A major goal in the still nascent human stem cell field is to utilize improved cell-based assays in the service of small-molecule therapeutics discovery and virtual early-phase clinical trials. Ajamete Kaykas, from the Novartis Institute for BioMedical Research, discussed the pharmaceutical pipeline to identify phenotypes in human pluripotent stem cell (hPSC)-derived neurons. He demonstrated that in a non-academic setting, it is possible to establish a library of more than 100 transgene-free hiPSCs as well as a clustered regularly interspaced short palindromic repeats (CRISPR) pipeline to create and screen clones for indels, knockout, and point mutation via high-throughput sequencing methods. In parallel, his group has tested the feasibility of scaling both directed differentiation as well as NGN2-induction protocols into 384-well plate format for high-throughput screening. Overall, both a robust hPSC pipeline for hiPSC-based modeling as well as standardized and automated differentiation methods are being established at Novartis, in collaboration with the Stanley Center, for the characterization and drug screening of SZ patients.
