CO2024001457A2 - Proteína de la cápside vp1 modificada aislada aav9 - Google Patents
Proteína de la cápside vp1 modificada aislada aav9Info
- Publication number
- CO2024001457A2 CO2024001457A2 CONC2024/0001457A CO2024001457A CO2024001457A2 CO 2024001457 A2 CO2024001457 A2 CO 2024001457A2 CO 2024001457 A CO2024001457 A CO 2024001457A CO 2024001457 A2 CO2024001457 A2 CO 2024001457A2
- Authority
- CO
- Colombia
- Prior art keywords
- aav9
- capsid protein
- isolated modified
- associated virus
- vector based
- Prior art date
Links
- 108090000565 Capsid Proteins Proteins 0.000 title abstract 3
- 102100023321 Ceruloplasmin Human genes 0.000 title abstract 3
- 241000702421 Dependoparvovirus Species 0.000 abstract 2
- 238000006467 substitution reaction Methods 0.000 abstract 2
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
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
-
- 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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
- C07K14/01—DNA viruses
- C07K14/075—Adenoviridae
-
- 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
-
- 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
- C12N15/861—Adenoviral 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- 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
- C12N2750/14141—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
- C12N2750/14151—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)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente solicitud está relacionada con los campos de terapia génica y biología molecular. Más específicamente la presente invención se refiere a una proteína de la cápside VP1 modificada aislada del virus adenoasociado de serotipo 9 (AAV9) que comprende una o más sustituciones de aminoácidos en comparación con la proteína de la cápside VP1 de AAV9 de tipo salvaje, tales sustituciones aumentan la eficiencia de la producción (ensamblaje) del vector basado en el virus adenoasociado recombinante de serotipo 9 (rAAV9), a una cápside y a un vector basado en la VP1 anterior, así como a los usos de la misma.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
RU2021124726A RU2021124726A (ru) | 2021-08-20 | Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе | |
PCT/RU2022/050258 WO2023022634A1 (ru) | 2021-08-20 | 2022-08-21 | Выделенный модифицированный белок vp1 капсида aav9 |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2024001457A2 true CO2024001457A2 (es) | 2024-06-17 |
Family
ID=85240922
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2024/0001457A CO2024001457A2 (es) | 2021-08-20 | 2024-02-12 | Proteína de la cápside vp1 modificada aislada aav9 |
Country Status (11)
Country | Link |
---|---|
EP (1) | EP4389760A1 (es) |
CN (1) | CN117836312A (es) |
AR (1) | AR126839A1 (es) |
AU (1) | AU2022329857A1 (es) |
CA (1) | CA3229573A1 (es) |
CO (1) | CO2024001457A2 (es) |
EC (1) | ECSP24013002A (es) |
IL (1) | IL310947A (es) |
MA (1) | MA64618A1 (es) |
TW (1) | TW202317769A (es) |
WO (1) | WO2023022634A1 (es) |
Family Cites Families (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DE3431140A1 (de) | 1984-08-24 | 1986-03-06 | Behringwerke Ag, 3550 Marburg | Enhancer fuer eukaryotische expressionssysteme |
GB8717430D0 (en) | 1987-07-23 | 1987-08-26 | Celltech Ltd | Recombinant dna product |
DE19827457C1 (de) * | 1998-06-19 | 2000-03-02 | Medigene Ag | Strukturprotein von AAV, seine Herstellung und Verwendung |
EP1453547B1 (en) * | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
EP3693025B1 (en) | 2011-04-22 | 2021-10-13 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
MY172457A (en) | 2012-04-18 | 2019-11-26 | Childrens Hospital Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
CA3209883A1 (en) * | 2013-07-22 | 2015-01-29 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
RU2751592C2 (ru) * | 2019-08-22 | 2021-07-15 | Общество С Ограниченной Ответственностью "Анабион" | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе |
-
2022
- 2022-08-19 AR ARP220102250A patent/AR126839A1/es unknown
- 2022-08-21 EP EP22858844.8A patent/EP4389760A1/en active Pending
- 2022-08-21 WO PCT/RU2022/050258 patent/WO2023022634A1/ru active Application Filing
- 2022-08-21 IL IL310947A patent/IL310947A/en unknown
- 2022-08-21 AU AU2022329857A patent/AU2022329857A1/en active Pending
- 2022-08-21 CN CN202280055177.4A patent/CN117836312A/zh active Pending
- 2022-08-21 MA MA64618A patent/MA64618A1/fr unknown
- 2022-08-21 CA CA3229573A patent/CA3229573A1/en active Pending
- 2022-08-22 TW TW111131553A patent/TW202317769A/zh unknown
-
2024
- 2024-02-12 CO CONC2024/0001457A patent/CO2024001457A2/es unknown
- 2024-02-19 EC ECSENADI202413002A patent/ECSP24013002A/es unknown
Also Published As
Publication number | Publication date |
---|---|
CN117836312A (zh) | 2024-04-05 |
EP4389760A1 (en) | 2024-06-26 |
IL310947A (en) | 2024-04-01 |
TW202317769A (zh) | 2023-05-01 |
ECSP24013002A (es) | 2024-03-01 |
AU2022329857A1 (en) | 2024-04-04 |
WO2023022634A1 (ru) | 2023-02-23 |
MA64618A1 (fr) | 2024-04-30 |
CA3229573A1 (en) | 2023-02-23 |
AR126839A1 (es) | 2023-11-22 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
CO2020004738A2 (es) | Variantes de cápsides de virus adenoasociados y métodos de uso de estas | |
CL2018003196A1 (es) | Cápsides variantes de virus adenoasociados y métodos de uso de estas. | |
PE20190401A1 (es) | Novedosas proteinas de la capside del virus adenoasociado | |
ECSP22021725A (es) | Prote?na VP1 modificada aislada de la c?pside del virus adeno-asociado de serotipo 5 (AAV5), c?pside y vector basado en la misma. | |
PE20210915A1 (es) | Capsides variantes de virus adenoasociados y su uso para inhibir la angiogenesis | |
RU2017142006A (ru) | Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов | |
CO2021016198A2 (es) | Composiciones útiles para el tratamiento de la enfermedad de pompe | |
PE20220930A1 (es) | Composiciones utiles en el tratamiento de leucodistrofia metacromatica | |
CO2022017334A2 (es) | Ácido nucleico codón-optimizado que codifica la proteína smn1 y su uso | |
CO2023009633A2 (es) | Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal | |
ECSP24013002A (es) | Proteína vp1 de la cápsida aav9 modificada aislada | |
AR123369A1 (es) | VACUNA BASADA EN AAV5 PARA LA INDUCCIÓN DE INMUNIDAD ESPECÍFICA CONTRA EL SARS-CoV-2 Y/O LA PREVENCIÓN DE LA INFECCIÓN POR CORONAVIRUS RELACIONADA CON EL SARS-CoV-2 | |
ECSP24013075A (es) | Proteina de cápside de aav5 modificada aislada vp1 | |
ECSP23067213A (es) | Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX y su uso | |
CO2024001455A2 (es) | Sistema de expresión de baculovirus | |
AR122251A1 (es) | Composiciones útiles para el tratamiento de la enfermedad de krabbe | |
CO2024001768A2 (es) | Método para obtener una cápside de virus adenoasociado modificada | |
AR123245A1 (es) | Nuevas cápsulas de aav y composiciones que las contienen | |
EA202192973A1 (ru) | Композиции, полезные для лечения болезни помпе |