CA2888982C - Platelet targeted treatment - Google Patents

Platelet targeted treatment Download PDF

Info

Publication number
CA2888982C
CA2888982C CA2888982A CA2888982A CA2888982C CA 2888982 C CA2888982 C CA 2888982C CA 2888982 A CA2888982 A CA 2888982A CA 2888982 A CA2888982 A CA 2888982A CA 2888982 C CA2888982 C CA 2888982C
Authority
CA
Canada
Prior art keywords
vector
gene
animal
human
stem cell
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CA2888982A
Other languages
English (en)
French (fr)
Other versions
CA2888982A1 (en
Inventor
David A. Wilcox
Sandra L. Haberichter
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Platelet Targeted Therapeutics LLC
Original Assignee
Platelet Targeted Therapeutics LLC
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Platelet Targeted Therapeutics LLC filed Critical Platelet Targeted Therapeutics LLC
Publication of CA2888982A1 publication Critical patent/CA2888982A1/en
Application granted granted Critical
Publication of CA2888982C publication Critical patent/CA2888982C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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

Landscapes

  • 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)
CA2888982A 2012-10-24 2013-10-24 Platelet targeted treatment Active CA2888982C (en)

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
CA2888982A1 CA2888982A1 (en) 2014-05-01
CA2888982C true CA2888982C (en) 2020-07-21

Family

ID=50545271

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2888982A Active CA2888982C (en) 2012-10-24 2013-10-24 Platelet targeted treatment

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
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
BR112015009229B1 (pt) 2021-07-20
EP2912186A4 (en) 2016-06-08
KR20150069017A (ko) 2015-06-22
US9982034B2 (en) 2018-05-29

Similar Documents

Publication Publication Date Title
US20240270822A1 (en) Platelet targeted treatment
AU2011377617B2 (en) Improved gene therapy methods
JP3941963B2 (ja) 全長第viii因子のレトロウイルス送達
Gangadharan et al. High-level expression of porcine factor VIII from genetically modified bone marrow–derived stem cells
US20090148425A1 (en) Therapeutic method for blood coagulation disorder
JP2018519827A (ja) 逆配向のヒトユビキチンcプロモーターを含むレトロウイルスベクター
Yamada et al. Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector
WO2005116225A1 (en) Method for introducing and expressing rna in cells
HK40057844A (en) Platelet targeted treatment
HK1211986B (en) Platelet targeted treatment
Coquin et al. Syncytins enable novel possibilities to transduce human or mouse primary B cells and to achieve well-tolerated in vivo gene transfer
KR100969271B1 (ko) 제8 혈액응고 인자와 폰 빌리 블란트 인자 변이체의 재조합 발현 벡터 시스템
KR100969268B1 (ko) 제8 혈액응고 인자와 폰 빌리 블란트 인자 변이체의 재조합 발현 벡터 시스템
Johnston Optimization of a Lentiviral Vector for use in an HSC Gene Therapy Protocol for Hemophilia A
Gutiérrez-Guerrero Gene editing as an alternative to retroviral vectors for Wiskott-Aldrich syndrome gene therapy
Ward Lentiviral vectors for treatment of haemophilia
Ohmori et al. Platelet-directed gene therapy
Abdul-Razak Correction of the classical scid mouse mutation by gene repair
Rey Genetic modification in hematopoietic stem cells, using lentiviral vectors, to target protein expression in megakaryocyte and platelets
HK1127549A (en) Therapeutic method for blood coagulation disorder
Moayeri Gene therapy for hemophilia A: Targeting long-term hematopoietic repopulating cells with a retroviral vector expressing a bioengineered human factor VIII gene

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20150421

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 11TH ANNIV.) - STANDARD

Year of fee payment: 11

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241018

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241018

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241018

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 12TH ANNIV.) - STANDARD

Year of fee payment: 12

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20251017

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20251017