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 PDFInfo
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- 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
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- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- 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
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- 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
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- 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
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- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Ophthalmology & Optometry (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (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)
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 (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| JP6985795B2 (ja) | 2013-09-26 | 2021-12-22 | 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 | 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 |
| 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 |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| WO2019077159A1 (en) * | 2017-10-20 | 2019-04-25 | INSERM (Institut National de la Santé et de la Recherche Médicale) | METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR |
| CA3095660A1 (en) * | 2018-02-15 | 2019-08-22 | Tomas Bjorklund | Modified viral capsids |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN112955557A (zh) | 2018-10-01 | 2021-06-11 | 奥特吉尼克斯制药公司 | 用于治疗丙酸血症的基因疗法 |
| US20210388343A1 (en) * | 2018-10-17 | 2021-12-16 | Children's Medical Research Institute | Nucleic acid molecules and methods for aav vector selection |
| CA3125294A1 (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 |
| WO2020264438A2 (en) | 2019-06-27 | 2020-12-30 | University Of Florida Research Foundation, Incorporated | Enhancing aav-mediated transduction of ocular tissues with hyaluronic acid |
| WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
| IL294781A (en) | 2020-02-14 | 2022-09-01 | Ultragenyx Pharmaceutical Inc | Gene therapy for treating cdkl5 deficiency disorder |
| EP4121544A1 (en) | 2020-03-19 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| EP4127189A1 (en) | 2020-03-31 | 2023-02-08 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| US20230233708A1 (en) * | 2020-04-14 | 2023-07-27 | University Of Florida Research Foundation, 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 |
| CN116568338A (zh) | 2020-07-29 | 2023-08-08 | 佛罗里达州大学研究基金会 | 改进的aav介导的x连锁视网膜劈裂症治疗 |
| EP4243848A4 (en) * | 2020-11-10 | 2025-07-23 | Avirmax Biopharma Inc | MODIFIED VIRAL CAPSIDS AND METHODS OF USE |
| TW202235618A (zh) * | 2021-01-05 | 2022-09-16 | 美國馬友醫藥教育研究基金會 | 治療眼內壓相關疾患 |
| US20250019721A1 (en) | 2021-08-11 | 2025-01-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| 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 |
| WO2024229049A1 (en) | 2023-05-01 | 2024-11-07 | University Of Florida Research Foundation, Incorporated | Dual aav vectors for treating stargardt disease |
| TW202525832A (zh) | 2023-08-31 | 2025-07-01 | 美商戴諾治療公司 | 衣殼多肽及其使用方法(二) |
| WO2025171227A1 (en) | 2024-02-08 | 2025-08-14 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| 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)
| 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)
| 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 |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| EP1610751A4 (en) | 2001-04-26 | 2006-05-24 | Univ Texas | THERAPEUTIC AGENT / LIGAND CONJUGATE COMPOSITIONS, ITS SYNTHESIS PROCESSES AND ITS APPLICATION |
| EP1487501B1 (en) | 2001-10-09 | 2012-12-05 | The Johns Hopkins University | A phosphatase associated with metastasis |
| 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 |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US7220577B2 (en) | 2002-08-28 | 2007-05-22 | University Of Florida Research Foundation, Inc. | Modified AAV |
| AU2003300368A1 (en) | 2002-12-26 | 2004-07-29 | Cemines, Llc. | Methods and compositions for the diagnosis, prognosis, and treatment of cancer |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| PL3235827T3 (pl) | 2003-06-19 | 2021-07-05 | Genzyme Corporation | Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| CA2591544A1 (en) | 2004-12-15 | 2006-06-22 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | 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 |
| BRPI0807160A2 (pt) | 2007-02-02 | 2018-09-25 | Baylor Res Institute | vacinas baseadas no direcionamento de antígeno ao dcir expresso em células apresentadoras de antígeno |
| 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 |
| HUE030719T2 (en) | 2007-04-09 | 2017-05-29 | Univ Florida | RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use |
| ES2634118T3 (es) | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
| ES2683695T3 (es) | 2010-01-12 | 2018-09-27 | The University Of North Carolina At Chapel Hill | Repeticiones terminales invertidas restrictivas para vectores virales |
| 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 |
| 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 |
| EP4234571A3 (en) | 2011-02-10 | 2023-09-27 | The University of North Carolina at Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| US9658216B2 (en) | 2012-01-31 | 2017-05-23 | Cell Cure Neurosciences Ltd. | Methods of selecting retinal pigmented epithelial cells |
| SI2839014T1 (sl) * | 2012-04-18 | 2021-05-31 | The Children's Hospital Of Philadelphia | Sestavek in postopki za zelo učinkovit prenos genov z uporabo variant kapside AAV-JA |
| EP2847337A4 (en) | 2012-05-09 | 2016-04-27 | Univ Oregon Health & Science | ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS |
| CA2904396A1 (en) | 2013-03-15 | 2014-09-18 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav vectors |
| WO2015108610A1 (en) * | 2013-11-08 | 2015-07-23 | 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 |
| US11000597B2 (en) | 2015-01-21 | 2021-05-11 | University Of Florida Research Foundation, Incorporated | 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 |
| EP3277819B1 (en) | 2015-03-24 | 2021-03-03 | 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 |
| US11266748B2 (en) | 2015-07-02 | 2022-03-08 | University Of Florida Research Foundation, Incorporated | Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| JP7406783B2 (ja) * | 2015-12-14 | 2023-12-28 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| AU2020329751A1 (en) | 2019-08-09 | 2022-02-24 | University Of Florida Research Foundation, Incorporated | AAV capsid variants for targeting human glioblastoma cells |
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2018
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- 2018-02-21 WO PCT/US2018/019050 patent/WO2018156654A1/en not_active Ceased
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Patent Citations (5)
| 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
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| WO2018156654A1 (en) | 2018-08-30 |
| US20220267383A1 (en) | 2022-08-25 |
| US11332502B2 (en) | 2022-05-17 |
| US20200002386A1 (en) | 2020-01-02 |
| US10793606B2 (en) | 2020-10-06 |
| AU2018224044A1 (en) | 2019-09-19 |
| US11767346B2 (en) | 2023-09-26 |
| JP2020508667A (ja) | 2020-03-26 |
| AU2018224044B2 (en) | 2024-01-25 |
| EP3585883A4 (en) | 2021-04-14 |
| CA3054131C (en) | 2021-05-25 |
| EP3585883A1 (en) | 2020-01-01 |
| CA3054131A1 (en) | 2018-08-30 |
| CA3114549A1 (en) | 2018-08-30 |
| US20210061863A1 (en) | 2021-03-04 |
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