IL280973A - Factor ix encoding nucleotides - Google Patents
Factor ix encoding nucleotidesInfo
- Publication number
- IL280973A IL280973A IL280973A IL28097321A IL280973A IL 280973 A IL280973 A IL 280973A IL 280973 A IL280973 A IL 280973A IL 28097321 A IL28097321 A IL 28097321A IL 280973 A IL280973 A IL 280973A
- Authority
- IL
- Israel
- Prior art keywords
- factor
- encoding nucleotides
- nucleotides
- encoding
- Prior art date
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- A61K48/005—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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/52—Genes encoding for enzymes or proenzymes
-
- 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
-
- 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
- C12N2750/14141—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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination
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)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Hematology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US16/105,583 US10842885B2 (en) | 2018-08-20 | 2018-08-20 | Factor IX encoding nucleotides |
GBGB1813528.5A GB201813528D0 (en) | 2018-08-20 | 2018-08-20 | Factor IX encoding nucleotides |
PCT/GB2019/052339 WO2020039183A1 (en) | 2018-08-20 | 2019-08-20 | Factor ix encoding nucleotides |
Publications (1)
Publication Number | Publication Date |
---|---|
IL280973A true IL280973A (en) | 2021-04-29 |
Family
ID=67810978
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
IL280973A IL280973A (en) | 2018-08-20 | 2021-02-18 | Factor ix encoding nucleotides |
Country Status (11)
Country | Link |
---|---|
EP (1) | EP3810770A1 (en) |
JP (1) | JP2021533799A (en) |
KR (1) | KR20210049129A (en) |
CN (1) | CN112739818A (en) |
AU (1) | AU2019323747A1 (en) |
BR (1) | BR112021002998A2 (en) |
CA (1) | CA3109579A1 (en) |
IL (1) | IL280973A (en) |
MX (1) | MX2021001901A (en) |
SG (1) | SG11202101560QA (en) |
WO (1) | WO2020039183A1 (en) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
CA3225312A1 (en) * | 2021-06-23 | 2022-12-29 | Inspirar Limited | Composition and method for treating hemophilia b |
CA3232470A1 (en) * | 2021-10-27 | 2023-05-04 | Leah SABIN | Compositions and methods for expressing factor ix for hemophilia b therapy |
Family Cites Families (10)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DK1804839T3 (en) * | 2004-09-22 | 2012-04-10 | St Jude Childrens Res Hospital | IMPROVED EXPRESSION OF FACTOR IX IN GENTHERAPY VECTORS |
TR201813067T4 (en) * | 2008-09-15 | 2018-09-21 | Uniqure Biopharma B V | The factor IX polypeptide mutant, its uses and a method for producing it. |
WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
RU2014144881A (en) * | 2012-04-17 | 2016-06-10 | Ф. Хоффманн-Ля Рош Аг | METHOD FOR EXPRESSION OF POLYEPEPTIDES USING MODIFIED NUCLEIC ACIDS |
CN103667346B (en) * | 2013-12-17 | 2016-01-20 | 扬州大学 | A kind of pRBE-HCR-hAAT-hFIXml plasmid and structure thereof and application |
EP3160478A4 (en) * | 2014-06-30 | 2018-05-16 | Bioverativ Therapeutics Inc. | Optimized factor ix gene |
GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
GB201508025D0 (en) * | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
JP6831779B2 (en) * | 2015-06-23 | 2021-02-17 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | Modified Factor IX and compositions, methods and uses for gene transfer into cells, organs and tissues |
CN116732066A (en) * | 2016-10-14 | 2023-09-12 | 四川至善唯新生物科技有限公司 | Preparation and application of high-activity blood coagulation factor IX mutant, recombinant protein and fusion protein |
-
2019
- 2019-08-20 KR KR1020217008345A patent/KR20210049129A/en not_active Application Discontinuation
- 2019-08-20 CA CA3109579A patent/CA3109579A1/en active Pending
- 2019-08-20 CN CN201980062207.2A patent/CN112739818A/en active Pending
- 2019-08-20 WO PCT/GB2019/052339 patent/WO2020039183A1/en unknown
- 2019-08-20 AU AU2019323747A patent/AU2019323747A1/en not_active Abandoned
- 2019-08-20 EP EP19762195.6A patent/EP3810770A1/en active Pending
- 2019-08-20 SG SG11202101560QA patent/SG11202101560QA/en unknown
- 2019-08-20 JP JP2021509867A patent/JP2021533799A/en active Pending
- 2019-08-20 MX MX2021001901A patent/MX2021001901A/en unknown
- 2019-08-20 BR BR112021002998-2A patent/BR112021002998A2/en unknown
-
2021
- 2021-02-18 IL IL280973A patent/IL280973A/en unknown
Also Published As
Publication number | Publication date |
---|---|
JP2021533799A (en) | 2021-12-09 |
BR112021002998A2 (en) | 2021-05-11 |
CN112739818A (en) | 2021-04-30 |
KR20210049129A (en) | 2021-05-04 |
SG11202101560QA (en) | 2021-03-30 |
MX2021001901A (en) | 2021-05-27 |
AU2019323747A1 (en) | 2021-03-04 |
WO2020039183A1 (en) | 2020-02-27 |
CA3109579A1 (en) | 2020-02-27 |
EP3810770A1 (en) | 2021-04-28 |
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