AU2014274457B2 - Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer - Google Patents
Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer Download PDFInfo
- Publication number
- AU2014274457B2 AU2014274457B2 AU2014274457A AU2014274457A AU2014274457B2 AU 2014274457 B2 AU2014274457 B2 AU 2014274457B2 AU 2014274457 A AU2014274457 A AU 2014274457A AU 2014274457 A AU2014274457 A AU 2014274457A AU 2014274457 B2 AU2014274457 B2 AU 2014274457B2
- Authority
- AU
- Australia
- Prior art keywords
- vectors
- cell
- cells
- accordance
- vector
- 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
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/19—Dendritic cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
- A61K40/24—Antigen-presenting cells [APC]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
- A61K40/34—Antigenic peptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
- A61K40/41—Vertebrate antigens
- A61K40/42—Cancer antigens
- A61K40/4244—Enzymes
- A61K40/4246—Telomerase or [telomerase reverse transcriptase [TERT]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
-
- 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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- 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
- 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
- C12N2750/14171—Demonstrated in vivo effect
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Manufacturing & Machinery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2020200041A AU2020200041B2 (en) | 2013-05-21 | 2020-01-03 | Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer |
| AU2022204246A AU2022204246A1 (en) | 2013-05-21 | 2022-06-17 | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13/899,481 | 2013-05-21 | ||
| US13/899,481 US9920097B2 (en) | 2007-04-09 | 2013-05-21 | Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use |
| PCT/US2014/039015 WO2014193716A2 (en) | 2013-05-21 | 2014-05-21 | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2020200041A Division AU2020200041B2 (en) | 2013-05-21 | 2020-01-03 | Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2014274457A1 AU2014274457A1 (en) | 2016-01-21 |
| AU2014274457B2 true AU2014274457B2 (en) | 2019-10-24 |
Family
ID=51023068
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2014274457A Active AU2014274457B2 (en) | 2013-05-21 | 2014-05-21 | Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer |
| AU2020200041A Active AU2020200041B2 (en) | 2013-05-21 | 2020-01-03 | Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer |
| AU2022204246A Abandoned AU2022204246A1 (en) | 2013-05-21 | 2022-06-17 | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2020200041A Active AU2020200041B2 (en) | 2013-05-21 | 2020-01-03 | Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer |
| AU2022204246A Abandoned AU2022204246A1 (en) | 2013-05-21 | 2022-06-17 | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer |
Country Status (9)
| Country | Link |
|---|---|
| EP (2) | EP2999791B1 (enExample) |
| JP (3) | JP6628286B2 (enExample) |
| KR (4) | KR102298579B1 (enExample) |
| CN (3) | CN111763690A (enExample) |
| AU (3) | AU2014274457B2 (enExample) |
| CA (1) | CA2917018A1 (enExample) |
| DK (1) | DK2999791T3 (enExample) |
| SG (2) | SG11201510757PA (enExample) |
| WO (1) | WO2014193716A2 (enExample) |
Families Citing this family (57)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5410017A (en) * | 1993-05-21 | 1995-04-25 | Rohm And Haas Company | Continuous thermal polycondensation process for preparing polypeptide polymers |
| 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 |
| 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 |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| US20180030096A1 (en) | 2015-02-03 | 2018-02-01 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| 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 |
| BR112019002904A2 (pt) * | 2016-08-16 | 2019-06-25 | The University Of North Carolina At Chapel Hill | métodos e composições para transferência gênica direcionada |
| IL264872B2 (en) * | 2016-08-18 | 2025-02-01 | Univ California | CRISPR-CAS genome engineering using a modular AAV delivery system |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| CN110892062A (zh) | 2017-05-05 | 2020-03-17 | 佛罗里达大学研究基金会 | 表达耳畸蛋白的组合物和方法 |
| DK3645551T5 (da) * | 2017-06-27 | 2024-08-26 | Regeneron Pharma | Tropisme-modificerede rekombinante virusvektorer og anvendelser deraf til targetteret indføring af genetisk materiale i menneskeceller |
| EP3679148A4 (en) * | 2017-09-08 | 2021-06-09 | Generation Bio Co. | LIPID NANOPARTICLE FORMULATIONS FROM NONVIRAL, CAPSID-FREE DNA VECTORS |
| JP2021510539A (ja) * | 2018-01-16 | 2021-04-30 | シーエルエス セラピューティクス リミテッドCLS Therapeutics Limited | デオキシリボヌクレアーゼ(DNase)活性を有する酵素の肝臓発現による疾患の処置 |
| US12480138B2 (en) * | 2018-01-17 | 2025-11-25 | MeiraGTx Gene Regulation Limited | Modified rAAV capsid protein for gene therapy |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| CN120665951A (zh) * | 2018-02-07 | 2025-09-19 | 吉尼松公司 | 杂合调控元件 |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | F8遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| CN112218880A (zh) * | 2018-03-29 | 2021-01-12 | 阿斯克肋匹奥生物制药公司 | 逃避中和的肝趋向性重组aav6载体 |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| JP7331011B2 (ja) | 2018-04-27 | 2023-08-22 | デシベル セラピューティクス インコーポレイテッド | ミオシン15プロモーター及びその使用 |
| EP3784697A4 (en) * | 2018-04-27 | 2022-07-06 | Spark Therapeutics, Inc. | Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same |
| JP7142815B2 (ja) * | 2018-06-21 | 2022-09-28 | 株式会社遺伝子治療研究所 | ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| EP3820885A4 (en) | 2018-07-11 | 2022-04-20 | The Brigham & Women's Hospital, Inc. | METHODS AND COMPOSITIONS FOR ADMINISTRATION OF DRUGS ACROSS THE BLOOD-BRAIN BARRIER |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| WO2020079256A1 (en) * | 2018-10-19 | 2020-04-23 | Genethon | Modulation, monitoring and prediction of the immune response directed against aav gene therapy vectors |
| AU2020208933B2 (en) | 2019-01-18 | 2025-08-28 | Centre National De La Recherche Scientifique (Cnrs) | AAV-mediated gene therapy restoring the otoferlin gene |
| CA3129422A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| WO2020163743A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| EP3736330A1 (en) * | 2019-05-08 | 2020-11-11 | European Molecular Biology Laboratory | Modified adeno-associated virus (aav) particles for gene therapy |
| EP3999120A4 (en) * | 2019-07-04 | 2024-02-21 | Children's Medical Research Institute | Methods and aav vectors for in vivo transduction |
| US20220282276A1 (en) * | 2019-07-11 | 2022-09-08 | Centre National De La Recherche Scientifique | Chemically-modified adeno-associated virus |
| AU2020347276A1 (en) * | 2019-09-12 | 2022-03-17 | Massachusetts Institute Of Technology | Engineered adeno-associated virus capsids |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療c型尼曼—匹克病之腺相關病毒載體 |
| US20230340526A1 (en) * | 2019-11-25 | 2023-10-26 | University Of Florida Research Foundation, Incorporated | Novel aav3b variants that target hepatocytes and evade the humoral immune response |
| US20230049066A1 (en) * | 2019-11-25 | 2023-02-16 | University Of Florida Research Foundation, Incorporated | Novel aav3b variants that target human hepatocytes in the liver of humanized mice |
| AU2021206256A1 (en) * | 2020-01-10 | 2022-07-28 | The Brigham And Women's Hospital, Inc. | Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer |
| 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 |
| CN115666658A (zh) | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | 具有提高的治疗ush1b的安全性的双aav-myo7a载体 |
| JP2023524577A (ja) * | 2020-05-05 | 2023-06-12 | 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 | 改変アデノ随伴ウイルス5カプシドおよびその使用 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復pah基因功能的腺相關病毒組成物及其使用方法 |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| TW202233584A (zh) | 2020-11-11 | 2022-09-01 | 歐洲分子生物學實驗室 | 用於基因治療之經修飾病毒顆粒 |
| CN113647359A (zh) * | 2021-08-15 | 2021-11-16 | 芜湖职业技术学院 | 注射衣霉素诱导急性肝损伤小鼠模型的构建方法 |
| WO2023072181A1 (en) * | 2021-10-28 | 2023-05-04 | Shanghai Vitalgen Biopharma Co., Ltd. | Adeno-associated virus capsid |
| WO2023201368A2 (en) * | 2022-04-15 | 2023-10-19 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| CN119380820B (zh) * | 2024-10-10 | 2025-06-17 | 四川大学华西医院 | 一种aav2衣壳蛋白变体设计方法、系统和存储介质 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6156303A (en) * | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| EP1486567A1 (en) * | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2357010B1 (en) * | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| 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 |
| MX2010012592A (es) * | 2008-05-20 | 2011-05-05 | Eos Neuroscience Inc | Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas. |
| 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 |
| CN103060331B (zh) * | 2012-12-05 | 2014-10-08 | 南京医科大学第一附属医院 | 介导膜稳定cd40l基因的衣壳蛋白突变的双链重组腺相关病毒制备方法与应用 |
| US20180030096A1 (en) * | 2015-02-03 | 2018-02-01 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
-
2014
- 2014-05-21 AU AU2014274457A patent/AU2014274457B2/en active Active
- 2014-05-21 KR KR1020217008950A patent/KR102298579B1/ko active Active
- 2014-05-21 CN CN202010551899.1A patent/CN111763690A/zh active Pending
- 2014-05-21 JP JP2016515056A patent/JP6628286B2/ja active Active
- 2014-05-21 WO PCT/US2014/039015 patent/WO2014193716A2/en not_active Ceased
- 2014-05-21 EP EP14733803.2A patent/EP2999791B1/en active Active
- 2014-05-21 SG SG11201510757PA patent/SG11201510757PA/en unknown
- 2014-05-21 DK DK14733803.2T patent/DK2999791T3/en active
- 2014-05-21 SG SG10201709552XA patent/SG10201709552XA/en unknown
- 2014-05-21 KR KR1020157036164A patent/KR102234672B1/ko active Active
- 2014-05-21 CN CN201480041247.6A patent/CN105408486B/zh active Active
- 2014-05-21 KR KR1020227033180A patent/KR102649543B1/ko active Active
- 2014-05-21 CN CN202010039122.7A patent/CN111411125A/zh active Pending
- 2014-05-21 KR KR1020217027678A patent/KR20210111878A/ko not_active Ceased
- 2014-05-21 CA CA2917018A patent/CA2917018A1/en active Pending
- 2014-05-21 EP EP18202680.7A patent/EP3492597A3/en active Pending
-
2019
- 2019-11-27 JP JP2019214352A patent/JP7021786B2/ja active Active
-
2020
- 2020-01-03 AU AU2020200041A patent/AU2020200041B2/en active Active
-
2022
- 2022-01-28 JP JP2022011418A patent/JP7344595B2/ja active Active
- 2022-06-17 AU AU2022204246A patent/AU2022204246A1/en not_active Abandoned
Non-Patent Citations (1)
| Title |
|---|
| B CHENG ET AL, "Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells", GENE THERAPY, (2011-07-21), vol. 19, no. 4, pages 375 - 384 * |
Also Published As
| Publication number | Publication date |
|---|---|
| CN105408486B (zh) | 2020-07-14 |
| JP6628286B2 (ja) | 2020-01-08 |
| JP2022068181A (ja) | 2022-05-09 |
| EP2999791B1 (en) | 2018-12-05 |
| KR102234672B1 (ko) | 2021-04-06 |
| KR20210037001A (ko) | 2021-04-05 |
| CN111411125A (zh) | 2020-07-14 |
| KR20210111878A (ko) | 2021-09-13 |
| EP3492597A2 (en) | 2019-06-05 |
| EP3492597A3 (en) | 2019-08-28 |
| CN111763690A (zh) | 2020-10-13 |
| JP7344595B2 (ja) | 2023-09-14 |
| JP2020048571A (ja) | 2020-04-02 |
| SG11201510757PA (en) | 2016-01-28 |
| JP7021786B2 (ja) | 2022-02-17 |
| AU2020200041B2 (en) | 2022-03-17 |
| CA2917018A1 (en) | 2014-12-04 |
| KR102298579B1 (ko) | 2021-09-07 |
| WO2014193716A2 (en) | 2014-12-04 |
| KR20220136477A (ko) | 2022-10-07 |
| WO2014193716A3 (en) | 2015-01-22 |
| AU2020200041A1 (en) | 2020-02-06 |
| KR20160010617A (ko) | 2016-01-27 |
| EP2999791A2 (en) | 2016-03-30 |
| HK1217730A1 (en) | 2017-01-20 |
| JP2016520311A (ja) | 2016-07-14 |
| AU2014274457A1 (en) | 2016-01-21 |
| DK2999791T3 (en) | 2019-03-18 |
| KR102649543B1 (ko) | 2024-03-21 |
| CN105408486A (zh) | 2016-03-16 |
| AU2022204246A1 (en) | 2022-07-21 |
| SG10201709552XA (en) | 2018-01-30 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2020200041B2 (en) | Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer | |
| US20210253644A1 (en) | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer | |
| US10815279B2 (en) | Capsid-modified rAAV vector compositions and methods therefor | |
| US12358954B2 (en) | Capsid-modified rAAV vector compositions and methods therefor | |
| HK40009693A (en) | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer | |
| HK40032412A (en) | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer | |
| HK1217730B (en) | Capsid-modified, raav3 vector compositions and methods of use in gene therapy of human liver cancer | |
| HK40039678A (en) | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer | |
| Class et al. | Patent application title: CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS AND METHODS THEREFOR Inventors: Arun Srivastava (Gainesville, FL, US) Arun Srivastava (Gainesville, FL, US) George V. Aslanidi (Gainesville, FL, US) Sergei Zolotukhin (Gainesville, FL, US) Sergei Zolotukhin (Gainesville, FL, US) Mavis Agbandje-Mckenna (Gainesville, FL, US) Kim M. Van Vliet (Gainesville, FL, US) Li Zhong (Boxborough, MA, US) Lakshmanan Govindasamy (Gainesville, FL, US) Assignees: University of Florida Research Foundation Inc. |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| DA3 | Amendments made section 104 |
Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE NAME OF THE INVENTOR TO READ SRIVASTAVA, ARUN; ZHONG, LI; ZOLOTUKHIN, SERGEI; ASLANIDI, GEORGE V.; AGBANDJE-MCKENNA, MAVIS; VAN VLIET, KIM M. AND LING, CHEN |
|
| FGA | Letters patent sealed or granted (standard patent) |