AR119869A1 - Proteína vp1 modificada aislada de la cápside del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en la misma - Google Patents
Proteína vp1 modificada aislada de la cápside del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en la mismaInfo
- Publication number
- AR119869A1 AR119869A1 ARP200102440A ARP200102440A AR119869A1 AR 119869 A1 AR119869 A1 AR 119869A1 AR P200102440 A ARP200102440 A AR P200102440A AR P200102440 A ARP200102440 A AR P200102440A AR 119869 A1 AR119869 A1 AR 119869A1
- Authority
- AR
- Argentina
- Prior art keywords
- capsid
- aav5
- protein
- adeno
- associated virus
- Prior art date
Links
Classifications
-
- 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
- C40—COMBINATORIAL TECHNOLOGY
- C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
- C40B40/00—Libraries per se, e.g. arrays, mixtures
- C40B40/04—Libraries containing only organic compounds
- C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
- C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
- A61K38/482—Serine endopeptidases (3.4.21)
- A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- 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
-
- 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
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
-
- 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
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)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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
Esta solicitud se relaciona con los campos de la terapia génica y la biología molecular. De manera más específica, la presente se refiere a una proteína VP1 alterada aislada de la cápside del virus adeno-asociado de serotipo (AAV5), que comprende una o más sustituciones de aminoácidos en comparación con la proteína VP1 de la cápside de AAV5 de tipo salvaje, que aumentan la eficiencia de transducción, así como también se relaciona con una cápside y un vector basado en la misma.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
RU2019126509A RU2751592C2 (ru) | 2019-08-22 | 2019-08-22 | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе |
Publications (1)
Publication Number | Publication Date |
---|---|
AR119869A1 true AR119869A1 (es) | 2022-01-19 |
Family
ID=74660332
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP200102440A AR119869A1 (es) | 2019-08-22 | 2020-08-31 | Proteína vp1 modificada aislada de la cápside del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en la misma |
Country Status (19)
Country | Link |
---|---|
US (1) | US20220306696A1 (es) |
EP (1) | EP4019642A4 (es) |
JP (1) | JP2022545121A (es) |
KR (1) | KR20220158674A (es) |
CN (1) | CN114981444A (es) |
AR (1) | AR119869A1 (es) |
AU (1) | AU2020331882A1 (es) |
BR (1) | BR112022003389A2 (es) |
CA (1) | CA3148964A1 (es) |
CO (1) | CO2022001833A2 (es) |
EC (1) | ECSP22021725A (es) |
IL (1) | IL290802A (es) |
JO (1) | JOP20220043A1 (es) |
MA (1) | MA56142B1 (es) |
MX (1) | MX2022002184A (es) |
PE (1) | PE20221254A1 (es) |
RU (1) | RU2751592C2 (es) |
TW (1) | TW202120690A (es) |
WO (1) | WO2021034222A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN113121654B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体 |
AR126839A1 (es) * | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta |
AR126840A1 (es) | 2021-08-20 | 2023-11-22 | Llc «Anabion» | Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta |
Family Cites Families (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DE19827457C1 (de) * | 1998-06-19 | 2000-03-02 | Medigene Ag | Strukturprotein von AAV, seine Herstellung und Verwendung |
EP2359869B1 (en) * | 2001-12-17 | 2018-12-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor |
KR20220056884A (ko) | 2011-04-22 | 2022-05-06 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법 |
RU2683497C2 (ru) | 2012-04-18 | 2019-03-28 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Композиция и способы высокоэффективного переноса генов с помощью вариантов капсида aav |
SG10201809075XA (en) * | 2013-07-22 | 2018-11-29 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
US10081659B2 (en) * | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
CA2996420A1 (en) * | 2015-09-28 | 2017-04-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
WO2017201121A1 (en) * | 2016-05-17 | 2017-11-23 | University Of Florida Research Foundation, Inc. | Recombinant aav for gene therapy in lungs |
-
2019
- 2019-08-22 RU RU2019126509A patent/RU2751592C2/ru active
-
2020
- 2020-08-21 KR KR1020227009552A patent/KR20220158674A/ko active Search and Examination
- 2020-08-21 EP EP20855760.3A patent/EP4019642A4/en active Pending
- 2020-08-21 MA MA56142A patent/MA56142B1/fr unknown
- 2020-08-21 AU AU2020331882A patent/AU2020331882A1/en active Pending
- 2020-08-21 TW TW109128700A patent/TW202120690A/zh unknown
- 2020-08-21 JP JP2022512768A patent/JP2022545121A/ja active Pending
- 2020-08-21 WO PCT/RU2020/000445 patent/WO2021034222A1/ru unknown
- 2020-08-21 US US17/637,099 patent/US20220306696A1/en active Pending
- 2020-08-21 PE PE2022000294A patent/PE20221254A1/es unknown
- 2020-08-21 CN CN202080074154.9A patent/CN114981444A/zh active Pending
- 2020-08-21 JO JOP/2022/0043A patent/JOP20220043A1/ar unknown
- 2020-08-21 CA CA3148964A patent/CA3148964A1/en active Pending
- 2020-08-21 BR BR112022003389A patent/BR112022003389A2/pt unknown
- 2020-08-21 MX MX2022002184A patent/MX2022002184A/es unknown
- 2020-08-31 AR ARP200102440A patent/AR119869A1/es unknown
-
2022
- 2022-02-21 CO CONC2022/0001833A patent/CO2022001833A2/es unknown
- 2022-02-22 IL IL290802A patent/IL290802A/en unknown
- 2022-03-21 EC ECSENADI202221725A patent/ECSP22021725A/es unknown
Also Published As
Publication number | Publication date |
---|---|
IL290802A (en) | 2022-04-01 |
AU2020331882A1 (en) | 2022-11-03 |
CA3148964A1 (en) | 2021-02-25 |
CN114981444A (zh) | 2022-08-30 |
MA56142B1 (fr) | 2023-09-27 |
WO2021034222A1 (ru) | 2021-02-25 |
MX2022002184A (es) | 2022-05-24 |
EP4019642A4 (en) | 2023-09-06 |
BR112022003389A2 (pt) | 2022-05-17 |
MA56142A1 (fr) | 2023-05-31 |
JOP20220043A1 (ar) | 2023-01-30 |
RU2751592C2 (ru) | 2021-07-15 |
RU2019126509A (ru) | 2021-06-04 |
PE20221254A1 (es) | 2022-08-16 |
TW202120690A (zh) | 2021-06-01 |
KR20220158674A (ko) | 2022-12-01 |
JP2022545121A (ja) | 2022-10-25 |
EP4019642A1 (en) | 2022-06-29 |
CO2022001833A2 (es) | 2022-08-09 |
US20220306696A1 (en) | 2022-09-29 |
ECSP22021725A (es) | 2022-08-31 |
RU2019126509A3 (es) | 2021-06-04 |
WO2021034222A9 (ru) | 2021-04-22 |
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