MX2019014379A - Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso. - Google Patents

Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso.

Info

Publication number
MX2019014379A
MX2019014379A MX2019014379A MX2019014379A MX2019014379A MX 2019014379 A MX2019014379 A MX 2019014379A MX 2019014379 A MX2019014379 A MX 2019014379A MX 2019014379 A MX2019014379 A MX 2019014379A MX 2019014379 A MX2019014379 A MX 2019014379A
Authority
MX
Mexico
Prior art keywords
gene expression
expression cassettes
clotting factor
optimized human
human clotting
Prior art date
Application number
MX2019014379A
Other languages
English (en)
Spanish (es)
Inventor
Xiao Xiao
Li Juan
Original Assignee
Univ North Carolina Chapel Hill
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 Univ North Carolina Chapel Hill filed Critical Univ North Carolina Chapel Hill
Publication of MX2019014379A publication Critical patent/MX2019014379A/es

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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/067—Hepatocytes
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0693—Tumour cells; Cancer cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • 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
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
MX2019014379A 2017-05-31 2018-05-31 Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso. MX2019014379A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762512833P 2017-05-31 2017-05-31
PCT/US2018/035267 WO2018222792A1 (en) 2017-05-31 2018-05-31 Optimized human clotting factor ix gene expression cassettes and their use

Publications (1)

Publication Number Publication Date
MX2019014379A true MX2019014379A (es) 2020-01-23

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019014379A MX2019014379A (es) 2017-05-31 2018-05-31 Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso.
MX2024007348A MX2024007348A (es) 2017-05-31 2019-11-29 Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso.

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MX2024007348A MX2024007348A (es) 2017-05-31 2019-11-29 Casetes de expresion optimizados del gen del factor de coagulacion ix humano y su uso.

Country Status (9)

Country Link
US (2) US11530402B2 (enExample)
EP (1) EP3630978A4 (enExample)
JP (3) JP7119009B2 (enExample)
KR (2) KR102625470B1 (enExample)
CN (2) CN108977452B (enExample)
AU (1) AU2018275549B2 (enExample)
CA (1) CA3064450A1 (enExample)
MX (2) MX2019014379A (enExample)
WO (1) WO2018222792A1 (enExample)

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WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
KR20220097891A (ko) 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
WO2021087007A1 (en) * 2019-10-28 2021-05-06 University Of Florida Research Foundation, Incorporated Gene therapy vectors
KR20220164743A (ko) * 2020-04-06 2022-12-13 호몰로지 메디슨, 인크. Ids 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
CN113817759B (zh) * 2020-07-10 2023-06-02 南京吉迈生物技术有限公司 修饰的因子ix、组合物、方法及其在基因治疗中的应用
CN115109788A (zh) * 2021-03-23 2022-09-27 北京锦篮基因科技有限公司 Aav介导的人脂蛋白脂酶肝脏异位表达载体及其用途
CN117693593A (zh) * 2021-06-23 2024-03-12 英斯培瑞有限公司 用于治疗b型血友病的组合物和方法
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
JP2024540085A (ja) * 2021-10-27 2024-10-31 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 血友病ｂ療法のための第ｉｘ因子を発現させるための組成物及び方法
CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2025143742A1 (ko) * 2023-12-26 2025-07-03 삼성바이오에피스 주식회사 혈액응고인자 IX를 암호화하는 코돈-최적화된 폴리뉴클레오티드 및 이를 포함하는 rAAV 생산용 플라스미드

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HUE051311T2 (hu) 2014-03-09 2021-03-01 Univ Pennsylvania Ornitin transzkarbamiláz (TC) deficiencia kezelésében alkalmas készítmények
KR102244434B1 (ko) * 2014-08-11 2021-04-23 삼성전자주식회사 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법
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AU2016215124B2 (en) 2015-02-06 2020-08-06 The University Of North Carolina At Chapel Hill Optimized human clotting Factor VIII gene expression cassettes and their use
IL256517B2 (en) * 2015-06-23 2024-06-01 Childrens Hospital Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
US10086089B2 (en) * 2015-09-18 2018-10-02 DNARx Systems and methods for nucleic acid expression in vivo
EP3442597A1 (en) * 2016-04-15 2019-02-20 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia b
CN106497949A (zh) * 2016-10-14 2017-03-15 上海交通大学医学院附属瑞金医院 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用
JP2020511521A (ja) * 2017-03-23 2020-04-16 ディーエヌエーアールエックス ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法

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Publication number Publication date
US20230091502A1 (en) 2023-03-23
CN114875051A (zh) 2022-08-09
MX2024007348A (es) 2024-06-26
US11530402B2 (en) 2022-12-20
US12209263B2 (en) 2025-01-28
CN108977452B (zh) 2022-07-19
US20200087646A1 (en) 2020-03-19
EP3630978A4 (en) 2021-03-24
JP2020522259A (ja) 2020-07-30
KR20200014318A (ko) 2020-02-10
JP2024097922A (ja) 2024-07-19
WO2018222792A1 (en) 2018-12-06
CA3064450A1 (en) 2018-12-06
KR20240010098A (ko) 2024-01-23
JP7119009B2 (ja) 2022-08-16
AU2018275549B2 (en) 2024-12-19
CN108977452A (zh) 2018-12-11
KR102625470B1 (ko) 2024-01-16
JP2022159358A (ja) 2022-10-17
AU2018275549A1 (en) 2019-11-21
EP3630978A1 (en) 2020-04-08

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