Retinitis pigmentosa (RP) is a group of genetic (Mendelian) rare eye diseases that affect the retina (the light-sensitive layer of tissue in the back of the eye). RP makes cells in the retina break down slowly over time, causing vision loss.

In a strategic R&D partnership with EyeStem, CrisprBits aims use the CRISPR editing technology to correct a single mutation in the RDH12 gene in iPSC cells derived from a patient. The mutation causes a truncated key protein RDH12 which would be corrected with a CRISPR-Cas9 derived editing system.

Using a variety of tools in the CrisprBits tool box and our intelligent guide design engine, various strategies for edits are being experimented upon in the lab. Single cells would be individually screened for the edit and differentiated into rods and cones which hold potential for a transplant and cure for the disease phenotype.