BR112015009229B1 - Composição tendo como alvo a expressão de genes para plaquetas, uso de uma célula tronco compreendendo a referida composição para tratar hemofilia e método in vitro ou ex vivo para gerar uma célula tronco modificada - Google Patents

Composição tendo como alvo a expressão de genes para plaquetas, uso de uma célula tronco compreendendo a referida composição para tratar hemofilia e método in vitro ou ex vivo para gerar uma célula tronco modificada Download PDF

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Publication number
BR112015009229B1
BR112015009229B1 BR112015009229-2A BR112015009229A BR112015009229B1 BR 112015009229 B1 BR112015009229 B1 BR 112015009229B1 BR 112015009229 A BR112015009229 A BR 112015009229A BR 112015009229 B1 BR112015009229 B1 BR 112015009229B1
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Prior art keywords
gene
expression
platelets
fviii
vector
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BR112015009229-2A
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Portuguese (pt)
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BR112015009229A2 (pt
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David A. Wilcox
Sandra L. Haberichter
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Platelet Targeted Therapeutics, Llc
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Publication of BR112015009229A2 publication Critical patent/BR112015009229A2/pt
Publication of BR112015009229B1 publication Critical patent/BR112015009229B1/pt

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    • 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)
    • 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
    • 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
    • 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)
BR112015009229-2A 2012-10-24 2013-10-24 Composição tendo como alvo a expressão de genes para plaquetas, uso de uma célula tronco compreendendo a referida composição para tratar hemofilia e método in vitro ou ex vivo para gerar uma célula tronco modificada BR112015009229B1 (pt)

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
BR112015009229A2 BR112015009229A2 (pt) 2017-11-14
BR112015009229B1 true BR112015009229B1 (pt) 2021-07-20

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015009229-2A BR112015009229B1 (pt) 2012-10-24 2013-10-24 Composição tendo como alvo a expressão de genes para plaquetas, uso de uma célula tronco compreendendo a referida composição para tratar hemofilia e método in vitro ou ex vivo para gerar uma célula tronco modificada

Country Status (12)

Country Link
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)

* 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
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

Family Cites Families (28)

* 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
CA2138997C (en) 1992-06-25 2003-06-03 Jean-Paul Prieels Vaccine composition containing adjuvants
GB9326253D0 (en) 1993-12-23 1994-02-23 Smithkline Beecham Biolog Vaccines
EP1167379A3 (en) 1994-07-15 2004-09-08 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
CA2253549C (en) 1996-06-18 2005-10-25 Alza Corporation Device for enhancing transdermal agent delivery or sampling
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
EP0957972B1 (en) 1996-12-20 2003-03-19 Alza Corporation Device and method for enhancing transdermal agent flux
US5993412A (en) 1997-05-19 1999-11-30 Bioject, Inc. Injection apparatus
GB9711990D0 (en) 1997-06-11 1997-08-06 Smithkline Beecham Biolog Vaccine
AU1145699A (en) 1997-09-05 1999-03-22 Smithkline Beecham Biologicals (Sa) Oil in water emulsions containing saponins
GB9718901D0 (en) 1997-09-05 1997-11-12 Smithkline Beecham Biolog Vaccine
CA2312900A1 (en) 1997-12-02 1999-06-10 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
CA2450125A1 (en) 2001-06-15 2002-12-27 Andre C. Schuh Gene therapy for hemophilia a
ATE527347T1 (de) * 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
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
EP1815021A2 (en) * 2004-11-03 2007-08-08 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
WO2011060242A2 (en) 2009-11-13 2011-05-19 Talecris Biotherapeutics, Inc. Von willebrand factor (vwf)-containing preparations, and methods, kits, and uses related thereto
US20120225028A1 (en) 2011-03-02 2012-09-06 Moshe Cohen Compositions and methods for mobilization of stem cells

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
KR101819803B1 (ko) 2018-01-17
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
EP2912186A4 (en) 2016-06-08
KR20150069017A (ko) 2015-06-22
US9982034B2 (en) 2018-05-29

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B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B06I Publication of requirement cancelled [chapter 6.9 patent gazette]

Free format text: ANULADA A PUBLICACAO CODIGO 6.6.1 NA RPI NO 2462 DE 13/03/2018 POR TER SIDO INDEVIDA.

B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B09A Decision: intention to grant [chapter 9.1 patent gazette]
B16A Patent or certificate of addition of invention granted [chapter 16.1 patent gazette]

Free format text: PRAZO DE VALIDADE: 20 (VINTE) ANOS CONTADOS A PARTIR DE 24/10/2013, OBSERVADAS AS CONDICOES LEGAIS.