BR112021023692A2 - Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêutica - Google Patents
Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêuticaInfo
- Publication number
- BR112021023692A2 BR112021023692A2 BR112021023692A BR112021023692A BR112021023692A2 BR 112021023692 A2 BR112021023692 A2 BR 112021023692A2 BR 112021023692 A BR112021023692 A BR 112021023692A BR 112021023692 A BR112021023692 A BR 112021023692A BR 112021023692 A2 BR112021023692 A2 BR 112021023692A2
- Authority
- BR
- Brazil
- Prior art keywords
- associated virus
- viral particle
- adeno
- aav
- methods
- Prior art date
Links
- 239000002245 particle Substances 0.000 title abstract 7
- 241000702421 Dependoparvovirus Species 0.000 title abstract 5
- 230000003612 virological effect Effects 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 3
- 108090000565 Capsid Proteins Proteins 0.000 title abstract 2
- 102100023321 Ceruloplasmin Human genes 0.000 title abstract 2
- -1 and Substances 0.000 title abstract 2
- 102000039446 nucleic acids Human genes 0.000 title abstract 2
- 108020004707 nucleic acids Proteins 0.000 title abstract 2
- 150000007523 nucleic acids Chemical class 0.000 title abstract 2
- 239000002773 nucleotide Substances 0.000 title abstract 2
- 125000003729 nucleotide group Chemical group 0.000 title abstract 2
- 238000004806 packaging method and process Methods 0.000 title abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 title abstract 2
- 241000288906 Primates Species 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 238000002560 therapeutic procedure Methods 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)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962852791P | 2019-05-24 | 2019-05-24 | |
| PCT/US2020/034328 WO2020242984A1 (en) | 2019-05-24 | 2020-05-22 | Modified viral particles and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112021023692A2 true BR112021023692A2 (pt) | 2022-01-04 |
Family
ID=71094822
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021023692A BR112021023692A2 (pt) | 2019-05-24 | 2020-05-22 | Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêutica |
Country Status (19)
| Country | Link |
|---|---|
| US (1) | US20220241430A1 (enExample) |
| EP (1) | EP3976631A1 (enExample) |
| JP (3) | JP2022533438A (enExample) |
| KR (1) | KR20220011664A (enExample) |
| CN (2) | CN114989267A (enExample) |
| AR (2) | AR118997A1 (enExample) |
| AU (1) | AU2020283537A1 (enExample) |
| BR (1) | BR112021023692A2 (enExample) |
| CA (1) | CA3140386A1 (enExample) |
| CL (2) | CL2021003096A1 (enExample) |
| CO (1) | CO2021017692A2 (enExample) |
| IL (1) | IL288233A (enExample) |
| MA (1) | MA56035A (enExample) |
| MX (1) | MX2021014338A (enExample) |
| PE (1) | PE20212357A1 (enExample) |
| PH (1) | PH12021552887A1 (enExample) |
| SG (1) | SG11202112917PA (enExample) |
| TW (1) | TW202110869A (enExample) |
| WO (1) | WO2020242984A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12421525B2 (en) | 2018-02-28 | 2025-09-23 | University of Pittsburgh—of the Commonwealth System of Higher Education | Conjugates comprising AAVs and CAS9 polypeptides |
| AR118997A1 (es) * | 2019-05-24 | 2021-11-17 | Regeneron Pharma | Partículas virales modificadas y usos de estas |
| 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 |
| JP2024525205A (ja) * | 2021-06-23 | 2024-07-10 | ダイノ セラピューティクス インコーポレイテッド | カプシドバリアント及びそれを使用する方法 |
| AU2023269134A1 (en) | 2022-05-09 | 2024-12-12 | 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 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1997005266A1 (en) | 1995-07-25 | 1997-02-13 | Introgene B.V. | Methods and means for targeted gene delivery |
| 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 |
| 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 |
| ATE317916T1 (de) * | 2001-11-13 | 2006-03-15 | Univ Pennsylvania | Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode |
| ES2602610T3 (es) | 2007-05-31 | 2017-02-21 | Medigene Ag | Proteína estructural mutada de un parvovirus |
| CN104131035A (zh) | 2009-12-10 | 2014-11-05 | 瑞泽恩制药公司 | 生产重链抗体的小鼠 |
| 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 |
| CN110885854A (zh) * | 2013-03-15 | 2020-03-17 | 北卡罗来纳-查佩尔山大学 | 双重聚糖结合aav载体的方法和组合物 |
| MX385339B (es) * | 2014-03-10 | 2025-03-18 | Uniqure Ip Bv | Vectores aav mejorados adicionales 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 |
| US10577627B2 (en) * | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| JP2020525020A (ja) * | 2017-06-27 | 2020-08-27 | 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． | ヒト細胞への遺伝物質の標的化導入のための指向性改変組換えウイルス粒子およびそれらの使用 |
| RS65569B1 (sr) | 2017-06-27 | 2024-06-28 | Regeneron Pharma | Rekombinantni virusni vektori modifikovani tropizmom i njihove upotrebe za ciljano uvođenje genetičkog materijala u humane ćelije |
| AR118997A1 (es) * | 2019-05-24 | 2021-11-17 | Regeneron Pharma | Partículas virales modificadas y usos de estas |
-
2020
- 2020-05-22 AR ARP200101465A patent/AR118997A1/es unknown
- 2020-05-22 CN CN202210622571.3A patent/CN114989267A/zh active Pending
- 2020-05-22 JP JP2021569402A patent/JP2022533438A/ja active Pending
- 2020-05-22 CN CN202080038612.3A patent/CN113874386A/zh active Pending
- 2020-05-22 KR KR1020217041074A patent/KR20220011664A/ko active Pending
- 2020-05-22 SG SG11202112917PA patent/SG11202112917PA/en unknown
- 2020-05-22 CA CA3140386A patent/CA3140386A1/en active Pending
- 2020-05-22 BR BR112021023692A patent/BR112021023692A2/pt unknown
- 2020-05-22 PE PE2021001934A patent/PE20212357A1/es unknown
- 2020-05-22 US US17/612,669 patent/US20220241430A1/en active Pending
- 2020-05-22 MA MA056035A patent/MA56035A/fr unknown
- 2020-05-22 TW TW109117155A patent/TW202110869A/zh unknown
- 2020-05-22 MX MX2021014338A patent/MX2021014338A/es unknown
- 2020-05-22 EP EP20733099.4A patent/EP3976631A1/en active Pending
- 2020-05-22 WO PCT/US2020/034328 patent/WO2020242984A1/en not_active Ceased
- 2020-05-22 AU AU2020283537A patent/AU2020283537A1/en active Pending
- 2020-05-22 PH PH1/2021/552887A patent/PH12021552887A1/en unknown
-
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
-
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 |
|---|---|
| MX2021014338A (es) | 2022-01-06 |
| AR124119A2 (es) | 2023-02-15 |
| EP3976631A1 (en) | 2022-04-06 |
| CL2021003096A1 (es) | 2022-09-20 |
| CO2021017692A2 (es) | 2022-01-17 |
| KR20220011664A (ko) | 2022-01-28 |
| MA56035A (fr) | 2022-04-06 |
| WO2020242984A9 (en) | 2021-01-14 |
| CA3140386A1 (en) | 2020-12-03 |
| CL2023000629A1 (es) | 2023-10-20 |
| AR118997A1 (es) | 2021-11-17 |
| JP2025119015A (ja) | 2025-08-13 |
| CN113874386A (zh) | 2021-12-31 |
| PH12021552887A1 (en) | 2022-09-05 |
| US20220241430A1 (en) | 2022-08-04 |
| CN114989267A (zh) | 2022-09-02 |
| IL288233A (en) | 2022-01-01 |
| JP2025032110A (ja) | 2025-03-11 |
| SG11202112917PA (en) | 2021-12-30 |
| TW202110869A (zh) | 2021-03-16 |
| PE20212357A1 (es) | 2021-12-17 |
| JP2022533438A (ja) | 2022-07-22 |
| AU2020283537A1 (en) | 2021-12-16 |
| WO2020242984A1 (en) | 2020-12-03 |
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