A Novel 3-D System for Cost-Effective Industrial Production of Pluripotent Cells

描述（申请人提供）：Primorigen Biosciences（R）摘要Primorigen Biosciences将使用第二阶段SBIR资金来推进其用于诱导多能干细胞（iPSC）的稳健扩增的新型三维系统，方法是a）在3L生物反应器中按比例扩大iPSC生产，B）验证生物反应器扩增的iPSC用于分化成具有药物筛选价值的谱系，和c）验证iPSC的大规模冷冻保存。I期研究成功地证明了iPSC在新系统中扩增的可行性，与标准二维培养相比，其额外的好处是大大降低了培养基和劳动力需求。第二阶段的研究将验证系统的更大规模的应用，如上所述。最终的商业目标是通过解决目前对多能干细胞的成本有效的大规模生产的技术和市场需求来实现干细胞及其衍生物的工业化，这是将干细胞用于治疗癌症的关键障碍。 广泛的药物筛选和临床治疗。为了确保这两种应用的实用性，第二阶段研究将继续第一阶段开始的工作，使系统无异种，并适应最终的GMP生产。最初的生产应用将针对需要大量表型相关细胞类型用于药物发现的公司。从长远来看，该系统的范式改变效率和可扩展性将适用于从干细胞中产生临床级治疗细胞。

DESCRIPTION (provided by applicant): Primorigen Biosciences (R) Abstract Primorigen Biosciences will use Phase II SBIR funds to advance its novel, 3-dimensional system for robust expansion of induced pluripotent stem cells (iPSCs) by a) scaling up iPSC production in 3L bioreactors, b) validating the bioreactor-expanded iPSCs for differentiatation into lineages of value for pharmaceutical drug screening, and c) validating large-scale cryopreservation of the iPSCs. Phase I studies successfully demonstrated feasibility of iPSC expansion in the new system, with the added benefits of substantially lowering media and labor requirements versus standard 2-dimensional culture. Phase II studies will validate the system for larger scale applications as described above. The ultimate commercial goals are to enable industrialization of stem cells and their derivatives by addressing the current technical and market need for cost-effective large scale production of pluripotent stem cells, a key barrier to the use stem cells for widespread drug screening and clinical therapeutics. To ensure utility for both applications, Phase II studies will continue the work initiated in Phase I to make the system xeno-free and adapatable for ultimate GMP production. The initial production applications will be targeted to companies requiring large quantities of phenotypically relevant cell types for drug discovery. Longer term, the system's paradigm-changing efficiency and scalability will be adapted to produce clinical-grade therapeutic cells derived from stem-cells.