Abstract

Experimental validation, expert knowledge, and serendipity have led to the identification of transcription factor (TF) cocktails with the power to drive the conversion of one cell type to another, including reprogramming of somatic cell types from pluripotent stem cells or directly to other somatic cell types (transdifferentiation). The MOGRIFY® platform presents a unique opportunity to collapse discovery timelines and enable novel therapeutic modalities by deploying a combination of data-driven in silico prediction and in vitro validation to identify optimal combinations of TFs for direct cell reprogramming. Here we’ll discuss its navigation of an almost infinite combinatorial search space, currently curbing the generation of therapeutically valuable cell types, and how we are leveraging the MOGRIFY technoloty to transform the lives of patients with degenerative diseases through a revolutionary class of in vivo reprogramming therapies.