JOP20240241A1 - حمض نووي مُحسَّن بالكودون يشفر بروتين fviii-bdd - Google Patents
حمض نووي مُحسَّن بالكودون يشفر بروتين fviii-bddInfo
- Publication number
- JOP20240241A1 JOP20240241A1 JOJO/P/2024/0241A JOP20240241A JOP20240241A1 JO P20240241 A1 JOP20240241 A1 JO P20240241A1 JO P20240241 A JOP20240241 A JO P20240241A JO P20240241 A1 JOP20240241 A1 JO P20240241A1
- Authority
- JO
- Jordan
- Prior art keywords
- codon
- dna encoding
- enhanced dna
- bdd
- fviii
- 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
- 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
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
- 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
-
- 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)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Hematology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| RU2022111734A RU2808564C2 (ru) | 2022-04-28 | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови VIII c делетированным B доменом, и ее применение | |
| PCT/RU2023/050094 WO2023211316A1 (en) | 2022-04-28 | 2023-04-18 | Codon-optimized nucleic acid encoding the fviii-bdd |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| JOP20240241A1 true JOP20240241A1 (ar) | 2024-10-27 |
Family
ID=88482201
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JOJO/P/2024/0241A JOP20240241A1 (ar) | 2022-04-28 | 2024-10-27 | حمض نووي مُحسَّن بالكودون يشفر بروتين fviii-bdd |
Country Status (20)
| Country | Link |
|---|---|
| US (1) | US20250282847A1 (https=) |
| EP (1) | EP4514836A1 (https=) |
| JP (1) | JP2025514313A (https=) |
| KR (1) | KR20250007615A (https=) |
| CN (1) | CN116970629A (https=) |
| AR (1) | AR129163A1 (https=) |
| AU (1) | AU2023261338A1 (https=) |
| CA (1) | CA3250665A1 (https=) |
| CL (1) | CL2024003296A1 (https=) |
| CO (1) | CO2024014690A2 (https=) |
| CR (1) | CR20240524A (https=) |
| IL (1) | IL316584A (https=) |
| JO (1) | JOP20240241A1 (https=) |
| MA (1) | MA68586A1 (https=) |
| MX (1) | MX2024013280A (https=) |
| PE (1) | PE20251838A1 (https=) |
| PY (1) | PY2331315A (https=) |
| TW (1) | TW202346601A (https=) |
| UY (1) | UY40241A (https=) |
| WO (1) | WO2023211316A1 (https=) |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
| EA202190827A1 (ru) * | 2015-11-13 | 2021-11-30 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| BR112021003399A2 (pt) * | 2018-08-24 | 2021-05-18 | Spark Therapeutics, Inc. | sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso |
-
2023
- 2023-04-18 KR KR1020247039728A patent/KR20250007615A/ko active Pending
- 2023-04-18 EP EP23796916.7A patent/EP4514836A1/en active Pending
- 2023-04-18 AU AU2023261338A patent/AU2023261338A1/en active Pending
- 2023-04-18 CA CA3250665A patent/CA3250665A1/en active Pending
- 2023-04-18 US US18/859,760 patent/US20250282847A1/en active Pending
- 2023-04-18 IL IL316584A patent/IL316584A/en unknown
- 2023-04-18 CR CR20240524A patent/CR20240524A/es unknown
- 2023-04-18 MA MA68586A patent/MA68586A1/fr unknown
- 2023-04-18 WO PCT/RU2023/050094 patent/WO2023211316A1/en not_active Ceased
- 2023-04-18 PE PE2024002342A patent/PE20251838A1/es unknown
- 2023-04-18 JP JP2024563594A patent/JP2025514313A/ja active Pending
- 2023-04-19 TW TW112114534A patent/TW202346601A/zh unknown
- 2023-04-26 UY UY0001040241A patent/UY40241A/es unknown
- 2023-04-27 CN CN202310468975.6A patent/CN116970629A/zh active Pending
- 2023-04-27 AR ARP230101022A patent/AR129163A1/es unknown
- 2023-04-27 PY PY202302331315A patent/PY2331315A/es unknown
-
2024
- 2024-10-27 JO JOJO/P/2024/0241A patent/JOP20240241A1/ar unknown
- 2024-10-28 CL CL2024003296A patent/CL2024003296A1/es unknown
- 2024-10-28 MX MX2024013280A patent/MX2024013280A/es unknown
- 2024-10-28 CO CONC2024/0014690A patent/CO2024014690A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CR20240524A (es) | 2025-05-05 |
| KR20250007615A (ko) | 2025-01-14 |
| CA3250665A1 (en) | 2023-11-02 |
| IL316584A (en) | 2024-12-01 |
| PY2331315A (es) | 2023-10-31 |
| CL2024003296A1 (es) | 2025-02-21 |
| AU2023261338A1 (en) | 2024-11-21 |
| TW202346601A (zh) | 2023-12-01 |
| PE20251838A1 (es) | 2025-07-17 |
| MX2024013280A (es) | 2024-12-06 |
| CO2024014690A2 (es) | 2025-03-06 |
| UY40241A (es) | 2023-10-13 |
| CN116970629A (zh) | 2023-10-31 |
| JP2025514313A (ja) | 2025-05-02 |
| MA68586A1 (fr) | 2025-04-30 |
| AR129163A1 (es) | 2024-07-24 |
| WO2023211316A1 (en) | 2023-11-02 |
| US20250282847A1 (en) | 2025-09-11 |
| EP4514836A1 (en) | 2025-03-05 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CL2021002739A1 (es) | Proteína crispr de prevotella y francisella 1 (cpf1) aislada de acidaminococcus sp. bv3l6; proteína de fusión que la comprende; ácido nucleico; vector; métodos para alterar el genoma de una célula o una molécula de adn bicatenario in vitro que utilizan dichas proteínas (divisional de sol. 201903004) | |
| PH12017502281A1 (en) | Thermostable cas9 nucleases | |
| EA202090034A1 (ru) | Способы модификации сплайсинга рнк | |
| JOP20230168A1 (ar) | التأثير التآزري لـ SMN1 و miR-23a في علاج ضمور العضلات النخاعي | |
| ATE455855T1 (de) | Gene von corynebacterium glutamicum kodierend für stoffwechselproteine | |
| CY1120789T1 (el) | Προϊοντα γονιδιων εκφραζομενα διαφορικα μεσα σε ογκους και εφαρμογη αυτων | |
| SG10201903381TA (en) | Artificial nucleic acid molecules | |
| AU2002327412A1 (en) | Methods and compositions relating to improved lentiviral vector production systems | |
| DE60045191D1 (de) | Corynebacterium glutamicum gene, die für stoffwechselproteine kodieren | |
| WO2022046935A3 (en) | Methods and compositions for treating glioblastoma | |
| KR100469936B1 (ko) | 사스 바이러스 항원의 세포표면 발현벡터 및 상기 벡터에의해 형질전환된 미생물 | |
| JOP20220327A1 (ar) | حمض نووي معزز بالكودون يشفَر بروتين smn1 | |
| JOP20240240A1 (ar) | حمض نووي معزول يشفر بروتين اندماج قائم على fviii-bdd وعلى ببتيد إشارة غير متجانس | |
| ATE532861T1 (de) | Expressionsvektor | |
| MX2022015802A (es) | Construcciones de anellovirus en tandem. | |
| JOP20240241A1 (ar) | حمض نووي مُحسَّن بالكودون يشفر بروتين fviii-bdd | |
| DK2066790T3 (da) | Galactosyltransferase | |
| EA202190676A1 (ru) | НУКЛЕАЗА PaCas9 | |
| WO2021231549A3 (en) | Engineered gene-editing proteins | |
| GEAP202416228A (en) | Method of producing a recombinant protein in a host cell which has a disabled rhamnose metabolism as well as expression vectors, host cells and recombinant proteins thereof | |
| JOP20230208A1 (ar) | حمض نووي مُحسّن بالكودون يشفر بروتين fix | |
| JOP20240270A1 (ar) | حمض نووي له نشاط مُروِّج واستخداماته | |
| WO2020188228A8 (en) | Methods of optimising expression and delivery of mitochondrial proteins | |
| WO2020132789A8 (zh) | 突变的人2Ig-B7-H3蛋白编码基因、重组载体、包含其的宿主细胞、药物组合物及其应用 | |
| KR20040053279A (ko) | 스트레스 저항성 및 내성 단백질을 코딩하는 유전자 |