You have worked tirelessly to discover a set of lead compounds, design your vectors and delivery systems, or engineer your target cell type. The next step in your R&D is pre-clinical, large animal studies. But there's a catch. None of the in-vitro models you have are species-specific. You don't have physiologically relevant data to indicate if your candidate will, or won't, have any off target effects in your large animal study. In essence - you're flying blind, and are about to commit months to years and hundreds of thousands to millions of dollars based on you and your advisors best guesses. Is there a better way? We think so.
That's why have begun developing large-animal pluripotent stem cell (iPSC) lines and kits to generate terminally differentiated cell types. You should have a species specific cell line model to rapidly investigate if your asset causes off target effects. The benefits are threefold. First, you can generate data in weeks, not months, to add to your toxicology portfolio. Second, this data can be fed back into your discovery engine to help develop assets that will clear large animal testing. Finally, species specific cell line models have the potential to help you select early stage assets that act on mechanisms (or don't have off target effects) in physiologies that are closer in kind to your end target: the patients and human beings you're working so hard to help.
