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CL2019003842A1 - Vectores virales recombinantes con tropismo modificado y usos de estos para la introducción dirigida de material genético a células humanas. - Google Patents

Vectores virales recombinantes con tropismo modificado y usos de estos para la introducción dirigida de material genético a células humanas.

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Publication number
CL2019003842A1
CL2019003842A1 CL2019003842A CL2019003842A CL2019003842A1 CL 2019003842 A1 CL2019003842 A1 CL 2019003842A1 CL 2019003842 A CL2019003842 A CL 2019003842A CL 2019003842 A CL2019003842 A CL 2019003842A CL 2019003842 A1 CL2019003842 A1 CL 2019003842A1
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CL
Chile
Prior art keywords
specific
recombinant viral
viral vectors
human cells
genetic material
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Application number
CL2019003842A
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English (en)
Inventor
Andrew J Murphy
Christos Kyratsous
Cheng Wang
Leah Sabin
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Regeneron Pharma
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Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of CL2019003842A1 publication Critical patent/CL2019003842A1/es

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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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Abstract

EN LA PRESENTE DESCRIPCIÓN SE PROPORCIONAN COMPOSICIONES Y MÉTODOS PARA REDIRECCIONAR PROTEÍNAS DE LA CÁPSIDE VIRAL/CÁPSIDES/ VECTORES RECOMBINANTES, POR EJEMPLO IN VIVO, CON UNA MOLÉCULA DE UNIÓN MULTIESPECÍFICA, TAL COMO UN ANTICUERPO BIESPECÍFICO, QUE SE UNE ESPECÍFICAMENTE A UN EPÍTOPO HETERÓLOGO PRESENTADO POR LA PROTEÍNA DE LA CÁPSIDE Y UNA PROTEÍNA EXPRESADA EN LA CÉLULA DE INTERÉS PARA EL SUMINISTRO DIRIGIDO DE UN NUCLEÓTIDO DE INTERÉS.
CL2019003842A 2017-06-27 2019-12-26 Vectores virales recombinantes con tropismo modificado y usos de estos para la introducción dirigida de material genético a células humanas. CL2019003842A1 (es)

Applications Claiming Priority (1)

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US201762525704P 2017-06-27 2017-06-27

Publications (1)

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CL2019003842A1 true CL2019003842A1 (es) 2020-07-24

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CL2019003842A CL2019003842A1 (es) 2017-06-27 2019-12-26 Vectores virales recombinantes con tropismo modificado y usos de estos para la introducción dirigida de material genético a células humanas.
CL2020003343A CL2020003343A1 (es) 2017-06-27 2020-12-22 Vectores virales recombinantes con tropismo modificado y usos de los mismos para la introducción dirigida de material genético a células humanas (divisional de la solicitud 201903842)
CL2024001376A CL2024001376A1 (es) 2017-06-27 2024-05-07 Vectores virales recombinantes con tropismo modificado y sus usos

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CL2024001376A CL2024001376A1 (es) 2017-06-27 2024-05-07 Vectores virales recombinantes con tropismo modificado y sus usos

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US (1) US20200140491A1 (es)
EP (2) EP4372008A3 (es)
JP (3) JP7372154B2 (es)
KR (3) KR20250160215A (es)
CN (2) CN110799524A (es)
AU (2) AU2018290882B2 (es)
BR (1) BR112019027854A2 (es)
CA (1) CA3066947A1 (es)
CL (3) CL2019003842A1 (es)
CO (1) CO2019014683A2 (es)
DK (1) DK3645551T5 (es)
ES (1) ES2978295T3 (es)
FI (1) FI3645551T3 (es)
HR (1) HRP20240773T1 (es)
HU (1) HUE066398T2 (es)
IL (2) IL317938A (es)
LT (1) LT3645551T (es)
MA (1) MA49513B1 (es)
MD (1) MD3645551T2 (es)
MX (2) MX2020000244A (es)
PE (1) PE20200488A1 (es)
PH (1) PH12019550266A1 (es)
PL (1) PL3645551T3 (es)
PT (1) PT3645551T (es)
RS (1) RS65569B1 (es)
SG (1) SG11201911610TA (es)
SI (1) SI3645551T1 (es)
SM (1) SMT202400189T1 (es)
WO (1) WO2019006043A1 (es)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019067840A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
AU2019354995B2 (en) 2018-10-02 2024-12-12 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
EP3736330A1 (en) * 2019-05-08 2020-11-11 European Molecular Biology Laboratory Modified adeno-associated virus (aav) particles for gene therapy
MA56035A (fr) 2019-05-24 2022-04-06 Regeneron Pharma Particules virales modifiées et leurs utilisations
PE20230767A1 (es) 2020-05-13 2023-05-09 Voyager Therapeutics Inc Redireccion del tropismo de las capsides de aav
CA3201533A1 (en) 2020-11-11 2022-05-19 European Molecular Biology Laboratory Modified viral particles for gene therapy
CN115873987A (zh) * 2021-09-30 2023-03-31 北京五加和基因科技有限公司 检测腺相关病毒载体感染滴度的方法和试剂盒
IL311861A (en) 2021-11-02 2024-06-01 Voyager Therapeutics Inc Aav capsid variants and uses thereof
PE20241212A1 (es) 2021-11-04 2024-06-06 Regeneron Pharma Particulas virales redirigidas a musculo esqueletico
CN114213505B (zh) * 2021-12-10 2022-09-20 和元生物技术(上海)股份有限公司 一种适用于特异感染u87-mg细胞的腺相关病毒突变体
CN119095866A (zh) * 2022-02-22 2024-12-06 北卡罗来纳大学查佩尔希尔分校 嵌合神经向性aav衣壳
CN115044615B (zh) * 2022-04-28 2025-09-05 三峡大学 一种靶向感染乳腺癌细胞的aav载体及应用
CA3256953A1 (en) * 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. VECTORS AND METHODS FOR IN VIVO ANTIBODY PRODUCTION
WO2023215947A1 (en) * 2022-05-13 2023-11-16 Children's Medical Research Institute Adeno-associated virus capsids
IL318636A (en) 2022-07-29 2025-03-01 Regeneron Pharma Viral particles retargeted to transferrin receptor 1
EP4619438A2 (en) 2022-11-14 2025-09-24 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
KR20250134733A (ko) * 2022-12-16 2025-09-11 리제너론 파아마슈티컬스, 인크. Aav 입자에 결합하는 항원-결합 분자 및 용도
WO2024131982A1 (zh) * 2022-12-23 2024-06-27 北京大学宁波海洋药物研究院 衣壳蛋白-抗体偶联物、修饰的病毒、药物组合和用途
KR20250150011A (ko) 2023-02-13 2025-10-17 리제너론 파마슈티칼스 인코포레이티드 항-인간 cacng1 항체를 이용한 근육 관련 장애 치료
CN121039163A (zh) 2023-05-02 2025-11-28 瑞泽恩制药公司 抗人m-钙粘蛋白(cdh15)抗体、缀合物及其用于将遗传有效负载递送至肌肉细胞的用途
CN118955652A (zh) * 2023-05-15 2024-11-15 杭州新昶基因技术有限公司 腺相关病毒衣壳蛋白、包含其的腺相关病毒及应用
EP4630457A1 (en) * 2023-08-18 2025-10-15 Regeneron Pharmaceuticals, Inc. Bispecific antigen-binding molecules and uses thereof
WO2025072501A2 (en) * 2023-09-26 2025-04-03 The Trustees Of Princeton University Method for converting farnesyl pyrophosphate synthase into a geranyl pyrophosphate synthase
WO2025151796A1 (en) 2024-01-11 2025-07-17 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to nervous system tissues
WO2025160340A2 (en) 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Combination immunosuppression for inhibiting an immune response and enabling immunogen administration and re-administration
US20250277048A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. The use of cd40 inhibitors for inhibiting an immune response and enabling immunogen administration and re-administration
CN119039401B (zh) * 2024-08-19 2025-04-11 广州派真生物技术有限公司 腺相关病毒突变体及其应用

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0840797B1 (en) 1995-07-25 2014-09-03 Crucell Holland B.V. Methods and means for targeted gene delivery
EP1053013A4 (en) 1998-02-06 2004-12-22 Uab Research Foundation ADENOVIRUS VECTOR CONTAINING A HETEROLOGICAL PEPTIDEPITOP IN THE HI LOOP OF THE FIBER BUTTON
DE19849643A1 (de) * 1998-10-29 2000-05-04 Deutsches Krebsforsch An das AAV-Kapsid bindender, den Zelltropismus verändernder Antikörper und Verfahren zum gerichteten Gentransfer
DE19933719A1 (de) * 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
EP1546359A4 (en) * 2002-09-12 2007-05-09 Molecular Probes Inc JOB-SPECIFIC MARKING OF AFFINITY DAYS IN FUSION PROTEINS
US7273835B2 (en) * 2004-08-04 2007-09-25 Honeywell International Inc. Azeotrope-like compositions of difluoromethane
US9296816B2 (en) 2005-04-15 2016-03-29 Macrogenics, Inc. Covalent diabodies and uses thereof
US9963510B2 (en) 2005-04-15 2018-05-08 Macrogenics, Inc. Covalent diabodies and uses thereof
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
KR20120027055A (ko) 2009-06-26 2012-03-20 리제네론 파라마큐티칼스 인코포레이티드 천연 면역글로불린 포맷을 가지는 용이하게 분리된 이중특이성 항체
CN102711449B (zh) 2009-12-10 2015-01-07 瑞泽恩制药公司 生产重链抗体的小鼠
FR2955773B1 (fr) * 2010-02-01 2017-05-26 Commissariat Energie Atomique Complexe moleculaire de ciblage des antigenes vers les cellules presentatrices d'antigene et ses applications pour la vaccination
WO2014022540A1 (en) 2012-08-02 2014-02-06 Regeneron Pharmaceuticals, Inc. Multivalent antigen-binding proteins
CN105408486B (zh) * 2013-05-21 2020-07-14 佛罗里达大学研究基金会有限公司 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途
JP6636498B2 (ja) 2014-03-21 2020-01-29 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. 単一ドメイン結合タンパク質を作る非ヒト動物
WO2016054554A1 (en) * 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
DE102015207516A1 (de) * 2015-04-23 2016-10-27 Paul-Ehrlich-Institut Bundesamt Für Sera Und Impfstoffe Kopplung von Proteinen von Interesse (POI) mit viralen Vektoren mittels Intein-vermittelten Proteinspleißens

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