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
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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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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70546Integrin superfamily
    • C07K14/7055Integrin beta1-subunit-containing molecules, e.g. CD29, CD49
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124Materials 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

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

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
MX2020007071A (es) 2018-01-05 2020-11-11 Platelet Biogenesis Inc Composiciones y metodos para producir megacariocitos.
WO2020006539A1 (en) 2018-06-29 2020-01-02 Platelet Biogenesis, Inc. Compositions for drug delivery and methods of use thereof
AU2019354370A1 (en) * 2018-10-05 2021-05-13 University Of Utah Research Foundation Methods of making platelets comprising modified receptors and uses thereof
US11767511B2 (en) 2018-11-30 2023-09-26 Cellphire, Inc. Platelets as delivery agents
WO2020113090A1 (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
CA3138529C (en) 2019-05-03 2025-10-07 Cellphire Inc Materials and methods for producing blood products
WO2021034719A1 (en) 2019-08-16 2021-02-25 Cellphire, Inc. Thrombosomes as an antiplatelet agent reversal agent
CA3170134A1 (en) 2020-02-04 2021-08-12 Cellphire, Inc. Treatment of von willebrand disease
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
US20220273724A1 (en) 2021-02-17 2022-09-01 Cellphire Inc. Freeze-dried platelet derivative compositions for treating antiplatelet induced coagulopathy

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040192599A1 (en) * 2001-06-15 2004-09-30 Schuh Andre C Gene therapy for hemophilia a

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2452892A (en) 1947-05-07 1948-11-02 Robert A Appleton Igniter for rockets
US4596556A (en) 1985-03-25 1986-06-24 Bioject, Inc. Hypodermic injection apparatus
EP0468520A3 (en) 1990-07-27 1992-07-01 Mitsui Toatsu Chemicals, Inc. Immunostimulatory remedies containing palindromic dna sequences
IL101715A (en) 1991-05-02 2005-06-19 Amgen Inc Recombinant dna-derived cholera toxin subunit analogs
IT1253009B (it) 1991-12-31 1995-07-10 Sclavo Ricerca S R L Mutanti immunogenici detossificati della tossina colerica e della tossina lt, loro preparazione ed uso per la preparazione di vaccini
EP0761231B1 (en) 1992-06-25 2000-01-12 SMITHKLINE BEECHAM BIOLOGICALS s.a. Vaccine composition containing adjuvants
GB9326253D0 (en) 1993-12-23 1994-02-23 Smithkline Beecham Biolog Vaccines
EP0772619B2 (en) 1994-07-15 2010-12-08 The University of Iowa Research Foundation Immunomodulatory oligonucleotides
UA56132C2 (uk) 1995-04-25 2003-05-15 Смітклайн Бічем Байолоджікалс С.А. Композиція вакцини (варіанти), спосіб стабілізації qs21 відносно гідролізу (варіанти), спосіб приготування композиції вакцини
DE69719761T2 (de) 1996-06-18 2003-12-18 Alza Corp., Palo Alto Vorrichtung zur verbesserung der transdermalen verabreichung von medikamenten oder der abnahme von körperflüssigkeiten
US5980898A (en) 1996-11-14 1999-11-09 The United States Of America As Represented By The U.S. Army Medical Research & Material Command Adjuvant for transcutaneous immunization
DK0957972T3 (da) 1996-12-20 2003-07-21 Alza Corp Anordning og fremgangsmåde til at forøge transdermal middelflux
US5993412A (en) 1997-05-19 1999-11-30 Bioject, Inc. Injection apparatus
GB9711990D0 (en) 1997-06-11 1997-08-06 Smithkline Beecham Biolog Vaccine
GB9718901D0 (en) 1997-09-05 1997-11-12 Smithkline Beecham Biolog Vaccine
DE69838992T2 (de) 1997-09-05 2008-12-24 Glaxosmithkline Biologicals S.A., Rixensart Öl-in-Wasser Emulsionen mit Saponinen
EP1035867A1 (en) 1997-12-02 2000-09-20 Powderject Vaccines, Inc. Transdermal delivery of particulate vaccine compositions
EP1048736A1 (en) * 1999-04-27 2000-11-02 Aventis Behring Gesellschaft mit beschränkter Haftung DNA-construct for the tissue specific expression of a blood coagulation factor
DK1412493T3 (da) * 2001-08-02 2012-01-09 Inst Clayton De La Rech Fremgangsmåder og sammensætninger der angår forbedrede lentiviral-vektor.-produktionsystemer
CA2471968C (en) 2001-10-06 2013-07-23 Merial Limited Immunostimulatory nucleic acids and use thereof
US7417026B2 (en) 2003-08-13 2008-08-26 Children's Hospital Medical Center Mobilization of hematopoietic cells
AU2005300688B2 (en) * 2004-11-03 2012-02-02 Almac Diagnostics Limited Transcriptome microarray technology and methods of using the same
KR100981092B1 (ko) * 2008-02-29 2010-09-08 고려대학교 산학협력단 제8 혈액응고 인자와 폰 빌리 블란트 인자 변이체의 재조합발현 벡터 시스템
WO2010075303A1 (en) * 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
US8147820B2 (en) 2009-09-14 2012-04-03 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Attenuated vaccine against fish pathogen Francisella sp
MX2012005527A (es) 2009-11-13 2012-08-08 Grifols Therapeutics Inc Preparaciones que contienen el factor de von willebrand (fvw) procedimientos, kits y aplicaciones relacionados con las mismas.
US20120225028A1 (en) * 2011-03-02 2012-09-06 Moshe Cohen Compositions and methods for mobilization of stem cells

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040192599A1 (en) * 2001-06-15 2004-09-30 Schuh Andre C Gene therapy for hemophilia a

Also Published As

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

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