CA3140386A1 - Modified viral particles and uses thereof - Google Patents
Modified viral particles and uses thereof Download PDFInfo
- Publication number
- CA3140386A1 CA3140386A1 CA3140386A CA3140386A CA3140386A1 CA 3140386 A1 CA3140386 A1 CA 3140386A1 CA 3140386 A CA3140386 A CA 3140386A CA 3140386 A CA3140386 A CA 3140386A CA 3140386 A1 CA3140386 A1 CA 3140386A1
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- CA
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- Prior art keywords
- aav
- protein
- amino acid
- acid sequence
- capsid protein
- 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.)
- Pending
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Classifications
-
- 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
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
- C07K14/315—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
- C12N2750/14151—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
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| US62/852,791 | 2019-05-24 | ||
| PCT/US2020/034328 WO2020242984A1 (en) | 2019-05-24 | 2020-05-22 | Modified viral particles and uses thereof |
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| CA3140386A1 true CA3140386A1 (en) | 2020-12-03 |
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| AU2019227910B2 (en) | 2018-02-28 | 2025-07-17 | The Regents Of The University Of California | A modular system for gene and protein delivery based on AAV |
| KR20220011664A (ko) * | 2019-05-24 | 2022-01-28 | 리제너론 파마슈티칼스 인코포레이티드 | 변형된 바이러스 입자 및 이의 용도 |
| WO2022187377A1 (en) * | 2021-03-02 | 2022-09-09 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Adeno-associated viruses and methods and materials for making and using adeno-associated viruses |
| WO2022232575A1 (en) * | 2021-04-30 | 2022-11-03 | Duke University | Compositions comprising adeno-associated virus chimera capsid proteins and methods of using the same |
| CA3222914A1 (en) * | 2021-06-23 | 2022-12-29 | Dyno Therapeutics, Inc. | Capsid variants and methods of using the same |
| CA3256953A1 (en) | 2022-05-09 | 2023-11-16 | Regeneron Pharmaceuticals, Inc. | VECTORS AND METHODS FOR IN VIVO ANTIBODY PRODUCTION |
| EP4536836A1 (en) | 2022-06-07 | 2025-04-16 | Regeneron Pharmaceuticals, Inc. | Pseudotyped viral particles for targeting tcr-expressing cells |
| WO2025042462A2 (en) * | 2023-08-24 | 2025-02-27 | Ohio State Innovation Foundation | Assays and methods for quality control of genome packaging for gene therapy |
| WO2025054526A1 (en) * | 2023-09-07 | 2025-03-13 | Regeneron Pharmaceuticals, Inc. | Production and purification of covalently surface modified adeno-associated virus |
| WO2025111473A1 (en) * | 2023-11-21 | 2025-05-30 | Regeneron Pharmaceuticals, Inc. | Covalently surface modified adeno-associated virus production by in vitro conjugation, and purification of covalently surface modified adeno-associated virus |
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| WO1997005266A1 (en) | 1995-07-25 | 1997-02-13 | Introgene B.V. | Methods and means for targeted gene delivery |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | 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 |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| DK1127150T3 (da) | 1998-11-05 | 2007-09-24 | Univ Pennsylvania | Nucleinsyresekvenser fra det adeno-associerede virus af serotype 1 samt vektorer og værtsceller, der indeholder disse |
| SG168422A1 (en) * | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2602610T3 (es) | 2007-05-31 | 2017-02-21 | Medigene Ag | Proteína estructural mutada de un parvovirus |
| MY172472A (en) | 2009-12-10 | 2019-11-26 | Regeneron Pharma | Mice that make heavy chain antibodies |
| GB201002362D0 (en) | 2010-02-11 | 2010-03-31 | Isis Innovation | Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds |
| IL293294B2 (en) * | 2013-03-15 | 2023-04-01 | Univ North Carolina Chapel Hill | Methods and compounds for ligation of paired aav glycan vectors |
| ES2990174T3 (es) * | 2014-03-10 | 2024-11-29 | Uniqure Ip Bv | Vectores de AAV mejorados producidos en células de insecto |
| BR112016021572A2 (pt) | 2014-03-21 | 2017-10-03 | Regeneron Pharma | Métodos para preparar um animal não humano geneticamente modificado e para produzir proteínas de ligação, hibridoma, ácido nucleico, célula, e, proteína de ligação |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| LT3645553T (lt) * | 2017-06-27 | 2023-04-25 | Regeneron Pharmaceuticals, Inc. | Modifikuoto tropizmo rekombinantinės viruso dalelės ir jų panaudojimas genetinės medžiagos tiksliniam įvedimui į žmogaus ląsteles |
| SI3645551T1 (sl) | 2017-06-27 | 2024-06-28 | Regeneron Pharmaceuticals, Inc. | S tropizmi spremenjeni rekombinantni virusni vektorji in njihova uporaba za tarčni vnos genetskega materiala v človeške celice |
| KR20220011664A (ko) * | 2019-05-24 | 2022-01-28 | 리제너론 파마슈티칼스 인코포레이티드 | 변형된 바이러스 입자 및 이의 용도 |
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| AR118997A1 (es) | 2021-11-17 |
| BR112021023692A2 (pt) | 2022-01-04 |
| CL2023000629A1 (es) | 2023-10-20 |
| CL2021003096A1 (es) | 2022-09-20 |
| EP3976631A1 (en) | 2022-04-06 |
| KR20220011664A (ko) | 2022-01-28 |
| US20220241430A1 (en) | 2022-08-04 |
| CN114989267A (zh) | 2022-09-02 |
| WO2020242984A9 (en) | 2021-01-14 |
| JP2022533438A (ja) | 2022-07-22 |
| CN113874386A (zh) | 2021-12-31 |
| MX2021014338A (es) | 2022-01-06 |
| CO2021017692A2 (es) | 2022-01-17 |
| PH12021552887A1 (en) | 2022-09-05 |
| SG11202112917PA (en) | 2021-12-30 |
| WO2020242984A1 (en) | 2020-12-03 |
| IL288233A (en) | 2022-01-01 |
| MA56035A (fr) | 2022-04-06 |
| AR124119A2 (es) | 2023-02-15 |
| JP2025032110A (ja) | 2025-03-11 |
| PE20212357A1 (es) | 2021-12-17 |
| JP2025119015A (ja) | 2025-08-13 |
| AU2020283537A1 (en) | 2021-12-16 |
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