Toward clinical trial in a dish: harnessing iPSC models in drug discovery

The advent of human induced pluripotent stem cell (iPSC) technology has revolutionized the development of disease modeling. Following the recent FDA announcement to reduce animal testing, the scientific research focus is shifting toward the use of these relevant in vitro models. iPSCs enable the generation of relevant cell models derived directly from human donors, providing a robust drug discovery platform capable of assessing complex human diseases phenotypes in a scalable manner.
This presentation highlights the development of a comprehensive platform to recapitulate an in vitro model of muscular diseases, such as dystrophies and myopathies, and neuromuscular disorders using iPSC-derived ioSkeletal Myocytes. The platform integrates gene expression, population marker evaluation via image-based High-Content analysis, calcium-based and contractility functional readouts, all optimized in miniaturized formats and suitable for drug discovery screening applications. 
Integrating these readouts into early-stage drug discovery pipelines accelerates the identification and functional validation of candidate compounds, bridging the gap between bench research and clinical application to expedite the development of effective therapies for muscular and neuromuscular diseases.