KR101819803B1 - 혈소판 표적화 치료 - Google Patents

혈소판 표적화 치료 Download PDF

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KR101819803B1
KR101819803B1 KR1020157012821A KR20157012821A KR101819803B1 KR 101819803 B1 KR101819803 B1 KR 101819803B1 KR 1020157012821 A KR1020157012821 A KR 1020157012821A KR 20157012821 A KR20157012821 A KR 20157012821A KR 101819803 B1 KR101819803 B1 KR 101819803B1
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gene
human
promoter
vector
expression
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KR20150069017A (ko
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데이비드 에이. 윌콕스
산드라 엘. 하베릭터
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플레이틀렛 타게티드 테라퓨틱스, 엘엘씨
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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
    • 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
    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70546—Integrin superfamily
    • C07K14/7055—Integrin beta1-subunit-containing molecules, e.g. CD29, CD49
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Developmental Biology & Embryology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020157012821A 2012-10-24 2013-10-24 혈소판 표적화 치료 Active KR101819803B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261717951P 2012-10-24 2012-10-24
US61/717,951 2012-10-24
PCT/US2013/066651 WO2014066663A1 (en) 2012-10-24 2013-10-24 Platelet targeted treatment

Publications (2)

Publication Number Publication Date
KR20150069017A KR20150069017A (ko) 2015-06-22
KR101819803B1 true KR101819803B1 (ko) 2018-01-17

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KR1020157012821A Active KR101819803B1 (ko) 2012-10-24 2013-10-24 혈소판 표적화 치료

Country Status (12)

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US (4) US9982034B2 (https=)
EP (2) EP2912186B1 (https=)
JP (2) JP2016501515A (https=)
KR (1) KR101819803B1 (https=)
BR (1) BR112015009229B1 (https=)
CA (1) CA2888982C (https=)
DK (1) DK2912186T3 (https=)
ES (1) ES2854754T3 (https=)
IL (1) IL238417B (https=)
PL (1) PL2912186T3 (https=)
WO (1) WO2014066663A1 (https=)
ZA (1) ZA201502945B (https=)

Families Citing this family (12)

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US11319528B2 (en) 2015-07-13 2022-05-03 University Of Utah Research Foundation Methods of making red blood cells and platelets in vitro and uses thereof
JP2021509812A (ja) 2018-01-05 2021-04-08 プレートレット バイオジェネシス， インコーポレイテッド 巨核球を産生するための組成物および方法
EP3813853A4 (en) 2018-06-29 2022-04-06 Platelet Biogenesis, Inc. DELIVERY COMPOSITIONS AND METHODS OF USE
JP2022512622A (ja) * 2018-10-05 2022-02-07 ユニヴァーシティー オブ ユタ リサーチ ファウンデーション 改変受容体を含む血小板の作製方法及びその使用法
CA3121200A1 (en) 2018-11-30 2020-06-04 Cellphire, Inc. Platelets as delivery agents
WO2020112963A1 (en) 2018-11-30 2020-06-04 Cellphire, Inc. Platelets as delivery agents
EP3937631A4 (en) * 2019-03-13 2023-01-04 Cellphire Inc. DOG PLALETLET PREPARATIONS
IL322149A (en) 2019-05-03 2025-09-01 Cellphire Inc Materials and methods for manufacturing blood products
CN114450066A (zh) 2019-08-16 2022-05-06 塞尔菲乐有限公司 作为抗血小板剂逆转剂的血栓小体
CA3170196A1 (en) 2020-02-04 2021-08-12 Cellphire, Inc. Anti-fibrinolytic loaded platelets
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
TW202245814A (zh) 2021-02-17 2022-12-01 美商賽菲爾公司 用於治療抗血小板誘導的凝血病之凍乾血小板衍生物組成物

Citations (1)

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US20040192599A1 (en) * 2001-06-15 2004-09-30 Schuh Andre C Gene therapy for hemophilia a

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Also Published As

Publication number Publication date
ZA201502945B (en) 2016-11-30
IL238417A0 (en) 2015-06-30
JP6975105B2 (ja) 2021-12-01
WO2014066663A1 (en) 2014-05-01
JP2016501515A (ja) 2016-01-21
CA2888982C (en) 2020-07-21
BR112015009229A2 (pt) 2017-11-14
PL2912186T3 (pl) 2021-06-14
US20240270822A1 (en) 2024-08-15
JP2018161152A (ja) 2018-10-18
HK1211986A1 (en) 2016-06-03
US20190270791A1 (en) 2019-09-05
IL238417B (en) 2019-07-31
US20150344541A1 (en) 2015-12-03
EP2912186B1 (en) 2021-01-06
US20180009874A1 (en) 2018-01-11
CA2888982A1 (en) 2014-05-01
DK2912186T3 (da) 2021-02-22
US10294291B2 (en) 2019-05-21
ES2854754T3 (es) 2021-09-22
EP2912186A1 (en) 2015-09-02
EP3859004A1 (en) 2021-08-04
BR112015009229B1 (pt) 2021-07-20
EP2912186A4 (en) 2016-06-08
KR20150069017A (ko) 2015-06-22
US9982034B2 (en) 2018-05-29

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