CL2024003295A1 - Ácido nucleico aislado que codifica proteína de fusión basada en fviii-bdd y péptido señal heterólogo - Google Patents
Ácido nucleico aislado que codifica proteína de fusión basada en fviii-bdd y péptido señal heterólogoInfo
- Publication number
- CL2024003295A1 CL2024003295A1 CL2024003295A CL2024003295A CL2024003295A1 CL 2024003295 A1 CL2024003295 A1 CL 2024003295A1 CL 2024003295 A CL2024003295 A CL 2024003295A CL 2024003295 A CL2024003295 A CL 2024003295A CL 2024003295 A1 CL2024003295 A1 CL 2024003295A1
- Authority
- CL
- Chile
- Prior art keywords
- bdd
- fusion protein
- signal peptide
- heterologous signal
- nucleic acid
- 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)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (1)
| 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 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2024003295A1 true CL2024003295A1 (es) | 2025-02-21 |
Family
ID=88519415
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2024003295A CL2024003295A1 (es) | 2022-04-28 | 2024-10-28 | Ácido nucleico aislado que codifica proteína de fusión basada en fviii-bdd y péptido señal heterólogo |
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 |
|---|---|---|---|---|
| SI2506868T1 (en) * | 2009-12-06 | 2018-04-30 | Bioverativ Therapeutics Inc. | HYPER AND HYBRID POLYPETTIDES FACTOR VIII-FC AND METHODS FOR THEIR USE |
| EP2989208A4 (en) * | 2013-04-22 | 2016-12-07 | Catalent Pharma Solutions Llc | DECORINAL COMPOSITIONS FOR VETERINARY MEDICINE AND USE THEREOF |
| RU2647769C2 (ru) * | 2015-12-30 | 2018-03-19 | Закрытое Акционерное Общество "Биокад" | Химерный улучшенный рекомбинантный фактор VIII |
| CA3012695A1 (en) * | 2016-02-01 | 2017-08-10 | Bioverativ Therapeutics Inc. | Optimized factor viii genes |
| US20220334124A1 (en) * | 2019-09-03 | 2022-10-20 | Sung Sub Kim | Method for ultra-rapidly selecting signal peptide to which individual barcode system for increasing protein productivity is introduced |
| CN114929735A (zh) * | 2019-11-01 | 2022-08-19 | 自由行疗法有限公司 | 因子viii构建体 |
-
2023
- 2023-04-18 AU AU2023261711A patent/AU2023261711A1/en active Pending
- 2023-04-18 MA MA68435A patent/MA68435A1/fr unknown
- 2023-04-18 IL IL316578A patent/IL316578A/en unknown
- 2023-04-18 CA CA3250663A patent/CA3250663A1/en active Pending
- 2023-04-18 CR CR20240525A patent/CR20240525A/es unknown
- 2023-04-18 PE PE2024002341A patent/PE20251064A1/es unknown
- 2023-04-18 JP JP2024563591A patent/JP2025515356A/ja active Pending
- 2023-04-18 KR KR1020247039725A patent/KR20250005425A/ko active Pending
- 2023-04-18 WO PCT/RU2023/050093 patent/WO2023211315A1/en not_active Ceased
- 2023-04-18 EP EP23796915.9A patent/EP4514975A1/en active Pending
- 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 CN CN202310468986.4A patent/CN117402901A/zh active Pending
- 2023-04-27 AR ARP230101023A patent/AR129164A1/es unknown
-
2024
- 2024-10-27 JO JOJO/P/2024/0240A patent/JOP20240240A1/ar unknown
- 2024-10-28 CO CONC2024/0014703A patent/CO2024014703A2/es unknown
- 2024-10-28 CL CL2024003295A patent/CL2024003295A1/es unknown
- 2024-10-28 MX MX2024013281A patent/MX2024013281A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CO2024014703A2 (es) | 2024-12-30 |
| AU2023261711A1 (en) | 2024-11-21 |
| PE20251064A1 (es) | 2025-04-09 |
| EP4514975A1 (en) | 2025-03-05 |
| CN117402901A (zh) | 2024-01-16 |
| AR129164A1 (es) | 2024-07-24 |
| CA3250663A1 (en) | 2023-11-02 |
| TW202342751A (zh) | 2023-11-01 |
| WO2023211315A1 (en) | 2023-11-02 |
| CR20240525A (es) | 2025-03-05 |
| UY40243A (es) | 2023-11-15 |
| JP2025515356A (ja) | 2025-05-14 |
| MX2024013281A (es) | 2024-12-06 |
| JOP20240240A1 (ar) | 2024-10-27 |
| IL316578A (en) | 2024-12-01 |
| PY2331055A (es) | 2023-12-05 |
| MA68435A1 (fr) | 2025-04-30 |
| KR20250005425A (ko) | 2025-01-09 |
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