Abstract To date, the existing markets lack a clinically suitable human neuronal cell source or cardiomyocyte source with adequate regenerative potential, which has been the major setback in developing safe and effective cell-based therapies for neurological and heart diseases. Pluripotent human embryonic stem cells (hESC), the nature source of human pluripotent stem cells (hPSC), offer a practically inexhaustible source of replacement cells to heal the damaged or lost tissues that have naturally limited capacity for renewal, such as the human brain and the human heart. Neurological and heart diseases incur exorbitant costs on the healthcare system worldwide; therefore, there is a strong focus on translating hESC research to provide newer and more efficient solutions for these therapeutic needs. However, a major bottleneck for clinical translation of hESC research is to enable a well-controlled efficient induction of hPSC to a particular clinically relevant lineage. Emerging hPSC-based PluriXcel technology platforms enable large scale production of high quality clinical-grade hPSC lines and direct conversion of such non-functional pluripotent cells by small molecule induction into a large commercial scale of functional human neuronal or cardiomyocyte derivatives, which not only constitutes clinically representative progresses in both human neuronal and cardiac therapeutic products for treating a wide range of incurable or hitherto untreatable neurological and heart diseases, but also offer manufacturing innovations for production scale up and creation of replacement tissue/ organ products. PluriXcel technology provides scalable platforms to ensure a high degree of efficacy and safety of hESC-derived cellular products, thus robust clinical benefits leading to therapies, and for cGMP manufacturing of large quantities of high quality clinical grade hESC-based cell therapy products to support clinical trials. PluriXcel technology platforms introduce stem cell treatment development and manufacturing innovations to achieve clinically-useful and therapeutically-viable levels of safety and efficacy in cell therapy product development, providing unique clinical-grade translating capacities for moving stem cell research from current studies in animals into human trials.
Keywords: regenerative medicine; stem cell technology; human embryonic stem cell; pluripotent; neuronal progenitor; cardiac precursor; cardiomyocyte; cell therapy; stem cell treatment; neurological disease; heart disease