iPierian presented data from its iPSC research programs at the International Society for Stem Cell Research (ISSCR) 8th Annual Meeting; entitled, “Discovery of disease gene expression signature in cells derived from Spinal Muscular Atrophy (NYSE:SMA) patients”, demonstrating that reprogrammed iPSC lines derived from SMA patients retain a “memory” of the gene expression profile of their parental cells. The relatively low variability of iPSC lines derived from individual patients also suggests that a single iPSC line is sufficient to represent a specific patient in disease modeling experiments.
“This study demonstrates that an epigenetic profile of a patient is retained in reprogrammed cells, which enables the study of patient-specific disease in an in-vitro environment,” said Dr. Michael Venuti, President and CSO of iPierian. “iPSCs offer the potential to study certain conditions in disease-specific human model systems for the first time, and we believe our iPS cell technology will ultimately lead to more effective, targeted therapies for patients worldwide.”
iPierian is the leading biopharmaceutical company focused on the industrialization of induced pluripotent stem cell (iPSC) technology, using cellular reprogramming and directed differentiation of patient cells for the discovery and development of new therapeutics. iPierian’s approach places the patient at the forefront of the drug discovery process in order to reduce drug development time and increase the probability of success for drug candidates by using precise human disease models to develop proprietary small molecule or biologic therapeutics. The initial proprietary therapeutic focus is neuro-degeneration, particularly spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS) and Parkinson’s disease, in addition to a broad therapeutic area program intended for partnering in metabolic disease.