SCIENCE + TECHNOLOGY

CoreX™

Correcting Genetic Defects

CoreX is a suite of biology technologies that allow the creation of exquisitely sensitive assays mimicking the disease state, enabling discovery of compounds that correct the mutational defect.

There are several mechanisms by which a small molecule can correct a genetic defect, as shown in the table on the left. All of these mechanisms have been demonstrated for small molecules, and all are available as CoreX assays.

ER → PM

CF

Endocytosis

FTD/AD

Recycling

CF

Lysosome

PD

Multiplex

FTD

CoreX allows creation of assays to probe each of these mechanisms, and we may take more than one approach to a given disease.

The CoreX suite includes patented biosensor technology licensed from the founders’ labs at Carnegie Mellon University along with proprietary assay methods developed at Sharp. A key aspect of CoreX is that the assays have the sensitivity and signal-to-noise that makes large scale high-throughput compound screening possible.