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 heterólogo

Info

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
Application number
CL2024003295A
Other languages
English (en)
Inventor
Mikhailovich Gershovich Pavel
Valentinovich MOROZOV Dmitry
Pavlovna Perepelkina Mariya
Veniaminovna Vlasova Elena
Vladimirovna Fomina Anastasiia
Aleksandrovna Markova Vitaliia
Original Assignee
Biocad Joint Stock Co
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from RU2022111695A external-priority patent/RU2818229C2/ru
Application filed by Biocad Joint Stock Co filed Critical Biocad Joint Stock Co
Publication of CL2024003295A1 publication Critical patent/CL2024003295A1/es

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
    • A61K38/17—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)

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 que codifica una proteína de fusión basada en FVIII-BDD (factor VIII de coagulación de dominio B suprimido) y en un péptido señal heterólogo, a un casete de expresión y a un vector basado en el mismo, a una célula huésped para producir la proteína de fusión basada en FVIII-BDD y en un péptido señal heterólogo, y además a diversos usos del vector anterior.
CL2024003295A 2022-04-28 2024-10-28 Ácido nucleico aislado que codifica proteína de fusión basada en fviii-bdd y péptido señal heterólogo CL2024003295A1 (es)

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 (es)
JP (1) JP2025515356A (es)
KR (1) KR20250005425A (es)
CN (1) CN117402901A (es)
AR (1) AR129164A1 (es)
AU (1) AU2023261711A1 (es)
CA (1) CA3250663A1 (es)
CL (1) CL2024003295A1 (es)
CO (1) CO2024014703A2 (es)
CR (1) CR20240525A (es)
IL (1) IL316578A (es)
JO (1) JOP20240240A1 (es)
MA (1) MA68435A1 (es)
MX (1) MX2024013281A (es)
PE (1) PE20251064A1 (es)
PY (1) PY2331055A (es)
TW (1) TW202342751A (es)
UY (1) UY40243A (es)
WO (1) WO2023211315A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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
CO2024014703A2 (es) 2024-12-30
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

Similar Documents

Publication Publication Date Title
CO2021002965A2 (es) Moléculas de ácido nucleico y sus usos para la terapia génica no viral
CL2024003295A1 (es) Ácido nucleico aislado que codifica proteína de fusión basada en fviii-bdd y péptido señal heterólogo
CO2023009633A2 (es) Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal
MX2022009301A (es) Sistemas y metodos para microorganismos recombinantes de alto rendimiento y usos de los mismos.
MX2022006496A (es) Proteinas gpcr de opsinas quimericas.
CR20220619A (es) Ácido nucleico codón-optimizado que codifica la proteína smn1 y su uso
BR112023003526A2 (pt) Proteínas de fusão de coronavírus imunogênicos e métodos relacionados
CN109575116A (zh) 一种线粒体定位导肽及其发现方法与应用
ZA202501953B (en) Masking polypeptide, activatable novel prodrugs and methods of use thereof
JP2016519575A5 (es)
MX2024001878A (es) Sistemas de edicion de gen que comprenden una guia de arn dirigida a estatmina 2 (stmn2) y usos de los mismos.
CL2024003296A1 (es) Ácido nucleico codón optimizado que codifica proteína del factor viii coagulación de dominio b suprimido
MX2021009161A (es) Acidos nucleicos recombinantes que contienen secuencias promotoras de alfaherpesvirus.
AR129543A1 (es) Proteínas de fusión que comprenden formas truncadas de iga proteasa y sus usos
PY2215552A (es) Ácido nucleico optimizado por codones que codifica la proteína del factor de coagulación ix, y su uso
MX2023003819A (es) Metodo de produccion de una proteina recombinante en una celula hospedera que tiene desactivado el metabolismo de la ramnosa, asi como sus vectores de expresion, celulas hospederas y proteinas recombinantes.
TH1803002079C3 (th) กรรมวิธีการสร้างเซลล์ไลน์คงสภาพแมลงหวี่ที่มีระบบติดตามเรืองแสงและการติดฉลากสำหรับการแยกรีคอมบิแนนท์โปรตีนของฟลาวิไวรัสให้บริสุทธิ์
CO2024001768A2 (es) Método para obtener una cápside de virus adenoasociado modificada
CR20240561A (es) Ácido nucleico con actividad promotora y uso del mismo
ES2161130A1 (es) Dna recombinante derivado del virus de la sharka y vector de expresion de proteinas heterologas basado en dicho dna recombinante.