AR124119A2 - Partículas virales modificadas y usos de estas - Google Patents
Partículas virales modificadas y usos de estasInfo
- Publication number
- AR124119A2 AR124119A2 ARP210103220A ARP210103220A AR124119A2 AR 124119 A2 AR124119 A2 AR 124119A2 AR P210103220 A ARP210103220 A AR P210103220A AR P210103220 A ARP210103220 A AR P210103220A AR 124119 A2 AR124119 A2 AR 124119A2
- Authority
- AR
- Argentina
- Prior art keywords
- aav
- capsid
- capsid protein
- particles
- protein
- Prior art date
Links
- 239000002245 particle Substances 0.000 title abstract 5
- 230000003612 virological effect Effects 0.000 title abstract 2
- 108090000565 Capsid Proteins Proteins 0.000 abstract 6
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 6
- 210000000234 capsid Anatomy 0.000 abstract 4
- 230000001225 therapeutic effect Effects 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 241000288906 Primates Species 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 238000011277 treatment modality Methods 0.000 abstract 1
Classifications
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- 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)
- Molecular Biology (AREA)
- Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Toxicology (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
En la presente se proporcionan composiciones y métodos para adaptar partículas de virus adenoasociado (AAV) que comprenden cápsides de AAV de animales no primates, AAV remoto, o una combinación de estos. El AAV adaptado, en consecuencia, puede ser una plataforma de terapia génica viable para el tratamiento de un paciente que lo necesita, y puede ser particularmente útil en pacientes excluidos de modalidades de tratamiento actuales que implican partículas de AAV terapéuticas actuales debido a su alto título de anticuerpos contra las partículas de AAV terapéuticas actuales. Las partículas virales de AAV recombinante comprenden (i) una cápside de AAV que comprende proteínas de la cápside de AAV VP1, VP2 y VP3, y (ii) empaquetada dentro de la cápside de AAV, una secuencia de ácido nucleico que comprende una secuencia de Repetición Terminal Invertida (ITR) de AAV, en donde al menos una de la proteína de la cápside VP1 de AAV, cualquier porción de la proteína de la cápside VP1 de AAV, la proteína de la cápside VP2 de AAV, cualquier porción de la proteína de la cápside VP2 de AAV, la proteína de la cápside VP3 de AAV, y cualquier porción de la proteína de la cápside VP3 de AAV comprende una secuencia de aminoácidos.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962852791P | 2019-05-24 | 2019-05-24 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR124119A2 true AR124119A2 (es) | 2023-02-15 |
Family
ID=71094822
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200101465A AR118997A1 (es) | 2019-05-24 | 2020-05-22 | Partículas virales modificadas y usos de estas |
| ARP210103220A AR124119A2 (es) | 2019-05-24 | 2021-11-19 | Partículas virales modificadas y usos de estas |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200101465A AR118997A1 (es) | 2019-05-24 | 2020-05-22 | Partículas virales modificadas y usos de estas |
Country Status (19)
| Country | Link |
|---|---|
| US (1) | US20220241430A1 (es) |
| EP (1) | EP3976631A1 (es) |
| JP (3) | JP2022533438A (es) |
| KR (1) | KR20220011664A (es) |
| CN (2) | CN114989267A (es) |
| AR (2) | AR118997A1 (es) |
| AU (1) | AU2020283537A1 (es) |
| BR (1) | BR112021023692A2 (es) |
| CA (1) | CA3140386A1 (es) |
| CL (2) | CL2021003096A1 (es) |
| CO (1) | CO2021017692A2 (es) |
| IL (1) | IL288233A (es) |
| MA (1) | MA56035A (es) |
| MX (1) | MX2021014338A (es) |
| PE (1) | PE20212357A1 (es) |
| PH (1) | PH12021552887A1 (es) |
| SG (1) | SG11202112917PA (es) |
| TW (1) | TW202110869A (es) |
| WO (1) | WO2020242984A1 (es) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200128112A (ko) | 2018-02-28 | 2020-11-11 | 유니버시티 오브 피츠버그-오브 더 커먼웰쓰 시스템 오브 하이어 에듀케이션 | Aav에 기반한 유전자 및 단백질 전달을 위한 모듈식 시스템 |
| JP2022533438A (ja) * | 2019-05-24 | 2022-07-22 | 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 | 改変ウイルス粒子およびその使用 |
| 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 |
| EP4314021A4 (en) * | 2021-04-30 | 2025-12-17 | Univ Duke | COMPOSITIONS WITH CHIMERATIVE CAPSIBILITIES FROM ADENO-ASSOCIATED VIRUS AND METHOD FOR USE THEM |
| 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 |
| WO2023240124A1 (en) | 2022-06-07 | 2023-12-14 | 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 |
| US20250163468A1 (en) * | 2023-11-21 | 2025-05-22 | Regeneron Pharmaceuticals, Inc. | Covalently surface modified adeno-associated virus production by in vitro conjugation, and purification of covalently surface modified adeno-associated virus |
Family Cites Families (16)
| 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 |
| US20030215422A1 (en) | 1996-09-11 | 2003-11-20 | John A. Chiorini | 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 |
| ES2313784T3 (es) * | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| AU768729B2 (en) | 1998-11-05 | 2004-01-08 | Trustees Of The University Of Pennsylvania, The | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| HU230406B1 (hu) * | 2001-11-13 | 2016-04-28 | The Trustees Of The University Of Pennsylvania | Eljárás adeno-asszociált vírus(AAV)szekvenciák detektálására és/vagy azonosítására és az azzal azonosított új szekvenciák izolálására |
| ES2602610T3 (es) | 2007-05-31 | 2017-02-21 | Medigene Ag | Proteína estructural mutada de un parvovirus |
| ES2595376T3 (es) | 2009-12-10 | 2016-12-29 | Regeneron Pharmaceuticals, Inc. | Ratones que producen anticuerpos de cadena pesada |
| 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 |
| CA3124228C (en) | 2014-03-21 | 2024-05-14 | Regeneron Pharmaceuticals, Inc. | Non-human animals that make single domain binding proteins |
| EP3151866B1 (en) * | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN116891534A (zh) * | 2017-06-27 | 2023-10-17 | 瑞泽恩制药公司 | 向性修饰的重组病毒粒子和其用于将遗传物质靶向引入至人类细胞中的用途 |
| BR112019027854A2 (pt) | 2017-06-27 | 2020-07-07 | Regeneron Pharmaceuticals, Inc. | proteínas de capsídeo viral recombinante, ácido nucleico isolado, vetor viral recombinante, composição, métodos de direcionamento do vetor viral recombinante de interesse a uma célula alvo, de distribuição de um nucleotídeo de interesse a uma célula alvo, de inativação de uma proteína do capsídeo viral e de produção de um vetor viral, célula empacotadora para produzir um vetor viral, e, molécula e proteína de ligação. |
| JP2022533438A (ja) * | 2019-05-24 | 2022-07-22 | 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 | 改変ウイルス粒子およびその使用 |
-
2020
- 2020-05-22 JP JP2021569402A patent/JP2022533438A/ja active Pending
- 2020-05-22 TW TW109117155A patent/TW202110869A/zh unknown
- 2020-05-22 CN CN202210622571.3A patent/CN114989267A/zh active Pending
- 2020-05-22 MX MX2021014338A patent/MX2021014338A/es unknown
- 2020-05-22 EP EP20733099.4A patent/EP3976631A1/en active Pending
- 2020-05-22 AR ARP200101465A patent/AR118997A1/es unknown
- 2020-05-22 WO PCT/US2020/034328 patent/WO2020242984A1/en not_active Ceased
- 2020-05-22 BR BR112021023692A patent/BR112021023692A2/pt unknown
- 2020-05-22 PE PE2021001934A patent/PE20212357A1/es unknown
- 2020-05-22 CA CA3140386A patent/CA3140386A1/en active Pending
- 2020-05-22 US US17/612,669 patent/US20220241430A1/en active Pending
- 2020-05-22 KR KR1020217041074A patent/KR20220011664A/ko active Pending
- 2020-05-22 PH PH1/2021/552887A patent/PH12021552887A1/en unknown
- 2020-05-22 SG SG11202112917PA patent/SG11202112917PA/en unknown
- 2020-05-22 MA MA056035A patent/MA56035A/fr unknown
- 2020-05-22 CN CN202080038612.3A patent/CN113874386A/zh active Pending
- 2020-05-22 AU AU2020283537A patent/AU2020283537A1/en active Pending
-
2021
- 2021-11-18 IL IL288233A patent/IL288233A/en unknown
- 2021-11-19 AR ARP210103220A patent/AR124119A2/es unknown
- 2021-11-23 CL CL2021003096A patent/CL2021003096A1/es unknown
- 2021-12-22 CO CONC2021/0017692A patent/CO2021017692A2/es unknown
-
2023
- 2023-03-03 CL CL2023000629A patent/CL2023000629A1/es unknown
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2024
- 2024-11-11 JP JP2024196799A patent/JP2025032110A/ja active Pending
-
2025
- 2025-05-22 JP JP2025085876A patent/JP2025119015A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3976631A1 (en) | 2022-04-06 |
| US20220241430A1 (en) | 2022-08-04 |
| CN113874386A (zh) | 2021-12-31 |
| PE20212357A1 (es) | 2021-12-17 |
| JP2025032110A (ja) | 2025-03-11 |
| JP2025119015A (ja) | 2025-08-13 |
| WO2020242984A1 (en) | 2020-12-03 |
| TW202110869A (zh) | 2021-03-16 |
| JP2022533438A (ja) | 2022-07-22 |
| IL288233A (en) | 2022-01-01 |
| CL2023000629A1 (es) | 2023-10-20 |
| SG11202112917PA (en) | 2021-12-30 |
| CO2021017692A2 (es) | 2022-01-17 |
| CN114989267A (zh) | 2022-09-02 |
| CL2021003096A1 (es) | 2022-09-20 |
| CA3140386A1 (en) | 2020-12-03 |
| KR20220011664A (ko) | 2022-01-28 |
| MA56035A (fr) | 2022-04-06 |
| WO2020242984A9 (en) | 2021-01-14 |
| MX2021014338A (es) | 2022-01-06 |
| AR118997A1 (es) | 2021-11-17 |
| PH12021552887A1 (en) | 2022-09-05 |
| BR112021023692A2 (pt) | 2022-01-04 |
| AU2020283537A1 (en) | 2021-12-16 |
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