BR112018008766A2 - variantes, composições e métodos e utilizações do fator viii reduzido cpg para o tratamento de transtornos hemostásicos - Google Patents

variantes, composições e métodos e utilizações do fator viii reduzido cpg para o tratamento de transtornos hemostásicos

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Publication number
BR112018008766A2
BR112018008766A2 BR112018008766A BR112018008766A BR112018008766A2 BR 112018008766 A2 BR112018008766 A2 BR 112018008766A2 BR 112018008766 A BR112018008766 A BR 112018008766A BR 112018008766 A BR112018008766 A BR 112018008766A BR 112018008766 A2 BR112018008766 A2 BR 112018008766A2
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Brazil
Prior art keywords
cpg
factor viii
variants
compositions
treatment
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BR112018008766A
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English (en)
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Hsien-I Shen Sam
Anguela Xavier
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Spark Therapeutics Inc
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Publication date
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Publication of BR112018008766A2 publication Critical patent/BR112018008766A2/pt

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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
    • C07K14/435—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
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    • 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
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    • 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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/86—Viral vectors
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    • C12P21/00—Preparation of peptides or proteins
    • 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
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    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
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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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  • Health & Medical Sciences (AREA)
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Abstract

as variantes de ácido nucleico reduzidas em cpg que codificam a proteína fviii e os modos de uso da mesma são divulgados. em modalidades particulares, variantes de ácidos nucleicos reduzidos com cpg que codificam para fviii s expressas mais eficientemente por culas, são segregadas em níveis aumentados por culas sobre proteínas do fator viii de tipo selvagem, exibem expressão e/ou atividade melhoradas sobre proteínas do fator viii do tipo selvagem ou s embaladas. mais eficientemente em vetores virais.
BR112018008766A 2015-10-30 2016-10-31 variantes, composições e métodos e utilizações do fator viii reduzido cpg para o tratamento de transtornos hemostásicos BR112018008766A2 (pt)

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)

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BR112018008766A2 true BR112018008766A2 (pt) 2018-10-30

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Country Status (19)

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US (2) US20180312571A1 (pt)
EP (1) EP3368560A4 (pt)
JP (2) JP7088831B2 (pt)
KR (1) KR20180090795A (pt)
CN (1) CN108368164A (pt)
AU (1) AU2016344030B2 (pt)
BR (1) BR112018008766A2 (pt)
CA (1) CA3002689A1 (pt)
CL (1) CL2018001107A1 (pt)
CO (1) CO2018005306A2 (pt)
IL (1) IL258959B1 (pt)
MX (2) MX2018005490A (pt)
MY (1) MY190926A (pt)
PE (2) PE20231949A1 (pt)
PH (1) PH12018501055A1 (pt)
RU (1) RU2745506C2 (pt)
SA (1) SA518391463B1 (pt)
SG (1) SG11201802972UA (pt)
WO (1) WO2017075619A1 (pt)

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KR20230161535A (ko) 2016-07-26 2023-11-27 바이오마린 파머수티컬 인크. 신규 아데노-관련 바이러스 캡시드 단백질
BR112020001979A2 (pt) * 2017-08-01 2020-08-18 Spark Therapeutics, Inc. métodos de terapia genética do fator viii (fviii)
KR20200035130A (ko) * 2017-08-09 2020-04-01 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
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JP2021522811A (ja) 2018-05-09 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド フェニルケトン尿症の治療方法
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WO2023211316A1 (en) * 2022-04-28 2023-11-02 Joint Stock Company "Biocad" Codon-optimized nucleic acid encoding the fviii-bdd
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PE20181168A1 (es) 2018-07-19
JP7088831B2 (ja) 2022-06-21
CO2018005306A2 (es) 2018-11-30
RU2018119710A (ru) 2019-12-06
KR20180090795A (ko) 2018-08-13
IL258959A (en) 2018-06-28
AU2016344030B2 (en) 2021-05-06
MX2023001600A (es) 2023-03-09
CA3002689A1 (en) 2017-05-04
US20180312571A1 (en) 2018-11-01
SG11201802972UA (en) 2018-05-30
SA518391463B1 (ar) 2022-12-13
EP3368560A1 (en) 2018-09-05
JP2019503649A (ja) 2019-02-14
PE20231949A1 (es) 2023-12-05
IL258959B1 (en) 2024-06-01
CL2018001107A1 (es) 2018-10-05
RU2745506C2 (ru) 2021-03-25
JP2022137029A (ja) 2022-09-21
US20170216408A1 (en) 2017-08-03
MX2018005490A (es) 2018-08-16
EP3368560A4 (en) 2019-03-27
US11168124B2 (en) 2021-11-09
PH12018501055A1 (en) 2019-01-28
AU2016344030A1 (en) 2018-05-10
MY190926A (en) 2022-05-20
WO2017075619A1 (en) 2017-05-04
RU2018119710A3 (pt) 2020-04-02
CN108368164A (zh) 2018-08-03

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