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

描述(由申请人提供)：Primorigen Biosciences(R)Primorigen Biosciences将利用SBIR第二阶段资金，通过a)在3L生物反应器中扩大诱导多能干细胞(IPSC)的生产，b)验证生物反应器扩展的IPSCs，以分化为药物药物筛选的价值谱系，以及c)验证IPSCs的大规模冷冻保存，从而推进其新颖的三维系统的稳健扩展。第一阶段研究成功地证明了IPSC在新系统中扩展的可行性，与标准的二维文化相比，大大降低了对介质和劳动力的要求。第二阶段研究将验证该系统是否适用于上述较大规模的应用。最终的商业目标是通过满足目前对具有成本效益的大规模生产多能干细胞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 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启动的工作，使该系统不含异物，并适用于最终的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，该系统改变范式的效率和可扩展性将被调整，以生产来自干细胞的临床级治疗细胞。

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.