CA3237630A1 - An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders - Google Patents
An improved expression cassette for packaging and expression of variant factor viii for the treatment of hemostasis disorders Download PDFInfo
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- CA3237630A1 CA3237630A1 CA3237630A CA3237630A CA3237630A1 CA 3237630 A1 CA3237630 A1 CA 3237630A1 CA 3237630 A CA3237630 A CA 3237630A CA 3237630 A CA3237630 A CA 3237630A CA 3237630 A1 CA3237630 A1 CA 3237630A1
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- PJVWKTKQMONHTI-UHFFFAOYSA-N warfarin Chemical compound OC=1C2=CC=CC=C2OC(=O)C=1C(CC(=O)C)C1=CC=CC=C1 PJVWKTKQMONHTI-UHFFFAOYSA-N 0.000 description 1
- 229960005080 warfarin Drugs 0.000 description 1
- 238000009736 wetting Methods 0.000 description 1
- 239000000080 wetting agent Substances 0.000 description 1
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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
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- 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
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- 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)
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- Animal Behavior & Ethology (AREA)
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- 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)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Diabetes (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (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)
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| Application Number | Priority Date | Filing Date | Title |
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| US201462036936P | 2014-08-13 | 2014-08-13 | |
| US62/036,936 | 2014-08-13 | ||
| CA2958141A CA2958141C (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 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2958141A Division CA2958141C (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)
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|---|---|
| CA3237630A1 true CA3237630A1 (en) | 2016-02-18 |
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| CA2958141A Active CA2958141C (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 |
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| Application Number | Title | Priority Date | Filing Date |
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| CA2958141A Active CA2958141C (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 |
Country Status (12)
| Country | Link |
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| US (1) | US11014975B2 (enExample) |
| EP (1) | EP3180022B1 (enExample) |
| JP (2) | JP2018506261A (enExample) |
| KR (1) | KR102665348B1 (enExample) |
| CN (1) | CN107427557B (enExample) |
| AU (1) | AU2015301598B2 (enExample) |
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| ES (1) | ES2993915T3 (enExample) |
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| SG (1) | SG11201701033QA (enExample) |
| WO (1) | WO2016025764A2 (enExample) |
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| US10058624B2 (en) * | 2015-04-16 | 2018-08-28 | Emory University | Recombinant promoters and vectors for protein expression in liver and use thereof |
| AU2016260401B2 (en) * | 2015-05-14 | 2022-05-19 | St. Jude Children's Research Hospital, Inc. | Nucleic acid molecules containing spacers and methods of use thereof |
| WO2017066579A1 (en) | 2015-10-14 | 2017-04-20 | 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 |
| CN116949052A (zh) | 2015-11-13 | 2023-10-27 | 武田药品工业株式会社 | 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体 |
| EA036944B1 (ru) | 2015-11-13 | 2021-01-19 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| WO2018102743A1 (en) | 2016-12-02 | 2018-06-07 | Bioverativ Therapeutics Inc. | Methods of treating hemophilic arthropathy using chimeric clotting factors |
| WO2019028192A1 (en) * | 2017-08-01 | 2019-02-07 | Spark Therapeutics, Inc. | METHODS OF GENE THERAPY TARGETING FACTOR VIII (FVIII) |
| US20210093735A1 (en) * | 2018-04-26 | 2021-04-01 | The University Of North Carolina At Chapel Hill | Methods and compositions for treatment of hemophilia |
| RS66972B1 (sr) | 2018-05-18 | 2025-07-31 | Bioverativ Therapeutics Inc | Metode lečenja hemofilije 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 |
| KR20210034013A (ko) | 2018-07-16 | 2021-03-29 | 박스알타 인코퍼레이티드 | 발현이 증가된 재조합 fviii 변이체들을 인코딩하는 바이러스 벡터를 이용한 혈우병의 유전적 치료법 |
| TWI851647B (zh) | 2019-01-16 | 2024-08-11 | 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變異體的病毒載體 |
| EP3736286A1 (en) * | 2019-05-09 | 2020-11-11 | Biotest AG | Single chain factor viii molecule |
| 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 |
| US20250101465A1 (en) * | 2021-05-11 | 2025-03-27 | City Of Hope | Adeno-associated virus compositions and methods of use thereof |
| CN119233998A (zh) * | 2022-07-07 | 2024-12-31 | 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 |
| CN118715025A (zh) * | 2022-10-08 | 2024-09-27 | 凌意(杭州)生物科技有限公司 | 用于增强基因表达的构建体 |
| CN115873099B (zh) * | 2022-12-30 | 2023-10-20 | 福因医药科技(武汉)有限公司 | 一种改造的重组凝血因子viii及其应用 |
| CN116715752B (zh) * | 2023-06-16 | 2024-11-15 | 苏州诺洁贝生物技术有限公司 | 凝血因子fviii蛋白变体、表达载体和用途 |
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| 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 |
| EP2357010B1 (en) * | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| ES2535877T3 (es) * | 2006-06-19 | 2015-05-18 | Asklepios Biopharmaceutical, Inc. | Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica |
| 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 |
| WO2011041770A1 (en) * | 2009-10-02 | 2011-04-07 | The Children's Hospital Of Philadelphia | Compositions and methods for enhancing coagulation factor viii function |
| WO2014008480A2 (en) * | 2012-07-06 | 2014-01-09 | Biogen Idec Ma Inc. | Cell line expressing single chain factor viii polypeptides and uses thereof |
| CA2888931C (en) | 2012-10-26 | 2023-09-05 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
| LT2956477T (lt) * | 2013-02-15 | 2021-02-10 | Bioverativ Therapeutics Inc. | Optimizuotas faktoriaus viii genas |
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2015
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- 2015-08-13 KR KR1020177006818A patent/KR102665348B1/ko active Active
- 2015-08-13 ES ES15832128T patent/ES2993915T3/es active Active
- 2015-08-13 AU AU2015301598A patent/AU2015301598B2/en active Active
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- 2015-08-13 SG SG11201701033QA patent/SG11201701033QA/en unknown
- 2015-08-13 JP JP2017507431A patent/JP2018506261A/ja active Pending
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- 2015-08-13 EP EP15832128.1A patent/EP3180022B1/en active Active
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2017
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2020
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Also Published As
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| EP3180022B1 (en) | 2024-07-03 |
| BR112017002781A2 (pt) | 2017-12-19 |
| JP2021003120A (ja) | 2021-01-14 |
| SG11201701033QA (en) | 2017-03-30 |
| AU2015301598A1 (en) | 2017-03-23 |
| ES2993915T3 (en) | 2025-01-13 |
| WO2016025764A2 (en) | 2016-02-18 |
| WO2016025764A3 (en) | 2016-04-07 |
| CN107427557B (zh) | 2022-01-04 |
| CN107427557A (zh) | 2017-12-01 |
| KR102665348B1 (ko) | 2024-05-16 |
| AU2015301598B2 (en) | 2020-07-16 |
| NZ729107A (en) | 2024-07-26 |
| ZA201701039B (en) | 2021-08-25 |
| JP2018506261A (ja) | 2018-03-08 |
| US20170233456A1 (en) | 2017-08-17 |
| KR20170084012A (ko) | 2017-07-19 |
| US11014975B2 (en) | 2021-05-25 |
| CA2958141C (en) | 2024-06-11 |
| JP7237903B2 (ja) | 2023-03-13 |
| SA517380883B1 (ar) | 2021-12-18 |
| CA2958141A1 (en) | 2016-02-18 |
| EP3180022A2 (en) | 2017-06-21 |
| EP3180022A4 (en) | 2019-02-20 |
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