PE20231949A1 - 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

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Publication number
PE20231949A1
PE20231949A1 PE2023001716A PE2023001716A PE20231949A1 PE 20231949 A1 PE20231949 A1 PE 20231949A1 PE 2023001716 A PE2023001716 A PE 2023001716A PE 2023001716 A PE2023001716 A PE 2023001716A PE 20231949 A1 PE20231949 A1 PE 20231949A1
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PE
Peru
Prior art keywords
factor viii
treatment
seq
acid variant
hemostasy
Prior art date
Application number
PE2023001716A
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English (en)
Inventor
Xavier Anguela
Sam Hsien-I Shen
Original Assignee
Spark Therapeutics Inc
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Publication date
Application filed by Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of PE20231949A1 publication Critical patent/PE20231949A1/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
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    • 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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    • 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
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    • 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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    • C12N15/85—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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    • 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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    • C12N2750/14011—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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Abstract

La presente invencion se refiere a una particula de AAV recombinante (rAAV) que comprende un genoma del vector AAV que comprende 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 comprende la SEC ID NO: 1 a 18, entre otros, y en donde el rAAV comprende proteina de la capside de AAV VP1, tal como la capside LK03 de SEC ID NO: 27 o SPK de SEC ID NO: 28, VP2 y VP3. Tambien se refiere a una composicion farmaceutica y a una celula huesped. La variante de acido nucleico proporciona una transcripcion y expresion incrementadas, exhibiendo una actividad mejorada sobre proteinas de Factor VIII de tipo natural, siendo util en el tratamiento de la hemofilia A.
PE2023001716A 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 PE20231949A1 (es)

Applications Claiming Priority (5)

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
PCT/US2016/059793 WO2017075619A1 (en) 2015-10-30 2016-10-31 Cpg reduced factor viii variants, compositions and methods and uses for treatment of hemostasis disorders

Publications (1)

Publication Number Publication Date
PE20231949A1 true PE20231949A1 (es) 2023-12-05

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ID=58630834

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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
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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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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US (2) US20180312571A1 (es)
EP (1) EP3368560A4 (es)
JP (2) 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) IL258959A (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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KR101952102B1 (ko) * 2017-12-07 2019-02-26 주식회사 지앤피바이오사이언스 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터
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JP2022137029A (ja) 2022-09-21
PH12018501055A1 (en) 2019-01-28
CN108368164A (zh) 2018-08-03
AU2016344030B2 (en) 2021-05-06
AU2016344030A1 (en) 2018-05-10
EP3368560A4 (en) 2019-03-27
SG11201802972UA (en) 2018-05-30
EP3368560A1 (en) 2018-09-05
SA518391463B1 (ar) 2022-12-13
KR20180090795A (ko) 2018-08-13
JP2019503649A (ja) 2019-02-14
US20180312571A1 (en) 2018-11-01
RU2018119710A (ru) 2019-12-06
CL2018001107A1 (es) 2018-10-05
PE20181168A1 (es) 2018-07-19
MX2018005490A (es) 2018-08-16
RU2018119710A3 (es) 2020-04-02
WO2017075619A1 (en) 2017-05-04
RU2745506C2 (ru) 2021-03-25
CO2018005306A2 (es) 2018-11-30
MY190926A (en) 2022-05-20
IL258959A (en) 2018-06-28
BR112018008766A2 (pt) 2018-10-30
US20170216408A1 (en) 2017-08-03
MX2023001600A (es) 2023-03-09
JP7088831B2 (ja) 2022-06-21
US11168124B2 (en) 2021-11-09
CA3002689A1 (en) 2017-05-04

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