Small Molecule Therapy

Our research team has identified a targeted approach to validate novel therapeutics for Batten disease using already FDA approved drugs, GRAS nutraceuticals and designer molecules that can potentially correct the mutated gene. This validation method involves patients’ own cells in culture to assess the effect of these compounds on restoring the function of the abnormal protein. As these agents are mostly safe for human use, there is high potential for lead compounds to move quickly into clinical trial.