MX2018009375A - Genes del factor viii optimizados. - Google Patents

Genes del factor viii optimizados.

Info

Publication number
MX2018009375A
MX2018009375A MX2018009375A MX2018009375A MX2018009375A MX 2018009375 A MX2018009375 A MX 2018009375A MX 2018009375 A MX2018009375 A MX 2018009375A MX 2018009375 A MX2018009375 A MX 2018009375A MX 2018009375 A MX2018009375 A MX 2018009375A
Authority
MX
Mexico
Prior art keywords
factor viii
optimized factor
codon optimized
present disclosure
sequences
Prior art date
Application number
MX2018009375A
Other languages
English (en)
Inventor
Liu Tongyao
Tan Siyuan
Original Assignee
Bioverativ Therapeutics Inc
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
Application filed by Bioverativ Therapeutics Inc filed Critical Bioverativ Therapeutics Inc
Publication of MX2018009375A publication Critical patent/MX2018009375A/es

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Classifications

    • 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)
    • 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
    • A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • C12N15/63—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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La presente descripción proporciona secuencias del factor FVIII optimizadas con codones, vectores y células hospedadoras que comprenden secuencias del factor FVIII optimizadas con codones, polipéptidos codificados mediante secuencias del factor FVIII optimizadas con codones, y métodos para producir los polipéptidos. La presente descripción también proporciona métodos para tratar los trastornos hemorrágicos, por ejemplo, la hemofilia, que comprende administrar al sujeto una secuencia de ácido nucleico del factor VIII optimizado con codones o el polipéptido codificado por esta.
MX2018009375A 2016-02-01 2017-01-31 Genes del factor viii optimizados. MX2018009375A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662289696P 2016-02-01 2016-02-01
US201662409739P 2016-10-18 2016-10-18
PCT/US2017/015879 WO2017136358A1 (en) 2016-02-01 2017-01-31 Optimized factor viii genes

Publications (1)

Publication Number Publication Date
MX2018009375A true MX2018009375A (es) 2018-09-05

Family

ID=58044178

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018009375A MX2018009375A (es) 2016-02-01 2017-01-31 Genes del factor viii optimizados.
MX2023001394A MX2023001394A (es) 2016-02-01 2018-08-01 Genes del factor viii optimizados.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023001394A MX2023001394A (es) 2016-02-01 2018-08-01 Genes del factor viii optimizados.

Country Status (25)

Country Link
US (2) US11753461B2 (es)
EP (2) EP4137570A1 (es)
JP (3) JP7217630B2 (es)
KR (1) KR20180118659A (es)
CN (2) CN109072214B (es)
AU (2) AU2017214378B2 (es)
BR (1) BR112018015659A2 (es)
CA (1) CA3012695A1 (es)
CL (1) CL2018002054A1 (es)
CO (1) CO2018009120A2 (es)
DK (1) DK3411478T3 (es)
ES (1) ES2926585T3 (es)
HK (1) HK1257078A1 (es)
HR (1) HRP20221089T1 (es)
HU (1) HUE059941T2 (es)
IL (1) IL260796A (es)
LT (1) LT3411478T (es)
MX (2) MX2018009375A (es)
PH (1) PH12018501628A1 (es)
PL (1) PL3411478T3 (es)
PT (1) PT3411478T (es)
RS (1) RS63548B1 (es)
SG (2) SG10201913278PA (es)
SI (1) SI3411478T1 (es)
WO (1) WO2017136358A1 (es)

Families Citing this family (18)

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DK2956477T4 (da) 2013-02-15 2024-04-15 Bioverativ Therapeutics Inc Optimeret faktor viii-gen
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BR112020002394A2 (pt) * 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. moléculas de ácidos nucleicos e usos das mesmas
TW201946929A (zh) 2018-02-01 2019-12-16 美商生物化學醫療公司 表現因子ｖｉｉｉ之慢病毒載體之使用
KR102245539B1 (ko) 2018-02-12 2021-04-29 주식회사 지앤피바이오사이언스 코어-쉘 구조의 마이크로 입자를 유효성분으로 포함하는 성장인자 유전자 발현 증가용 조성물
WO2020033863A1 (en) * 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
MX2021006648A (es) * 2018-12-06 2021-07-07 Bioverativ Therapeutics Inc Uso de vectores lentivirales que expresan el factor ix.
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CA3136720A1 (en) 2019-04-17 2020-10-22 Codiak Biosciences, Inc. Compositions of exosomes and aav
TW202126284A (zh) * 2019-09-30 2021-07-16 美商百歐維拉提夫治療公司 慢病毒載體配製物
CN111647625A (zh) * 2019-12-25 2020-09-11 深圳三智医学科技有限公司 一种提高人凝血因子ix表达水平的方法
CN111808863B (zh) * 2020-06-23 2021-05-28 康霖生物科技（杭州）有限公司 一种密码子优化的凝血因子viii基因及其构建体
JP2024532261A (ja) 2021-08-23 2024-09-05 バイオベラティブ セラピューティクス インコーポレイテッド 操作ｉｔｒ配列および使用方法
MX2024002333A (es) 2021-08-23 2024-03-07 Bioverativ Therapeutics Inc Sistema de expresion de baculovirus.
WO2023028456A1 (en) * 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Optimized factor viii genes
JP2024537797A (ja) * 2021-09-30 2024-10-16 バイオベラティブ セラピューティクス インコーポレイテッド 低下した免疫原性を有する第ｖｉｉｉ因子ポリペプチドをコードする核酸
WO2023205300A2 (en) * 2022-04-20 2023-10-26 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
WO2023211315A1 (en) * 2022-04-28 2023-11-02 Joint Stock Company "Biocad" Isolated nucleic acid that encodes fusion protein based on fviii-bdd and on heterologous signal peptide

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