Abstract

Genome Editing as an Emerging Technology to Functionalize the Human Genome and Accelerate Targeted Drug Discovery

Arumugham Raghunathan, Sr. Director, Horizon Discovery Ltd

The ability to rapidly, accurately and stably engineer human genomes without introducing confounding off-target effects promises to transform the pursuit of novel targeted, or personalized medicines. Towards this aim, Horizon creates cell line disease models with modified genes at their endogenous locus (based on rAAV, ZFN and CRISPR technologies) and has built a large suite of genetically-defined and patient-relevant X-MAN™ (gene-X, Mutant And Normal) human isogenic cell line pairs, that include the introduction of subtle point mutations and SNPs, just as they occur in real patients. These disease models are now being used by many industrial and academic researchers to elucidate novel targets and biomarkers and are directing drugs towards specific patient populations.