CR20230429A - Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX, y su uso - Google Patents
Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX, y su usoInfo
- Publication number
- CR20230429A CR20230429A CR20230429A CR20230429A CR20230429A CR 20230429 A CR20230429 A CR 20230429A CR 20230429 A CR20230429 A CR 20230429A CR 20230429 A CR20230429 A CR 20230429A CR 20230429 A CR20230429 A CR 20230429A
- Authority
- CR
- Costa Rica
- Prior art keywords
- nucleic acid
- codon
- optimized nucleic
- acid encoding
- fix
- Prior art date
Links
- 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
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 102000004169 proteins and genes Human genes 0.000 title abstract 2
- 108010076282 Factor IX Proteins 0.000 abstract 3
- 102000013831 Coagulation factor IX Human genes 0.000 abstract 2
- 229940105774 coagulation factor ix Drugs 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
Classifications
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- 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
- C12N15/67—General methods for enhancing the expression
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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)
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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
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
-
- 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
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- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
-
- 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)
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Mycology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
La presente solicitud está relacionada con los campos de la genética, la terapia génica y la biología molecular. Más específicamente, la presente invención se refiere a un ácido nucleico aislado optimizado en codones que codifica la proteína FIX (factor de coagulación IX), un casete de expresión y un vector basado en el mismo, así como un virus recombinante basado en el AAV5 (virus adenoasociado de serotipo 5) para aumentar la expresión del gen FIX en las células diana, y su uso.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
RU2021105703A RU2021105703A (ru) | 2021-03-05 | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови IX, и ее применение | |
PCT/RU2022/050073 WO2022186734A1 (en) | 2021-03-05 | 2022-03-05 | Codon-optimized nucleic acid encoding the fix protein |
Publications (1)
Publication Number | Publication Date |
---|---|
CR20230429A true CR20230429A (es) | 2024-03-08 |
Family
ID=83154378
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CR20230429A CR20230429A (es) | 2021-03-05 | 2022-03-05 | Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación IX, y su uso |
Country Status (19)
Country | Link |
---|---|
US (1) | US20240076691A1 (es) |
EP (1) | EP4288549A1 (es) |
JP (1) | JP2024509224A (es) |
KR (1) | KR20230154067A (es) |
CN (1) | CN117716038A (es) |
AR (1) | AR125041A1 (es) |
AU (1) | AU2022230548A1 (es) |
BR (1) | BR112023018003A2 (es) |
CA (1) | CA3212809A1 (es) |
CL (1) | CL2023002629A1 (es) |
CO (1) | CO2023011679A2 (es) |
CR (1) | CR20230429A (es) |
EC (1) | ECSP23067213A (es) |
IL (1) | IL305679A (es) |
MA (1) | MA62371A1 (es) |
MX (1) | MX2023010350A (es) |
TW (1) | TW202246505A (es) |
UY (1) | UY39659A (es) |
WO (1) | WO2022186734A1 (es) |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6458563B1 (en) * | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
US10842853B2 (en) * | 2017-05-22 | 2020-11-24 | Baxalta Incorporated | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B |
-
2022
- 2022-03-04 TW TW111108106A patent/TW202246505A/zh unknown
- 2022-03-05 KR KR1020237034078A patent/KR20230154067A/ko unknown
- 2022-03-05 EP EP22763678.4A patent/EP4288549A1/en active Pending
- 2022-03-05 AU AU2022230548A patent/AU2022230548A1/en active Pending
- 2022-03-05 US US18/280,338 patent/US20240076691A1/en active Pending
- 2022-03-05 CR CR20230429A patent/CR20230429A/es unknown
- 2022-03-05 JP JP2023554061A patent/JP2024509224A/ja active Pending
- 2022-03-05 MA MA62371A patent/MA62371A1/fr unknown
- 2022-03-05 CN CN202280017566.8A patent/CN117716038A/zh active Pending
- 2022-03-05 WO PCT/RU2022/050073 patent/WO2022186734A1/en active Application Filing
- 2022-03-05 BR BR112023018003A patent/BR112023018003A2/pt unknown
- 2022-03-05 MX MX2023010350A patent/MX2023010350A/es unknown
- 2022-03-05 IL IL305679A patent/IL305679A/en unknown
- 2022-03-05 CA CA3212809A patent/CA3212809A1/en active Pending
- 2022-03-07 AR ARP220100514A patent/AR125041A1/es unknown
- 2022-03-07 UY UY0001039659A patent/UY39659A/es unknown
-
2023
- 2023-09-04 CO CONC2023/0011679A patent/CO2023011679A2/es unknown
- 2023-09-04 CL CL2023002629A patent/CL2023002629A1/es unknown
- 2023-09-05 EC ECSENADI202367213A patent/ECSP23067213A/es unknown
Also Published As
Publication number | Publication date |
---|---|
IL305679A (en) | 2023-11-01 |
UY39659A (es) | 2022-09-30 |
KR20230154067A (ko) | 2023-11-07 |
CN117716038A (zh) | 2024-03-15 |
CL2023002629A1 (es) | 2024-04-01 |
AR125041A1 (es) | 2023-05-31 |
EP4288549A1 (en) | 2023-12-13 |
JP2024509224A (ja) | 2024-02-29 |
US20240076691A1 (en) | 2024-03-07 |
MA62371A1 (fr) | 2024-03-29 |
BR112023018003A2 (pt) | 2023-12-26 |
TW202246505A (zh) | 2022-12-01 |
CO2023011679A2 (es) | 2024-01-15 |
MX2023010350A (es) | 2023-09-22 |
ECSP23067213A (es) | 2023-10-31 |
WO2022186734A1 (en) | 2022-09-09 |
CA3212809A1 (en) | 2022-09-09 |
AU2022230548A1 (en) | 2023-09-28 |
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