JP2016501515A - 血小板標的化治療 - Google Patents

血小板標的化治療 Download PDF

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Publication number
JP2016501515A
JP2016501515A JP2015539804A JP2015539804A JP2016501515A JP 2016501515 A JP2016501515 A JP 2016501515A JP 2015539804 A JP2015539804 A JP 2015539804A JP 2015539804 A JP2015539804 A JP 2015539804A JP 2016501515 A JP2016501515 A JP 2016501515A
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gene
nucleotides
vector
platelets
expression
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JP2015539804A
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English (en)
Japanese (ja)
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JP2016501515A5 (enExample
Inventor
ウィルコックス，デイヴィッド，エー．
ヘーバーリクター，サンドラ，エル．
Original Assignee
プレートレット ターゲテッド セラピューティクス，エルエルシー
プレートレット ターゲテッド セラピューティクス，エルエルシー
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Application filed by プレートレット ターゲテッド セラピューティクス，エルエルシー, プレートレット ターゲテッド セラピューティクス，エルエルシー filed Critical プレートレット ターゲテッド セラピューティクス，エルエルシー
Publication of JP2016501515A publication Critical patent/JP2016501515A/ja
Publication of JP2016501515A5 publication Critical patent/JP2016501515A5/ja
Priority to JP2018135886A priority Critical patent/JP6975105B2/ja
Pending legal-status Critical Current

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    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
JP2015539804A 2012-10-24 2013-10-24 血小板標的化治療 Pending JP2016501515A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2018135886A JP6975105B2 (ja) 2012-10-24 2018-07-19 血小板標的化治療

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2018135886A Division JP6975105B2 (ja) 2012-10-24 2018-07-19 血小板標的化治療

Publications (2)

Publication Number Publication Date
JP2016501515A true JP2016501515A (ja) 2016-01-21
JP2016501515A5 JP2016501515A5 (enExample) 2016-11-10

Family

ID=50545271

Family Applications (2)

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JP2015539804A Pending JP2016501515A (ja) 2012-10-24 2013-10-24 血小板標的化治療
JP2018135886A Active JP6975105B2 (ja) 2012-10-24 2018-07-19 血小板標的化治療

Family Applications After (1)

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JP2018135886A Active JP6975105B2 (ja) 2012-10-24 2018-07-19 血小板標的化治療

Country Status (12)

Country Link
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
WO2017011550A1 (en) 2015-07-13 2017-01-19 University Of Utah Research Foundation Methods of making red blood cells and platelets in vitro and uses thereof
US20200291356A1 (en) * 2019-03-13 2020-09-17 Cellphire, Inc. Canine blood platelet preparations
EP3735457A4 (en) 2018-01-05 2021-08-25 Platelet Biogenesis, Inc. COMPOSITIONS AND METHODS FOR PRODUCING MEGACARYOCYTES
US12403161B2 (en) 2018-06-29 2025-09-02 Stellular Bio, Inc. Compositions for drug delivery and methods of use thereof
US20220033776A1 (en) * 2018-10-05 2022-02-03 University Of Utah Research Foundation Methods of making platelets comprising modified receptors and uses thereof
EP3887402A4 (en) 2018-11-30 2022-10-26 Cellphire, Inc. PLATELETS AS RELEASING AGENT
WO2020112963A1 (en) 2018-11-30 2020-06-04 Cellphire, Inc. Platelets as delivery agents
BR112021021984A2 (pt) 2019-05-03 2022-03-03 Cellphire Inc Materiais e métodos para produzir produtos de sangue
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
CA3170201A1 (en) 2020-02-04 2021-08-12 Cellphire, Inc. Methods of treating congenital hemophilia with 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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2000333687A (ja) * 1999-04-27 2000-12-05 Aventis Behring Gmbh 血液凝固因子の組織特異的発現のためのｄｎａ構築体
US20040192599A1 (en) * 2001-06-15 2004-09-30 Schuh Andre C Gene therapy for hemophilia a
US20120225028A1 (en) * 2011-03-02 2012-09-06 Moshe Cohen Compositions and methods for mobilization of stem cells

Family Cites Families (25)

* 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
ATE156710T1 (de) 1992-06-25 1997-08-15 Smithkline Beecham Biolog Adjuvantien enthaltende impfstoffzusammensetzung
GB9326253D0 (en) 1993-12-23 1994-02-23 Smithkline Beecham Biolog Vaccines
US6194388B1 (en) 1994-07-15 2001-02-27 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
ZA975326B (en) 1996-06-18 1998-01-14 Alza Corp Device and method for enhancing transdermal flux of agents being delivered or sampled.
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
JP4153999B2 (ja) 1996-12-20 2008-09-24 アルザ・コーポレーション 経皮作用剤流量を強化するための組成物と方法
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
CA2302554C (en) 1997-09-05 2007-04-10 Smithkline Beecham Biologicals S.A. Oil in water emulsions containing saponins
NZ504894A (en) 1997-12-02 2002-12-20 Powderject Vaccines Inc Crystalline vaccine and adjuvant compositions jointly and separately administered to animals using a transdermal or transmucosally delivery technique
DK1412493T3 (da) 2001-08-02 2012-01-09 Inst Clayton De La Rech Fremgangsmåder og sammensætninger der angår forbedrede lentiviral-vektor.-produktionsystemer
US20050238660A1 (en) 2001-10-06 2005-10-27 Babiuk Lorne A Cpg formulations and related methods
WO2005017160A2 (en) 2003-08-13 2005-02-24 Children's Hospital Medical Center Mobilization of hematopoietic cells
JP2008518610A (ja) 2004-11-03 2008-06-05 アルマック ダイアグノスティックス リミテッド トランスクリプトームマイクロアレイ技法およびそれを使用する方法
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
RU2579977C2 (ru) * 2009-11-13 2016-04-10 Грифольс Терапьютикс Инк. СОДЕРЖАЩИЕ ФАКТОР ФОН ВИЛЛЕБРАНДА (vWF) ПРЕПАРАТЫ И СПОСОБЫ, НАБОРЫ И ПРИМЕНЕНИЯ, СВЯЗАННЫЕ С НИМИ

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2000333687A (ja) * 1999-04-27 2000-12-05 Aventis Behring Gmbh 血液凝固因子の組織特異的発現のためのｄｎａ構築体
US20040192599A1 (en) * 2001-06-15 2004-09-30 Schuh Andre C Gene therapy for hemophilia a
US20120225028A1 (en) * 2011-03-02 2012-09-06 Moshe Cohen Compositions and methods for mobilization of stem cells

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BIOCHEM. BIOPHYS. RES. COMMUN., vol. 156(1), JPN6018010731, 1988, pages 595 - 601, ISSN: 0003765660 *
Q.SHI, ET AL, JOURNAL OF THROMBOSIS AND HAEMOSTASIS, vol. 5, JPN6017029033, 2007, pages 352 - 361, ISSN: 0003765659 *
Q.SHI, ET AL, MOLECULAR GENETICS AND METABOLISM, vol. 79, JPN6017029031, 2003, pages 25 - 33, ISSN: 0003765657 *
S.L.HABERICHTER, ET AL, ARTERIOSCLER THROMB VASC BIOL., vol. 22, JPN6017029036, 2002, pages 921 - 926, ISSN: 0003765658 *

Also Published As

Publication number Publication date
IL238417B (en) 2019-07-31
US20150344541A1 (en) 2015-12-03
US9982034B2 (en) 2018-05-29
HK1211986A1 (en) 2016-06-03
PL2912186T3 (pl) 2021-06-14
DK2912186T3 (da) 2021-02-22
CA2888982A1 (en) 2014-05-01
US20240270822A1 (en) 2024-08-15
EP2912186A1 (en) 2015-09-02
IL238417A0 (en) 2015-06-30
BR112015009229B1 (pt) 2021-07-20
EP2912186B1 (en) 2021-01-06
BR112015009229A2 (pt) 2017-11-14
EP3859004A1 (en) 2021-08-04
US20180009874A1 (en) 2018-01-11
EP2912186A4 (en) 2016-06-08
WO2014066663A1 (en) 2014-05-01
ZA201502945B (en) 2016-11-30
CA2888982C (en) 2020-07-21
US10294291B2 (en) 2019-05-21
US20190270791A1 (en) 2019-09-05
KR101819803B1 (ko) 2018-01-17
KR20150069017A (ko) 2015-06-22
JP2018161152A (ja) 2018-10-18
ES2854754T3 (es) 2021-09-22
JP6975105B2 (ja) 2021-12-01

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