Innovative Human Embryonic Stem Cell Technology Platforms --- PluriXcel
Provide proprietary clinical-grade translating capabilities to address key challenges to traditional medicine & biofabrication, and offers currently the only available human cell sources in large quantity and high quality with adequate cellular capacity to regenerate the contractile heart muscle and the neuron circuitry, overcoming major bottlenecks for tissue repair & biofabrication, including: PluriXcel-DCS (defined culture system) Platform; PluriXcel-SMI (small molecule induction)-Heart Platform; PluriXcel-SMI-Neuron Platform. These cutting-edge hESC technology platforms support hESC-based stem cell therapy product downstream development, including development and large-scale production of clinical-grade scalable CNS- and heart-related human stem/progenitor/precursor cells and functional human neuronal and heart-muscle cell/tissue/organ products to support clinical trials of neuronal/cardiomyocyte regeneration or replacement therapies as well as CNS/heart tissue/organ biofabrication.
Creation of clinical-grade human stem cell lines/products/banks: providing defined culture systems (DCS) for well-controlled efficient derivation, maintenance, and differentiation of high quality clinical-grade stable hESC lines, and for creating cGMP-compatible/quality clinical-grade master/working cell banks (MCB/WCB) & tool boxes.
Scale-up production of high quality contractile human heart muscle cells at a commercial quantity necessary for heart tissue biofabrication and heart regenerative therapies: providing scale-up cGMP manufacturing capability for high efficient direct conversion of non-functional pluripotent hESC into a large commercial scale of high quality clinical-grade functional human heart cell therapy products by small molecule induction, adequate for development of safe and effective stem cell therapies for heart muscle regeneration and repair, and for creation of replacement heart tissue/organ products or models targeting heart disease and failure.
Scale-up production of high quality human neurons at a commercial quantity necessary for CNS tissue biofabrication and neuronal regenerative therapies: providing scale-up cGMP manufacturing capability for high efficient direct conversion of non-functional pluripotent hESC into a large commercial scale of high quality clinical-grade functional human neuronal cell therapy products by small molecule induction, adequate for development of safe and effective stem cell therapies for a wide range of neurological disorders, and for creation of CNS-related replacement tissue/organ products or models targeting neurological disorders.
Human Embryonic Stem Cell (hESC)-based PluriXcel Platforms Focus on Developing Regenerative Medicine Advanced Therapy (RMAT) Targeting Serious or Life-threatening Diseases and Conditions in Unmet Medical Needs that have been Big Challenges for Traditional Medicine
Traditional drug development usually starts with drug leads discovered in non-human simple model organisms, thus requires lengthy and costly both demonstration in animal model testing and establishment of proof-of-concept and safety in human trials. As a result, millions of drug leads have vanished before even reach clinical trials, and for few lucky ones, have encountered the very high drug failure rate in human trials. Among those very few drugs that eventually obtained their market approvals, there were not any cures, or even meaningfully effective treatments, for Parkinson’s disease, Alzheimer disease, stroke, spinal cord and brain injuries, heart disease and failure, or a host of other disorders that destroy lives. Unlike traditional R&D, hESC-based RMAT products have been developed directly with human cells with proof-of-concept already established in humans, which simplifies the development process, lowers the costs, shortens the time consumption, and increases the probability of clinical success dramatically. PluriXcel technology platforms are designed and developed specifically to address practical issues essential for designating any human stem cell as a safe and effective cell therapy product for filing IND and entry into human trials, including the benefits in high efficiency, stability, low tumor risk, high purity, high efficacy in repair, safety, as well as stem cell production at large scale, over all other existing stem cell sources or products. Patents and exclusive rights have been secured to access the key translating technologies and capabilities in the regenerative medicine market, which allow derive and establish therapeutically viable and clinically useful high quality hESC-derived human cell therapy products/tools/banks, design and execute a cost-effective cell therapy product development and manufacturing strategy, and implement cGMP-compatible/quality manufacturing processes for production scale-up and creation of replacement cell/tissue/organ products.