Synopsis "PluriXcel: Emerging Technologies of Regenerative Medicine (in English)"
The limit capacity of heart muscle and brain cells for self-repair constitutes a significant challenge to traditional medicine for tissue and function restoration in seeking cures for a wide range of heart diseases and neurological disorders. Given their limited capacity for self-repair, cell-based therapy represents a promising therapeutic approach closest to provide a cure to restore normal tissue and function. However, the existing markets lack a scalable clinically-suitable human neuronal or heart cell source with adequate regenerative potential, which has been the major setback in developing safe and effective cell-based therapies. To date, the need to restore vital tissue and function for a wide range of neurological and heart diseases remains a daunting challenge to the conventional mode of drugs and treatments. The pluripotent human embryonic stem cells (hESC), the nature source of human pluripotent stem cells (hPSC), have unlimited expansion and differentiation capabilities, offering a practically inexhaustible source of replacement cells for tissue and function restoration. Therefore, they have been regarded as an ideal source to provide an unlimited supply of clinically-relevant functional human cells to heal the damaged or lost tissues that have naturally limited capacity for self-repair, such as the human brain and heart. As neurological and heart diseases incur exorbitant costs on the healthcare system worldwide, there is a strong focus on translating hPSC research to provide newer, more efficient solutions for these unmet therapeutic needs. However, a persistent challenge for clinical translation is to enable a well-controlled and efficient induction of non-functional hPSC exclusively and uniformly to a specific clinically relevant functional lineage. PluriXcel is a pioneer in stem cell therapeutics and remarkable advancement in stem cell research related to the differentiation of non-functional hPSC into specific functional lineages by small molecule induction. The PluriXcel technology platforms offer currently the only available human cell products with the pharmacological capacity to regenerate neurons and contractile heart muscles that allow restitution of function in the clinic. PluriXcel technological breakthroughs allow the achievement of a highly efficient direct conversion of clinical-grade hPSC into a large supply of high-purity human neuronal cells or heart muscle cells with adequate capacity to regenerate neurons or contractile heart muscles for cell regeneration or replacement therapies, as well as for tissue or organ biofabrication. The PluriXcel platforms not only constitute 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 human tissue and organ products. Medical innovations of PluriXcel technology provide scalable platforms to ensure a high degree of efficacy and safety of hPSC-derived cellular products, thus robust clinical benefits leading to therapies, for treating major human diseases challenging traditional medicine. Manufacturing innovations of PluriXcel technology provide scale-up cGMP manufacturing capability for production of large quantities of high quality clinical-grade hPSC-based cell therapy products to support clinical trials and tissue or organ engineering/biofabrication, improving the availability, reproducibility, accessibility, and standardization of manufacturing materials, technologies, and processes to create human repairing or replacing cell, tissue, and organ products. Medical and manufacturing innovations of PluriXcel technology provide life scientists and clinicians with novel, efficient, and powerful resources and tools to address major health concerns, which will shape the future of medicine and bring new therapeutics into the market.
