CGMP Compliant Closed Cell Culture System for Reproducible De-differentiation of human somatic cells into iPSCs

CGMP合规封闭式细胞培养系统用于人的可再生去分化 体细胞转化为iPSC 摘要 基于iPSC的个性化细胞疗法的进展目前受到医学领域的挑战的阻碍。 治疗性细胞的生物制造。尽管有一些方法使衍生、增长和 虽然iPSC的分化更有效，但重编程功效仍存在显著的变异性， 基因组完整性和源自患者组织样品的iPSC的发育潜力。这些 变异性包括批次依赖性或技术人员依赖性分化效率、细菌或真菌 污染风险、电池维护期间的CO2或O2浓度水平压力、高成本或交叉污染风险。 集中式生物制造设施的污染风险和cGMP标准或监管要求 合规同一来源iPSCs体外生长特性的差异 并且它们不能再分化成所需的组织类型将在治疗中引起严重的问题。的 基于iPSC的个性化医疗的进一步发展目前受到难以产生iPSC的限制 以可承受的成本为大量人口提供服务。因此，Biopico Systems Inc将通过以下方式解决这些挑战： 开发符合cGMP的自动化封闭细胞培养系统，用于可重复的去分化 转化为iPSCs。为了使Biopico的“CellsMX”系统商业化， 培养基交换系统和定制mRNA/培养基配制前端的集成， 将在本II期研究中进行重编程。CellsMX系统将提供高质量 向客户保证根据cGMP指导方针进行大规模生产，因为运营许可证已颁发给 生物实体沿着细胞如何产生、测试和释放用于治疗用途。此外，甚至， 如果大量患者需要基于iPSC的个性化细胞疗法，在CellsMX封闭系统下， 患者细胞不会交叉污染，并且该系统可以部署在护理点，避免高风险。 与运输、物流、跟踪和记录相关的成本和风险。虽然患者特异性 iPSC策略减少免疫刺激将推动CellsMX的初始细分市场 Biopico将为几种此类治疗性培养工艺开发一套产品。

CGMP Compliant Closed Cell Culture System for Reproducible De-differentiation of human somatic cells into iPSCs Abstract The advancement of iPSC-based personalized cell therapies is currently hindered by the challenges in the biomanufacturing of therapeutic cells. Despite approaches that have made the derivation, growth and differentiation of iPSCs more efficient, there remains significant variability in reprogramming efficacy, genomic integrity and developmental potential of iPSCs derived from patient tissue samples. These variabilities include lot-dependent or technician-dependent differentiation efficiency, bacterial or fungal contamination risks, CO2 or O2 concentration level stresses during cell maintenance, high costs or cross- contamination risks with centralized biomanufacturing facility and requirement of cGMP criteria or regulatory compliance. The difference of iPSCs derived from the same sample in their in-vitro growth characteristics and their inability to re-differentiate into the desired tissue type will cause serious problems in therapy. The further advance of iPSC-based personalized medicine is currently limited by the difficulty to generate iPSCs for large populations and at an affordable cost. Therefore Biopico Systems Inc will solve such challenges by developing an automated cGMP Compliant Closed Cell Culture System for reproducible de-differentiation of human somatic cells into iPSCs. To commercialize Biopico's “CellsMX” system, optimization of closed media exchange system and integration of customized mRNA/ media formulation front-end for reprogramming will be performed in this Phase II research. The CellsMX system will provide quality assurance to the customers for mass production under cGMP guidelines, as operating license are issued to biological entities along with how cells are produced, tested, and released for therapeutic use. Further, even, if a large number of patients need iPSC-based personalized cell therapies, under CellsMX closed system, patient cells are not cross-contaminated and the system can be deployed at the point of care avoiding high costs and risks associated with the transportation, logistics, tracking, and recording. While patient-specific iPSC strategy's reduction of immunologic stimulus will drive the initial market segment for the CellsMX system, Biopico will develop a suite of products for several of such therapeutic culture processes.