BR112017002781A2 - cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasia - Google Patents
cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasiaInfo
- Publication number
- BR112017002781A2 BR112017002781A2 BR112017002781A BR112017002781A BR112017002781A2 BR 112017002781 A2 BR112017002781 A2 BR 112017002781A2 BR 112017002781 A BR112017002781 A BR 112017002781A BR 112017002781 A BR112017002781 A BR 112017002781A BR 112017002781 A2 BR112017002781 A2 BR 112017002781A2
- Authority
- BR
- Brazil
- Prior art keywords
- factor viii
- packaging
- treatment
- expression
- viii variants
- Prior art date
Links
- 229960000301 factor viii Drugs 0.000 title abstract 6
- 230000023597 hemostasis Effects 0.000 title 1
- 238000004806 packaging method and process Methods 0.000 title 1
- 102000004169 proteins and genes Human genes 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 230000000694 effects Effects 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
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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
- 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
-
- 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
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- 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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Hematology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Diabetes (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
esta invenção divulga variantes do fator viii e métodos de uso dos mesmos. em modalidades particulares, variantes do fator viii são expressas de forma mais eficiente pelas células do que as proteínas fator viii do tipo selvagem, são secretadas em níveis aumentados pelas células do que as proteínas fator viii do tipo selvagem, exibem atividade melhorada em relação às proteínas fator viii do tipo selvagem e são acondicionadas de forma mais eficiente em vetores virais.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201462036936P | 2014-08-13 | 2014-08-13 | |
PCT/US2015/045142 WO2016025764A2 (en) | 2014-08-13 | 2015-08-13 | An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112017002781A2 true BR112017002781A2 (pt) | 2017-12-19 |
Family
ID=55304765
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112017002781A BR112017002781A2 (pt) | 2014-08-13 | 2015-08-13 | cassete de expressão aprimorado para acondicionamento e expressão de variantes do fator viii para o tratamento de distúrbios de hemostasia |
Country Status (11)
Country | Link |
---|---|
US (1) | US11014975B2 (pt) |
EP (1) | EP3180022A4 (pt) |
JP (2) | JP2018506261A (pt) |
CN (1) | CN107427557B (pt) |
AU (1) | AU2015301598B2 (pt) |
BR (1) | BR112017002781A2 (pt) |
CA (2) | CA2958141A1 (pt) |
SA (1) | SA517380883B1 (pt) |
SG (1) | SG11201701033QA (pt) |
WO (1) | WO2016025764A2 (pt) |
ZA (1) | ZA201701039B (pt) |
Families Citing this family (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2016168728A2 (en) * | 2015-04-16 | 2016-10-20 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
EP3294309A4 (en) * | 2015-05-14 | 2019-01-16 | St. Jude Children's Research Hospital, Inc. | NUCLEIC ACID MOLECULES WITH SPACERS AND METHOD FOR USE THEREOF |
AU2016338565B2 (en) | 2015-10-14 | 2021-11-18 | Audentes Therapeutics, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
PE20231949A1 (es) * | 2015-10-30 | 2023-12-05 | Spark Therapeutics Inc | VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA |
BR112018009732A8 (pt) | 2015-11-13 | 2019-02-26 | Baxalta GmbH | ?polinucleotídeo, vetor de vírus adeno-associado, partícula de vírus adeno-associado, célula hospedeira, métodos para produzir uma partícula de vírus adeno-associado, para tratar hemofilia a, para transduzir uma célula hospedeira, e, uso de uma partícula de vírus adeno-associado? |
TW202302627A (zh) | 2015-11-13 | 2023-01-16 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體之病毒載體 |
MX2020001402A (es) * | 2017-08-01 | 2021-01-29 | Spark Therapeutics Inc | Metodos de terapia genica del factor viii (fviii). |
SG11202010767SA (en) | 2018-05-18 | 2020-11-27 | Bioverativ Therapeutics Inc | Methods of treating hemophilia a |
CN108795986A (zh) * | 2018-05-31 | 2018-11-13 | 深圳市免疫基因治疗研究院 | 一种a型血友病慢病毒载体、慢病毒及其制备方法和应用 |
TW202039546A (zh) | 2019-01-16 | 2020-11-01 | 美商巴克斯歐塔公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
CN111218446B (zh) * | 2019-12-25 | 2023-03-28 | 劲帆生物医药科技(武汉)有限公司 | 一种肝脏特异性启动子及其应用 |
WO2022241041A2 (en) * | 2021-05-11 | 2022-11-17 | City Of Hope | Adeno-associated virus compositions and methods of use thereof |
WO2024007978A1 (zh) * | 2022-07-07 | 2024-01-11 | 深圳新诺微环生物科技有限公司 | 一种连接肽、包含连接肽的凝血八因子蛋白或其变体及其用途 |
CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6114148C1 (en) | 1996-09-20 | 2012-05-01 | Gen Hospital Corp | High level expression of proteins |
US6924365B1 (en) * | 1998-09-29 | 2005-08-02 | Transkaryotic Therapies, Inc. | Optimized messenger RNA |
US6221349B1 (en) * | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
EP3085389A1 (en) * | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
EP2037892B1 (en) * | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
US20120028900A1 (en) | 2006-06-30 | 2012-02-02 | Kaufman Randal J | Method of producing factor viii proteins by recombinant methods |
CN101597616A (zh) * | 2008-06-04 | 2009-12-09 | 上海同科生物科技有限公司 | 一种提高基因重组人凝血因子ⅷ表达量的方法 |
AU2013202564B2 (en) | 2008-06-24 | 2015-09-17 | Csl Behring Gmbh | Factor VIII, von Willebrand factor or complexes thereof with prolonged in vivo half-life |
GB0911870D0 (en) * | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
EP2482841B2 (en) | 2009-10-02 | 2022-09-28 | The Children's Hospital Of Philadelphia | Compositions and methods for enhancing coagulation factor viii function |
EP2870250B2 (en) * | 2012-07-06 | 2022-06-29 | Bioverativ Therapeutics Inc. | Cell line expressing single chain factor viii polypeptides and uses thereof |
AU2013336601B2 (en) * | 2012-10-26 | 2018-01-25 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
DK3889173T3 (da) * | 2013-02-15 | 2023-10-09 | Bioverativ Therapeutics Inc | Optimeret faktor viii-gen |
-
2015
- 2015-08-13 WO PCT/US2015/045142 patent/WO2016025764A2/en active Application Filing
- 2015-08-13 BR BR112017002781A patent/BR112017002781A2/pt active Search and Examination
- 2015-08-13 CN CN201580055574.1A patent/CN107427557B/zh active Active
- 2015-08-13 US US15/503,120 patent/US11014975B2/en active Active
- 2015-08-13 AU AU2015301598A patent/AU2015301598B2/en active Active
- 2015-08-13 EP EP15832128.1A patent/EP3180022A4/en active Pending
- 2015-08-13 CA CA2958141A patent/CA2958141A1/en active Pending
- 2015-08-13 SG SG11201701033QA patent/SG11201701033QA/en unknown
- 2015-08-13 CA CA3237630A patent/CA3237630A1/en active Pending
- 2015-08-13 JP JP2017507431A patent/JP2018506261A/ja active Pending
-
2017
- 2017-02-10 ZA ZA2017/01039A patent/ZA201701039B/en unknown
- 2017-02-12 SA SA517380883A patent/SA517380883B1/ar unknown
-
2020
- 2020-09-14 JP JP2020153781A patent/JP7237903B2/ja active Active
Also Published As
Publication number | Publication date |
---|---|
US20170233456A1 (en) | 2017-08-17 |
AU2015301598A1 (en) | 2017-03-23 |
AU2015301598B2 (en) | 2020-07-16 |
JP2018506261A (ja) | 2018-03-08 |
KR20170084012A (ko) | 2017-07-19 |
CN107427557A (zh) | 2017-12-01 |
WO2016025764A2 (en) | 2016-02-18 |
WO2016025764A3 (en) | 2016-04-07 |
SG11201701033QA (en) | 2017-03-30 |
CA3237630A1 (en) | 2016-02-18 |
JP2021003120A (ja) | 2021-01-14 |
ZA201701039B (en) | 2021-08-25 |
EP3180022A4 (en) | 2019-02-20 |
CA2958141A1 (en) | 2016-02-18 |
JP7237903B2 (ja) | 2023-03-13 |
CN107427557B (zh) | 2022-01-04 |
EP3180022A2 (en) | 2017-06-21 |
US11014975B2 (en) | 2021-05-25 |
SA517380883B1 (ar) | 2021-12-18 |
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Legal Events
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B08F | Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette] | ||
B08G | Application fees: restoration [chapter 8.7 patent gazette] | ||
B08F | Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette] |
Free format text: REFERENTE A 4A ANUIDADE. |
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B08G | Application fees: restoration [chapter 8.7 patent gazette] | ||
B07D | Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette] |
Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS. |
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B07E | Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette] | ||
B06U | Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette] | ||
B07A | Application suspended after technical examination (opinion) [chapter 7.1 patent gazette] |