CY1123792T1 - Συνθεσεις για καταστολη σχηματiσμου αναστολεων εναντι του παραγοντα viii σε ασθενεις με αιμορροφιλια α - Google Patents

Συνθεσεις για καταστολη σχηματiσμου αναστολεων εναντι του παραγοντα viii σε ασθενεις με αιμορροφιλια α

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Publication number
CY1123792T1
CY1123792T1 CY20201101009T CY201101009T CY1123792T1 CY 1123792 T1 CY1123792 T1 CY 1123792T1 CY 20201101009 T CY20201101009 T CY 20201101009T CY 201101009 T CY201101009 T CY 201101009T CY 1123792 T1 CY1123792 T1 CY 1123792T1
Authority
CY
Cyprus
Prior art keywords
protein
formation
patients
reactions
coagulation factor
Prior art date
Application number
CY20201101009T
Other languages
English (en)
Inventor
Roland W. Herzog
Henry Daniell
Original Assignee
The Trustees Of The University Of Pennsylvania
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 The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of CY1123792T1 publication Critical patent/CY1123792T1/el

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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
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • C07K14/28—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria from Vibrionaceae (F)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8201—Methods for introducing genetic material into plant cells, e.g. DNA, RNA, stable or transient incorporation, tissue culture methods adapted for transformation
    • C12N15/8214—Plastid transformation
    • 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
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8241—Phenotypically and genetically modified plants via recombinant DNA technology
    • C12N15/8242—Phenotypically and genetically modified plants via recombinant DNA technology with non-agronomic quality (output) traits, e.g. for industrial processing; Value added, non-agronomic traits
    • C12N15/8257—Phenotypically and genetically modified plants via recombinant DNA technology with non-agronomic quality (output) traits, e.g. for industrial processing; Value added, non-agronomic traits for the production of primary gene products, e.g. pharmaceutical products, interferon
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K36/00—Medicinal preparations of undetermined constitution containing material from algae, lichens, fungi or plants, or derivatives thereof, e.g. traditional herbal medicines
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Abstract

Η θεραπεία αντικατάστασης πρωτεϊνών για ασθενείς με αιμορροφιλία ή άλλες κληρονομικές ανεπάρκειες πρωτεΐνης συχνά περιπλέκεται από παθογόνες αποκρίσεις αντισωμάτων, συμπεριλαμβανομένων αντισωμάτων που εξουδετερώνουν τη θεραπευτική πρωτεΐνη ή που προδιαθέτουν σε αναφυλακτικές αντιδράσεις που ενδέχεται να θέτουν σε κίνδυνο τη ζωή με σχηματισμό lgE. Χρησιμοποιώντας την αιμορροφιλία ποντικού ως μοντέλο, έχουμε αναπτύξει ένα προφυλακτικό πρωτόκολλο ενάντια σε τέτοιες αντιδράσεις που δεν είναι επεμβατικές και δεν περιλαμβάνουν ανοσοκαταστολή ή γενετικό χειρισμό των κυττάρων του ασθενούς. Στοματική παράδοση του παράγοντα πήξης που εκφράζεται σε χλωροπλάστες, βιοενθυλακωμένη σε φυτικά κύτταρα, μπλοκάρει αποτελεσματικά τον σχηματισμό ανασταλτικών αντισωμάτων στη θεραπεία αντικατάστασης πρωτεϊνών. Οι τίτλοι αναστολέων ήταν ως επί το πλείστον μη ανιχνεύσιμοι και έως και 100 φορές χαμηλότεροι σε υποκείμενα υπό αγωγή σε σύγκριση με τους ελέγχους. Επιπλέον, αυτή η θεραπεία εξάλειψε θανατηφόρες αναφυλακτικές αντιδράσεις που εμφανίστηκαν μετά από 4 έως 6 εκθέσεις σε πρωτεΐνη ενδοφλέβιου παράγοντα πήξης.
CY20201101009T 2013-11-15 2020-10-22 Συνθεσεις για καταστολη σχηματiσμου αναστολεων εναντι του παραγοντα viii σε ασθενεις με αιμορροφιλια α CY1123792T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361905069P 2013-11-15 2013-11-15
US201361905071P 2013-11-15 2013-11-15
PCT/US2014/065994 WO2015073988A1 (en) 2013-11-15 2014-11-17 Compositions and methods for suppression of inhibitor formation against factor viii in hemophilia a patients

Publications (1)

Publication Number Publication Date
CY1123792T1 true CY1123792T1 (el) 2022-03-24

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US (3) US10233216B2 (el)
EP (2) EP3763381A1 (el)
AU (1) AU2014348289B2 (el)
CA (1) CA2967269C (el)
CY (1) CY1123792T1 (el)
DK (1) DK3068869T3 (el)
ES (1) ES2821882T3 (el)
HR (1) HRP20201680T1 (el)
HU (1) HUE050869T2 (el)
LT (1) LT3068869T (el)
PL (1) PL3068869T3 (el)
PT (1) PT3068869T (el)
RS (1) RS60929B1 (el)
SI (1) SI3068869T1 (el)
WO (1) WO2015073988A1 (el)

Families Citing this family (6)

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Publication number Priority date Publication date Assignee Title
EP2881463A1 (en) * 2013-12-09 2015-06-10 DRK-Blutspendedienst Baden-Württemberg-Hessen gGmbH Factor IX variants with clotting activity in absence of their cofactor and/or with increased F.IX clotting activity and their use for treating bleeding disorders
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CA3043995A1 (en) * 2015-11-16 2017-05-26 The Trustees Of The University Of Pennsylvania Targeted delivery of therapeutic proteins bioencapsulated in plant cells to cell types of interest for the treatment of disease
US11332754B2 (en) 2016-03-20 2022-05-17 The Trustees Of The University Of Pennsylvania Codon optimization for increasing transgene expression in chloroplasts of higher seed plants
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides

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Publication number Priority date Publication date Assignee Title
US20110179530A1 (en) 2001-01-23 2011-07-21 University Of Central Florida Research Foundation, Inc. Pharmaceutical Proteins, Human Therapeutics, Human Serum Albumin Insulin, Native Cholera Toxin B Subunit on Transgenic Plastids
WO2004005467A2 (en) 2002-07-03 2004-01-15 University Of Central Florida Expression of human interferon in transgenic chloroplasts
EP2080803B1 (en) 2000-03-01 2011-05-25 Auburn University Plastid transformation vectors for expressing proinsulin fused to the cholera toxin B-subunit in plants
WO2001064023A1 (en) 2000-03-02 2001-09-07 Auburn University Marker free transgenic plants: engineering the chloroplast genome without the use of antibiotic selection
EP2284274B1 (en) 2001-12-26 2015-08-26 University Of Central Florida Expression of protective antigens in transgenic chloroplasts and the production of improved vaccines
WO2006025879A2 (en) * 2004-05-11 2006-03-09 Wyeth Novel polynucleotides related to oligonucleotide arrays to monitor gene expression
GB0709803D0 (en) * 2007-05-22 2007-07-04 Ucl Business Plc Predictive method
WO2011057243A2 (en) * 2009-11-09 2011-05-12 University Of Central Florida Research Foundation, Inc. Administration of plant expressed oral tolerance agents
US8454957B2 (en) * 2010-06-17 2013-06-04 Stemnion, Inc. Methods for treating coagulation disorders
US20130017997A1 (en) * 2010-08-19 2013-01-17 Amunix Operating Inc. Factor VIII Compositions and Methods of Making and Using Same
US10865419B2 (en) * 2011-10-24 2020-12-15 The Trustees Of The University Of Pennsylvania Orally administered plastid expressed cholera toxin B subunit-exendin 4 as treatment for type 2 diabetes
CN108465106B (zh) * 2012-01-12 2022-05-27 普吉特海湾血液中心 降低经受因子viii疗法的个体中针对因子viii的免疫原性的方法

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Publication number Publication date
LT3068869T (lt) 2020-10-26
WO2015073988A1 (en) 2015-05-21
EP3763381A1 (en) 2021-01-13
US20200270313A1 (en) 2020-08-27
SI3068869T1 (sl) 2020-11-30
HUE050869T2 (hu) 2021-01-28
US10233216B2 (en) 2019-03-19
PL3068869T3 (pl) 2021-03-08
PT3068869T (pt) 2020-10-08
AU2014348289B2 (en) 2020-11-26
AU2014348289A1 (en) 2016-06-16
EP3068869A4 (en) 2017-04-05
CA2967269C (en) 2022-04-26
ES2821882T3 (es) 2021-04-28
CA2967269A1 (en) 2015-02-21
US20160289277A1 (en) 2016-10-06
RS60929B1 (sr) 2020-11-30
US20190194265A1 (en) 2019-06-27
DK3068869T3 (da) 2020-09-07
US10556932B2 (en) 2020-02-11
EP3068869B1 (en) 2020-07-22
EP3068869A1 (en) 2016-09-21
HRP20201680T1 (hr) 2021-03-19

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