CO2024014703A2 - Ácido nucleico aislado que codifica una proteína de fusión basada en fviii-bdd y en un péptido señal heterólogo, y uso de la misma - Google Patents
Ácido nucleico aislado que codifica una proteína de fusión basada en fviii-bdd y en un péptido señal heterólogo, y uso de la mismaInfo
- Publication number
- CO2024014703A2 CO2024014703A2 CONC2024/0014703A CO2024014703A CO2024014703A2 CO 2024014703 A2 CO2024014703 A2 CO 2024014703A2 CO 2024014703 A CO2024014703 A CO 2024014703A CO 2024014703 A2 CO2024014703 A2 CO 2024014703A2
- Authority
- CO
- Colombia
- Prior art keywords
- bdd
- fviii
- fusion protein
- signal peptide
- heterologous signal
- Prior art date
Links
- 108010076504 Protein Sorting Signals Proteins 0.000 title abstract 3
- 108020001507 fusion proteins Proteins 0.000 title abstract 3
- 102000037865 fusion proteins Human genes 0.000 title abstract 3
- 108020004707 nucleic acids Proteins 0.000 title abstract 2
- 102000039446 nucleic acids Human genes 0.000 title abstract 2
- 150000007523 nucleic acids Chemical class 0.000 title abstract 2
- 102000001690 Factor VIII Human genes 0.000 abstract 1
- 108010054218 Factor VIII Proteins 0.000 abstract 1
- 229940105778 coagulation factor viii Drugs 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
- 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
- A61K38/17—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
- 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
- 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
- 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
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
-
- 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
- C07K14/76—Albumins
- C07K14/765—Serum albumin, e.g. HSA
-
- 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
- C12N15/62—DNA sequences coding for fusion proteins
- C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- 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
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
-
- 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
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Hematology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| RU2022111695A RU2818229C2 (ru) | 2022-04-28 | Выделенная нуклеиновая кислота, которая кодирует слитый белок на основе FVIII-BDD и гетерологичного сигнального пептида, и ее применение | |
| PCT/RU2023/050093 WO2023211315A1 (en) | 2022-04-28 | 2023-04-18 | Isolated nucleic acid that encodes fusion protein based on fviii-bdd and on heterologous signal peptide |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2024014703A2 true CO2024014703A2 (es) | 2024-12-30 |
Family
ID=88519415
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2024/0014703A CO2024014703A2 (es) | 2022-04-28 | 2024-10-28 | Ácido nucleico aislado que codifica una proteína de fusión basada en fviii-bdd y en un péptido señal heterólogo, y uso de la misma |
Country Status (19)
| Country | Link |
|---|---|
| EP (1) | EP4514975A1 (https=) |
| JP (1) | JP2025515356A (https=) |
| KR (1) | KR20250005425A (https=) |
| CN (1) | CN117402901A (https=) |
| AR (1) | AR129164A1 (https=) |
| AU (1) | AU2023261711A1 (https=) |
| CA (1) | CA3250663A1 (https=) |
| CL (1) | CL2024003295A1 (https=) |
| CO (1) | CO2024014703A2 (https=) |
| CR (1) | CR20240525A (https=) |
| IL (1) | IL316578A (https=) |
| JO (1) | JOP20240240A1 (https=) |
| MA (1) | MA68435A1 (https=) |
| MX (1) | MX2024013281A (https=) |
| PE (1) | PE20251064A1 (https=) |
| PY (1) | PY2331055A (https=) |
| TW (1) | TW202342751A (https=) |
| UY (1) | UY40243A (https=) |
| WO (1) | WO2023211315A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN120365434A (zh) * | 2024-01-23 | 2025-07-25 | 上海苹谱医疗科技有限公司 | 一种提高凝血因子ⅷ分泌水平的双信号肽及其应用 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL3326643T3 (pl) * | 2009-12-06 | 2021-10-25 | Bioverativ Therapeutics Inc. | Chimeryczne i hybrydowe polipeptydy czynnika VIII-FC oraz sposoby ich zastosowania |
| JP2016519110A (ja) * | 2013-04-22 | 2016-06-30 | キャタレント ファーマ ソリューションズ リミテッド ライアビリティ カンパニー | 獣医学的デコリン組成物及びそれらの使用 |
| RU2647769C2 (ru) * | 2015-12-30 | 2018-03-19 | Закрытое Акционерное Общество "Биокад" | Химерный улучшенный рекомбинантный фактор VIII |
| WO2017136358A1 (en) * | 2016-02-01 | 2017-08-10 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
| CN114651065B (zh) * | 2019-09-03 | 2024-08-20 | 五松尖端医疗产业振兴财团 | 导入用于提高蛋白质生产性的单个条形码系统的信号肽超高速筛选方法 |
| EP4051704A2 (en) * | 2019-11-01 | 2022-09-07 | Freeline Therapeutics Limited | Factor viii construct |
-
2023
- 2023-04-18 AU AU2023261711A patent/AU2023261711A1/en active Pending
- 2023-04-18 IL IL316578A patent/IL316578A/en unknown
- 2023-04-18 KR KR1020247039725A patent/KR20250005425A/ko active Pending
- 2023-04-18 JP JP2024563591A patent/JP2025515356A/ja active Pending
- 2023-04-18 EP EP23796915.9A patent/EP4514975A1/en active Pending
- 2023-04-18 CA CA3250663A patent/CA3250663A1/en active Pending
- 2023-04-18 MA MA68435A patent/MA68435A1/fr unknown
- 2023-04-18 CR CR20240525A patent/CR20240525A/es unknown
- 2023-04-18 PE PE2024002341A patent/PE20251064A1/es unknown
- 2023-04-18 WO PCT/RU2023/050093 patent/WO2023211315A1/en not_active Ceased
- 2023-04-19 TW TW112114536A patent/TW202342751A/zh unknown
- 2023-04-26 UY UY0001040243A patent/UY40243A/es unknown
- 2023-04-27 PY PY202302331055A patent/PY2331055A/es unknown
- 2023-04-27 AR ARP230101023A patent/AR129164A1/es unknown
- 2023-04-27 CN CN202310468986.4A patent/CN117402901A/zh active Pending
-
2024
- 2024-10-27 JO JOJO/P/2024/0240A patent/JOP20240240A1/ar unknown
- 2024-10-28 MX MX2024013281A patent/MX2024013281A/es unknown
- 2024-10-28 CL CL2024003295A patent/CL2024003295A1/es unknown
- 2024-10-28 CO CONC2024/0014703A patent/CO2024014703A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| PY2331055A (es) | 2023-12-05 |
| TW202342751A (zh) | 2023-11-01 |
| WO2023211315A1 (en) | 2023-11-02 |
| MA68435A1 (fr) | 2025-04-30 |
| KR20250005425A (ko) | 2025-01-09 |
| MX2024013281A (es) | 2024-12-06 |
| AR129164A1 (es) | 2024-07-24 |
| CL2024003295A1 (es) | 2025-02-21 |
| CN117402901A (zh) | 2024-01-16 |
| CA3250663A1 (en) | 2023-11-02 |
| IL316578A (en) | 2024-12-01 |
| PE20251064A1 (es) | 2025-04-09 |
| JOP20240240A1 (ar) | 2024-10-27 |
| AU2023261711A1 (en) | 2024-11-21 |
| UY40243A (es) | 2023-11-15 |
| JP2025515356A (ja) | 2025-05-14 |
| CR20240525A (es) | 2025-03-05 |
| EP4514975A1 (en) | 2025-03-05 |
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