CRISPR Cas in drug discovery: precision medicine meets precision (in vitro) models

Today, therapeutic discovery encounters new challenges that demand innovative solutions. CRISPR Cas has revolutionized the speed and accuracy of rewriting genetic information. It finds its application in gene therapy but also serves as a precise tool for validating drug targets identified by omics, thus fueling today’s drug discovery pipelines. For instance, single-cell transcriptomics have provided unprecedented insights into pathways and altered cell states in cystic fibrosis (CF) lungs. Yet, achieving a matching high-throughput and single-cell resolution at the functional level remains elusive. Indeed, CFTR expression has been shown to be diverse across airway epithelial cell types, while the functional consequences are not fully understood. Nevertheless, these insights are critical for tailoring precision medicine strategies, such as gene therapy, to the appropriate cell in the lung.

Precision disease models are essential to translate precision medicine strategies into the therapies of the future. By integrating omics and scalable CRISPR Cas gene editing in patient derived tissues, we can create beyond state of the art precision disease models, enabling innovative drug discovery, which is particularly crucial in the field of complex and chronic diseases.