CA3064450A1 - Optimized human clotting factor ix gene expression cassettes and their use - Google Patents

Optimized human clotting factor ix gene expression cassettes and their use Download PDF

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Publication number
CA3064450A1
CA3064450A1 CA3064450A CA3064450A CA3064450A1 CA 3064450 A1 CA3064450 A1 CA 3064450A1 CA 3064450 A CA3064450 A CA 3064450A CA 3064450 A CA3064450 A CA 3064450A CA 3064450 A1 CA3064450 A1 CA 3064450A1
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polynucleotide
sequence
vector
seq
nucleotide sequence
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French (fr)
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Xiao Xiao
Juan Li
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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    • 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
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    • 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
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • 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
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    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • 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
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    • 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
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    • C12N2800/00—Nucleic acids vectors
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    • C12N2800/00—Nucleic acids vectors
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    • 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)

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3064450A 2017-05-31 2018-05-31 Optimized human clotting factor ix gene expression cassettes and their use Pending CA3064450A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762512833P 2017-05-31 2017-05-31
US62/512,833 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

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CA3064450A1 true CA3064450A1 (en) 2018-12-06

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CA3064450A Pending CA3064450A1 (en) 2017-05-31 2018-05-31 Optimized human clotting factor ix gene expression cassettes and their use

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US (2) US11530402B2 (https=)
EP (1) EP3630978A4 (https=)
JP (3) JP7119009B2 (https=)
KR (2) KR102625470B1 (https=)
CN (2) CN114875051A (https=)
AU (1) AU2018275549B2 (https=)
CA (1) CA3064450A1 (https=)
MX (2) MX2019014379A (https=)
WO (1) WO2018222792A1 (https=)

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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
JP2024521537A (ja) * 2021-06-23 2024-05-31 フロンテーラ セラピューティクス，インク． 血友病ｂを処置するための組成物および方法
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
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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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CN116392609A (zh) * 2017-03-23 2023-07-07 Dnarx公司 用于体内核酸表达的系统和方法

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EP3630978A4 (en) 2021-03-24
CN108977452B (zh) 2022-07-19
JP2020522259A (ja) 2020-07-30
JP2024097922A (ja) 2024-07-19
JP2022159358A (ja) 2022-10-17
KR20200014318A (ko) 2020-02-10
US20200087646A1 (en) 2020-03-19
KR102625470B1 (ko) 2024-01-16
CN114875051A (zh) 2022-08-09
US11530402B2 (en) 2022-12-20
KR20240010098A (ko) 2024-01-23
MX2019014379A (es) 2020-01-23
MX2024007348A (es) 2024-06-26
US12209263B2 (en) 2025-01-28
AU2018275549B2 (en) 2024-12-19
CN108977452A (zh) 2018-12-11
WO2018222792A1 (en) 2018-12-06
JP7119009B2 (ja) 2022-08-16
US20230091502A1 (en) 2023-03-23
EP3630978A1 (en) 2020-04-08
AU2018275549A1 (en) 2019-11-21

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