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PE20181168A1 - VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA - Google Patents

VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA

Info

Publication number
PE20181168A1
PE20181168A1 PE2018000713A PE2018000713A PE20181168A1 PE 20181168 A1 PE20181168 A1 PE 20181168A1 PE 2018000713 A PE2018000713 A PE 2018000713A PE 2018000713 A PE2018000713 A PE 2018000713A PE 20181168 A1 PE20181168 A1 PE 20181168A1
Authority
PE
Peru
Prior art keywords
vector
nucleic acid
hemostasia
cpg
treatment
Prior art date
Application number
PE2018000713A
Other languages
English (en)
Inventor
Xavier Anguela
Sam Hsien-I Shen
Original Assignee
Spark Therapeutics Inc
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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of PE20181168A1 publication Critical patent/PE20181168A1/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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
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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
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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
    • C12N2750/14145—Special targeting system for viral vectors

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Abstract

SE REFIERE A UNA VARIANTE DE ACIDO NUCLEICO QUE CODIFICA EL FACTOR VIII (FVIII) QUE TIENE UNA DELECION DEL DOMINIO B, EN DONDE LA VARIANTE DE ACIDO NUCLEICO TIENE UN 92% A 100% DE IDENTIDAD CON LA SEC ID NO: 7; DONDE DICHA VARIANTE DE ACIDO NUCLEICO TIENE 20 O MENOS DINUCLEOTIDOS DE CITOSINA-GUANINA (CpG). TAMBIEN SE REFIERE A UN VECTOR DE EXPRESION SELECCIONADO DEL GRUPO QUE CONSISTE EN UN VECTOR DE VIRUS ASOCIADO A ADENOVIRUS (AAV), UN VECTOR RETROVIRAL, UN VECTOR ADENOVIRICO, UN PLASMIDO O UN VECTOR LENTIVIRAL; A UNA CELULA HUESPED, Y UNA COMPOSICION FARMACEUTICA QUE COMPRENDE DICHA VARIANTE DE ACIDO NUCLEICO, LA CUAL ES UTIL PARA EL TRATAMIENTO DE TRASTORNOS ASOCIADOS CON LA HEMOSTASIA ABERRANTE
PE2018000713A 2015-10-30 2016-10-31 VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA PE20181168A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562249001P 2015-10-30 2015-10-30
US201662331872P 2016-05-04 2016-05-04
US201662349532P 2016-06-13 2016-06-13
US201662357874P 2016-07-01 2016-07-01

Publications (1)

Publication Number Publication Date
PE20181168A1 true PE20181168A1 (es) 2018-07-19

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Application Number Title Priority Date Filing Date
PE2023001716A PE20231949A1 (es) 2015-10-30 2016-10-30 VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
PE2018000713A PE20181168A1 (es) 2015-10-30 2016-10-31 VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA

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PE2023001716A PE20231949A1 (es) 2015-10-30 2016-10-30 VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA

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US (3) US20180312571A1 (es)
EP (1) EP3368560A4 (es)
JP (3) JP7088831B2 (es)
KR (1) KR20180090795A (es)
CN (1) CN108368164A (es)
AU (1) AU2016344030B2 (es)
BR (1) BR112018008766A2 (es)
CA (1) CA3002689A1 (es)
CL (1) CL2018001107A1 (es)
CO (1) CO2018005306A2 (es)
IL (1) IL258959B2 (es)
MX (2) MX2018005490A (es)
MY (1) MY190926A (es)
PE (2) PE20231949A1 (es)
PH (1) PH12018501055A1 (es)
RU (1) RU2745506C2 (es)
SA (1) SA518391463B1 (es)
SG (1) SG11201802972UA (es)
WO (1) WO2017075619A1 (es)

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