid protein and uses thereof
WO2023022633A1 (ru) Выделенный модифицированный белок vp1 капсида aav5
HK40030486A (en) Compositions and methods for treating retinal disorders
HK40030486B (en) Compositions and methods for treating retinal disorders

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210222

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210222

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220218

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220518

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220715

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20221003

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221223

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230118

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230202

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230301

R150 Certificate of patent or registration of utility model

Ref document number: 7237843

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250