SG11201510757PA - 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 cancerInfo
- Publication number
- SG11201510757PA SG11201510757PA SG11201510757PA SG11201510757PA SG11201510757PA SG 11201510757P A SG11201510757P A SG 11201510757PA SG 11201510757P A SG11201510757P A SG 11201510757PA SG 11201510757P A SG11201510757P A SG 11201510757PA SG 11201510757P A SG11201510757P A SG 11201510757PA
- Authority
- SG
- Singapore
- Prior art keywords
- capsid
- modified
- gene therapy
- liver cancer
- human liver
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title 1
- 201000007270 liver cancer Diseases 0.000 title 1
- 208000014018 liver neoplasm Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/63—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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- 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
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K39/46—Cellular immunotherapy
- A61K39/461—Cellular immunotherapy characterised by the cell type used
- A61K39/4615—Dendritic cells
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- A—HUMAN NECESSITIES
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- A61K39/462—Cellular immunotherapy characterized by the effect or the function of the cells
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- A61K39/4634—Antigenic peptides; polypeptides
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- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/4644—Cancer antigens
- A61K39/464454—Enzymes
- A61K39/464457—Telomerase or [telomerase reverse transcriptase [TERT]
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- 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
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- A—HUMAN NECESSITIES
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- 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
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- A—HUMAN NECESSITIES
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- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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- 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
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- 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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- 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
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- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- 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
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
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- 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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- 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
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
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- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
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- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
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- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
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- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Mycology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Oncology (AREA)
- Manufacturing & Machinery (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)
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Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
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 |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201510757PA true SG11201510757PA (en) | 2016-01-28 |
Family
ID=51023068
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201709552XA SG10201709552XA (en) | 2013-05-21 | 2014-05-21 | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer |
SG11201510757PA SG11201510757PA (en) | 2013-05-21 | 2014-05-21 | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10201709552XA SG10201709552XA (en) | 2013-05-21 | 2014-05-21 | Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer |
Country Status (10)
Country | Link |
---|---|
EP (2) | EP2999791B1 (ru) |
JP (3) | JP6628286B2 (ru) |
KR (4) | KR102234672B1 (ru) |
CN (3) | CN111411125A (ru) |
AU (3) | AU2014274457B2 (ru) |
CA (1) | CA2917018A1 (ru) |
DK (1) | DK2999791T3 (ru) |
HK (1) | HK1217730A1 (ru) |
SG (2) | SG10201709552XA (ru) |
WO (1) | WO2014193716A2 (ru) |
Families Citing this family (39)
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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 |
US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
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 |
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
WO2016126857A1 (en) | 2015-02-03 | 2016-08-11 | 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 |
SG10202107733QA (en) | 2015-09-28 | 2021-09-29 | Univ North Carolina Chapel Hill | Methods and compositions for antibody-evading virus vectors |
WO2017070476A2 (en) * | 2015-10-22 | 2017-04-27 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav3 capsid library |
US11124546B2 (en) * | 2015-12-14 | 2021-09-21 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
AU2017313064B2 (en) * | 2016-08-16 | 2024-05-02 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
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 |
CA3054131C (en) * | 2017-02-21 | 2021-05-25 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
DK3645551T3 (da) * | 2017-06-27 | 2024-05-06 | Regeneron Pharma | Tropisme-modificerede rekombinante virusvektorer og anvendelser deraf til targetteret indføring af genetisk materiale i menneskeceller |
KR20200051708A (ko) * | 2017-09-08 | 2020-05-13 | 제너레이션 바이오 컴퍼니 | 비-바이러스성 캡시드-비함유 dna 벡터의 지질 나노입자 제형 |
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 |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
WO2019161365A1 (en) | 2018-02-19 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof |
CA3095127A1 (en) * | 2018-03-29 | 2019-10-03 | Asklepios Biopharmaceutical, Inc. | Liver tropic recombinant aav6 vectors that evade neutralization |
WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
US20210363192A1 (en) * | 2018-04-27 | 2021-11-25 | 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 |
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 |
PL3911354T3 (pl) * | 2019-01-18 | 2024-03-11 | Institut Pasteur | Terapia genowa zależna od aav przywracająca gen otoferliny |
MX2021011468A (es) | 2019-03-21 | 2021-12-15 | Vectores de virus adenoasociados recombinantes. | |
EP3736330A1 (en) * | 2019-05-08 | 2020-11-11 | European Molecular Biology Laboratory | Modified adeno-associated virus (aav) particles for gene therapy |
AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
JP2022540226A (ja) * | 2019-07-11 | 2022-09-14 | サントル ナショナル ドゥ ラ ルシェルシュ シアンティフィック | 化学修飾型アデノ随伴ウイルス |
WO2021076925A1 (en) | 2019-10-17 | 2021-04-22 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
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 |
WO2021108468A1 (en) * | 2019-11-25 | 2021-06-03 | University Of Florida Research Foundation, Incorporated | Novel aav3b variants that target human hepatocytes in the liver of humanized mice |
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 |
AU2021267856A1 (en) * | 2020-05-05 | 2022-12-01 | The University Of North Carolina At Chapel Hill | Modified adeno-associated virus 5 capsids and uses thereof |
EP4244209A1 (en) | 2020-11-11 | 2023-09-20 | European Molecular Biology Laboratory | Modified viral particles for gene therapy |
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 |
CN118201947A (zh) * | 2021-10-28 | 2024-06-14 | 上海天泽云泰生物医药有限公司 | 腺相关病毒衣壳 |
WO2023201368A2 (en) * | 2022-04-15 | 2023-10-19 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
Family Cites Families (8)
Publication number | Priority date | Publication date | Assignee | Title |
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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 |
DK2359867T3 (en) * | 2005-04-07 | 2015-01-05 | Univ Pennsylvania | A method for increasing an AAV vector function |
DK2191001T3 (en) * | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
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 |
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. |
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 |
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 |
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CN111411125A (zh) | 2020-07-14 |
KR20220136477A (ko) | 2022-10-07 |
EP3492597A3 (en) | 2019-08-28 |
AU2014274457A1 (en) | 2016-01-21 |
SG10201709552XA (en) | 2018-01-30 |
JP7021786B2 (ja) | 2022-02-17 |
JP2020048571A (ja) | 2020-04-02 |
CA2917018A1 (en) | 2014-12-04 |
AU2014274457B2 (en) | 2019-10-24 |
DK2999791T3 (en) | 2019-03-18 |
JP7344595B2 (ja) | 2023-09-14 |
WO2014193716A3 (en) | 2015-01-22 |
AU2022204246A1 (en) | 2022-07-21 |
KR20160010617A (ko) | 2016-01-27 |
KR102234672B1 (ko) | 2021-04-06 |
KR102298579B1 (ko) | 2021-09-07 |
EP2999791A2 (en) | 2016-03-30 |
CN105408486B (zh) | 2020-07-14 |
EP3492597A2 (en) | 2019-06-05 |
JP6628286B2 (ja) | 2020-01-08 |
CN105408486A (zh) | 2016-03-16 |
KR20210037001A (ko) | 2021-04-05 |
AU2020200041A1 (en) | 2020-02-06 |
KR20210111878A (ko) | 2021-09-13 |
AU2020200041B2 (en) | 2022-03-17 |
JP2022068181A (ja) | 2022-05-09 |
CN111763690A (zh) | 2020-10-13 |
WO2014193716A2 (en) | 2014-12-04 |
JP2016520311A (ja) | 2016-07-14 |
HK1217730A1 (zh) | 2017-01-20 |
EP2999791B1 (en) | 2018-12-05 |
KR102649543B1 (ko) | 2024-03-21 |
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