Bioengineered Recombinant FVIII
生物工程重组 FVIII
基本信息
- 批准号:8796739
- 负责人:
- 金额:$ 67.36万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2013
- 资助国家:美国
- 起止时间:2013-02-01 至 2017-01-31
- 项目状态:已结题
- 来源:
- 关键词:AdolescenceAnabolismAnimal ModelBiochemicalBiological ProductsBiomanufacturingBiomedical EngineeringBioreactorsBlood Coagulation FactorBreathingCapitalCell Culture TechniquesCell LineCellsClinicalComplexDevelopmentDevelopment PlansDiseaseDocumentationDoseEndoplasmic ReticulumEnsureFactor VIIIFamily suidaeFeedbackFermentationGlycoproteinsGoalsGolgi ApparatusGrowthHIVHalf-LifeHamstersHealthHealthcareHemophilia AHumanIntellectual PropertyInvestigational DrugsInvestigational New Drug ApplicationKidneyKilogramLegal patentM cellMammalian CellMarketingMessenger RNAMethodsModelingPerformancePersonsPharmaceutical PreparationsPharmacologyPharmacology and ToxicologyPharmacy facilityPhasePlasmaPost-Translational Protein ProcessingPreparationPriceProcessProductionProductivityPropertyProteinsRecombinant ProteinsRecombinantsRecordsRecoveryReference StandardsRegulatory AffairsReplacement TherapyRodentRunningSafetySerumSerum-Free Culture MediaSmall Business Innovation Research GrantSuspension substanceSuspensionsSystemTechnologyTechnology TransferTestingTherapeuticToxic effectTransgenesVirusbasebiophysical techniquescommercializationcostcost effectivedensitydesignexperiencehuman F8 proteinimprovedinnovationmanufacturing processmanufacturing process developmentmeetingsmortalitynoveloperationpre-clinicalpreclinical safetyrecombinant antihemophilic factor VIIIresponsescale uptechnology development
项目摘要
DESCRIPTION (provided by applicant): The manufacturing of recombinant coagulation factor VIII is a complex, labor and capital intensive endeavor. Of the recombinant proteins currently marketed, coagulation factor VIII is manufactured with the lowest efficiency and is by far the most expensive on a per unit mass basis with a pharmacy price of $10,000,000 per gram. The goal of the current application is to develop a product technology and a manufacturing process that can improve the production volume and pharmacy price of factor VIII biologics. Expression Therapeutics has developed and patented two technologies, a bioengineered factor VIII transgene that demonstrates 100-fold greater expression than standard human factor VIII, and a high performance baby hamster kidney-derived (BHK) cell line, designated BHK-MS, that can be cultured and expanded indefinitely and at high density in serum-free medium under suspension bioreactor conditions. In the current application, we propose to complete pre-clinical manufacturing, product characterization and pharmacology and toxicity studies necessary to submit an Investigational New Drug application.
描述(由申请人提供):重组凝血因子VIII的生产是一项复杂的劳动力和资本密集型奋进。在目前市售的重组蛋白中,凝血因子VIII的生产效率最低,并且在每单位质量的基础上是迄今为止最昂贵的,每克的制药价格为10,000,000美元。本申请的目标是开发能够提高因子VIII生物制剂的生产量和制药价格的产品技术和生产工艺。Expression Therapeutics已开发并获得两项技术的专利,一种是经生物工程改造的因子VIII转基因,其表达量是标准人因子VIII的100倍,另一种是高性能幼仓鼠肾衍生(BHK)细胞系,命名为BHK-MS,该细胞系可在悬浮生物反应器条件下在无血清培养基中以高密度无限培养和扩增。在当前申请中,我们建议完成提交研究性新药申请所需的临床前生产、产品表征以及药理学和毒性研究。
项目成果
期刊论文数量(0)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
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Gabriela Denning其他文献
Gabriela Denning的其他文献
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{{ truncateString('Gabriela Denning', 18)}}的其他基金
Manufacturing Process Development for Bioengineered FVIII
生物工程 FVIII 的制造工艺开发
- 批准号:
8454386 - 财政年份:2013
- 资助金额:
$ 67.36万 - 项目类别:
Development of a novel production platform for complex recombinant biotherapeutic
开发复杂重组生物治疗的新型生产平台
- 批准号:
8251701 - 财政年份:2012
- 资助金额:
$ 67.36万 - 项目类别:
Bioengineered factor VIII gene therapy for hemophilia A
针对血友病 A 的生物工程因子 VIII 基因治疗
- 批准号:
8313425 - 财政年份:2012
- 资助金额:
$ 67.36万 - 项目类别:
Clinical Testing of the First Suspension BHK-M Cell Platform Derived Biotherapeutic
第一个悬浮 BHK-M 细胞平台衍生的生物治疗药物的临床测试
- 批准号:
9759962 - 财政年份:2012
- 资助金额:
$ 67.36万 - 项目类别:
Bioengineered factor VIII gene therapy for hemophilia A
针对血友病 A 的生物工程因子 VIII 基因治疗
- 批准号:
8714274 - 财政年份:2012
- 资助金额:
$ 67.36万 - 项目类别:
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