MX2018005272A - Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos. - Google Patents

Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos.

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Publication number
MX2018005272A
MX2018005272A MX2018005272A MX2018005272A MX2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A MX 2018005272 A MX2018005272 A MX 2018005272A
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MX
Mexico
Prior art keywords
liver
transgene
methods
factor viii
expression cassettes
Prior art date
Application number
MX2018005272A
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English (en)
Inventor
E Riley Brigit
K Lee Gary
Original Assignee
Sangamo Therapeutics Inc
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Publication date
Application filed by Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of MX2018005272A publication Critical patent/MX2018005272A/es

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    • 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
    • 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
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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    • 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
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    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses

Abstract

La presente invención pertenece al ámbito de la terapia génica, particularmente la administración dirigida de construcciones que codifican transgenes al hígado para la expresión de proteínas beneficiosas (terapéuticas). En particular, la invención se refiere al tratamiento de hemofilias, tal como la hemofilia A. La presente invención describe composiciones y métodos para expresar un transgén en una célula hepática. El transgén se puede expresar de forma extracromosómica (episomica) o puede integrarse en el genoma de la célula hepática. En algunas modalidades, el transgén codifica una proteína implicada en la cascada de coagulación. En modalidades preferidas, el transgén codifica un polipéptido FVIII.
MX2018005272A 2015-10-28 2016-07-13 Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos. MX2018005272A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562247469P 2015-10-28 2015-10-28
US201662307897P 2016-03-14 2016-03-14
US201662315438P 2016-03-30 2016-03-30
US201662326229P 2016-04-22 2016-04-22
US201662355106P 2016-06-27 2016-06-27
PCT/US2016/042099 WO2017074526A1 (en) 2015-10-28 2016-07-13 Liver-specific constructs, factor viii expression cassettes and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2018005272A true MX2018005272A (es) 2018-09-18

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MX2018005272A MX2018005272A (es) 2015-10-28 2016-07-13 Construcciones específicas del hígado, casetes de expresión del factor viii y métodos de uso de estos.

Country Status (18)

Country Link
US (4) US10143760B2 (es)
EP (1) EP3368664A4 (es)
JP (2) JP6909212B2 (es)
KR (1) KR20180069054A (es)
CN (2) CN108473976B (es)
AU (1) AU2016343887B2 (es)
BR (1) BR112018008519A2 (es)
CA (1) CA3002524A1 (es)
CO (1) CO2018005215A2 (es)
IL (1) IL258826B2 (es)
MX (1) MX2018005272A (es)
MY (1) MY189674A (es)
PE (1) PE20181206A1 (es)
PH (1) PH12018500927A1 (es)
SA (1) SA518391456B1 (es)
SG (1) SG11201803547RA (es)
WO (1) WO2017074526A1 (es)
ZA (1) ZA201802723B (es)

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