CN110770346B - 多倍体腺相关病毒载体及其制备和使用方法 - Google Patents
多倍体腺相关病毒载体及其制备和使用方法 Download PDFInfo
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- CN110770346B CN110770346B CN201880031964.9A CN201880031964A CN110770346B CN 110770346 B CN110770346 B CN 110770346B CN 201880031964 A CN201880031964 A CN 201880031964A CN 110770346 B CN110770346 B CN 110770346B
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- C12N15/09—Recombinant DNA-technology
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- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16641—Use of virus, viral particle or viral elements as a vector
- C12N2710/16644—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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- C12N2750/14011—Parvoviridae
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- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- C12N2750/14123—Virus like particles [VLP]
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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
- C12N2750/14142—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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- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- Health & Medical Sciences (AREA)
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- Virology (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
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- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202311805114.9A CN117801075A (zh) | 2017-03-15 | 2018-03-15 | 多倍体腺相关病毒载体及其制备和使用方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762471762P | 2017-03-15 | 2017-03-15 | |
| US62/471762 | 2017-03-15 | ||
| US201762520901P | 2017-06-16 | 2017-06-16 | |
| US62/520901 | 2017-06-16 | ||
| US201862630558P | 2018-02-14 | 2018-02-14 | |
| US62/630558 | 2018-02-14 | ||
| PCT/US2018/022725 WO2018170310A1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202311805114.9A Division CN117801075A (zh) | 2017-03-15 | 2018-03-15 | 多倍体腺相关病毒载体及其制备和使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN110770346A CN110770346A (zh) | 2020-02-07 |
| CN110770346B true CN110770346B (zh) | 2024-01-12 |
Family
ID=63522576
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201880031964.9A Active CN110770346B (zh) | 2017-03-15 | 2018-03-15 | 多倍体腺相关病毒载体及其制备和使用方法 |
| CN202311805114.9A Pending CN117801075A (zh) | 2017-03-15 | 2018-03-15 | 多倍体腺相关病毒载体及其制备和使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202311805114.9A Pending CN117801075A (zh) | 2017-03-15 | 2018-03-15 | 多倍体腺相关病毒载体及其制备和使用方法 |
Country Status (9)
| Country | Link |
|---|---|
| EP (3) | EP3610023B1 (https=) |
| JP (1) | JP7132934B2 (https=) |
| CN (2) | CN110770346B (https=) |
| AU (2) | AU2018234695B2 (https=) |
| CA (1) | CA3054711A1 (https=) |
| ES (1) | ES2966692T3 (https=) |
| IL (3) | IL316259A (https=) |
| NZ (1) | NZ756587A (https=) |
| WO (1) | WO2018170310A1 (https=) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| WO2019006418A2 (en) | 2017-06-30 | 2019-01-03 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | 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 |
| WO2019216932A1 (en) * | 2018-05-07 | 2019-11-14 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| US20200069819A1 (en) * | 2018-05-14 | 2020-03-05 | Biomarin Pharmaceutical Inc. | Stable expression of aav vectors in juvenile subjects |
| MX2021005727A (es) * | 2018-11-16 | 2021-09-10 | Spark Therapeutics Inc | Ensayo in vitro para detectar potenciadores e inhibidores de la transducción de vectores virales adenoasociados (aav) y/o para detectar o cuantificar anticuerpos de unión anti-aav. |
| CN113395975A (zh) * | 2018-11-20 | 2021-09-14 | 宾夕法尼亚州大学信托人 | 用于靶向血脑屏障的组合物和方法 |
| AU2019418750A1 (en) * | 2019-01-02 | 2021-07-22 | Intima Bioscience, Inc. | Modified adeno-associated viral vectors for use in genetic engineering |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| EP3955970A1 (en) * | 2019-04-15 | 2022-02-23 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
| PE20220014A1 (es) * | 2019-04-29 | 2022-01-11 | Univ Pennsylvania | Nuevas capsides de aav y composiciones que las contienen |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
| AR122013A1 (es) * | 2020-05-05 | 2022-08-03 | Univ Duke | Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso |
| CN116121274B (zh) * | 2020-07-29 | 2024-11-19 | 北京三诺佳邑生物技术有限责任公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| JP2024515626A (ja) * | 2021-04-16 | 2024-04-10 | アスクレピオス バイオファーマシューティカル, インコーポレイテッド | 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン |
| CA3242989A1 (en) * | 2021-12-21 | 2023-06-29 | Research Institute At Nationwide Children's Hospital | MATERIALS AND METHODS FOR THE TREATMENT OF MUSCULAR BELLY DYSTROPHY |
| EP4703468A1 (en) * | 2024-08-29 | 2026-03-04 | Sartorius Albumedix Limited | Methods and uses |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0766569B1 (en) * | 1994-06-30 | 2006-01-11 | University Of Pittsburgh | Aav capsid vehicles for molecular transfer |
| WO2012112578A2 (en) * | 2011-02-14 | 2012-08-23 | The Children's Hospital Of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| US20150023924A1 (en) * | 2013-07-22 | 2015-01-22 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
Family Cites Families (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| AU7966987A (en) | 1986-09-08 | 1988-04-07 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| WO1990005142A1 (en) | 1988-11-10 | 1990-05-17 | Imperial Cancer Research Technology Ltd. | Polypeptides |
| US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| CA2223837C (en) | 1995-06-07 | 2010-05-25 | University Of North Carolina At Chapel Hill | Aav transduction of myoblasts |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| DE69636937T3 (de) | 1995-12-15 | 2011-01-05 | Virxsys Corp. | Durch trans-spaltung erhaltene therapeutische molekule |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| EP1082413B1 (en) | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| CA2349838C (en) | 1998-11-05 | 2011-06-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| CA2373110A1 (en) * | 2000-03-14 | 2001-09-20 | Neurologix, Inc. | Production of chimeric capsid vectors |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
| US6648075B2 (en) * | 2001-07-13 | 2003-11-18 | Weatherford/Lamb, Inc. | Method and apparatus for expandable liner hanger with bypass |
| US7071172B2 (en) | 2002-04-30 | 2006-07-04 | The University Of North Carolina At Chapel Hill | Secretion signal vectors |
| WO2004027019A2 (en) * | 2002-05-01 | 2004-04-01 | University Of Florida Research Foundation, Inc. | Improved raav expression systems for genetic modification of specific capsid proteins |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| JP4346526B2 (ja) | 2004-08-31 | 2009-10-21 | 株式会社東芝 | 半導体集積回路装置 |
| CN101056539B (zh) | 2004-09-09 | 2012-12-12 | 综合医院公司 | 调节心脏细胞中的磷酸酶活性 |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| JP2008539698A (ja) | 2005-04-29 | 2008-11-20 | 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 | 転写後レベルでの核酸発現調節のための方法および組成物 |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| US7989606B2 (en) | 2006-02-10 | 2011-08-02 | The University Of Cincinnati | Phosphatase inhibitor protein-1 as a regulator of cardiac function |
| WO2008027084A2 (en) * | 2006-04-28 | 2008-03-06 | The Trustees Of The University Of Pennsylvania | Modified aav vectors having reduced capsid immunogenicity and use thereof |
| JP5575486B2 (ja) | 2007-01-18 | 2014-08-20 | ユニヴァーシティ オブ ミズーリー−コロンビア | 筋鞘にnNOSを回復させる合成ミニ/ミクロジストロフィン遺伝子 |
| US20090215879A1 (en) | 2008-02-26 | 2009-08-27 | University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| US20120093775A1 (en) * | 2009-03-27 | 2012-04-19 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
| US9441206B2 (en) | 2011-10-28 | 2016-09-13 | The University Of North Carolina At Chapel Hill | Cell line for production of adeno-associated virus |
| AU2013249202B2 (en) | 2012-04-18 | 2018-08-09 | The Children's Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using AAV capsid variants |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| WO2016210170A1 (en) * | 2015-06-23 | 2016-12-29 | The Children's Hospital Of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
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2018
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Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0766569B1 (en) * | 1994-06-30 | 2006-01-11 | University Of Pittsburgh | Aav capsid vehicles for molecular transfer |
| WO2012112578A2 (en) * | 2011-02-14 | 2012-08-23 | The Children's Hospital Of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| US20150023924A1 (en) * | 2013-07-22 | 2015-01-22 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
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| NZ756587A (en) | 2025-12-19 |
| AU2024227303A1 (en) | 2024-10-31 |
| IL268891B2 (en) | 2024-09-01 |
| EP3610023A4 (en) | 2021-01-20 |
| IL268891A (en) | 2019-10-31 |
| IL312266B2 (en) | 2025-03-01 |
| IL312266B1 (en) | 2024-11-01 |
| CN110770346A (zh) | 2020-02-07 |
| JP7132934B2 (ja) | 2022-09-07 |
| IL268891B1 (en) | 2024-05-01 |
| CA3054711A1 (en) | 2018-09-20 |
| EP4303225A2 (en) | 2024-01-10 |
| EP3610023A1 (en) | 2020-02-19 |
| AU2018234695B2 (en) | 2024-10-03 |
| EP3610023B1 (en) | 2023-10-04 |
| EP4303225A3 (en) | 2024-04-10 |
| AU2018234695A1 (en) | 2019-09-12 |
| EP4303225B1 (en) | 2025-11-26 |
| JP2020511127A (ja) | 2020-04-16 |
| WO2018170310A1 (en) | 2018-09-20 |
| ES2966692T3 (es) | 2024-04-23 |
| IL316259A (en) | 2024-12-01 |
| CN117801075A (zh) | 2024-04-02 |
| IL312266A (en) | 2024-06-01 |
| EP4707292A2 (en) | 2026-03-11 |
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