Drug discovery in engineered cell systems
RHO-associated autosomal dominant retinitis pigmentosa (RHO-adRP) is an inherited retinal disease with no disease-modifying treatment that affects thousands of patients.
Octant is using the Navigator to design small molecule correctors that mitigate toxic accumulation of misfolded rhodopsin in the retina. We’ve engineered human cells to read out on trafficking and biophysical properties of RHO mutations, generating millions of data points from each screen that guide drug design.