Disease selection is guided by the presence of validated genetic drivers in both rare and common disorders. The team is translating novel molecular insights into therapeutic candidate drugs that will degrade these disease drivers via enhanced autophagy. Neurodegeneration, inflammation, metabolic disease and neuromuscular disorders are specific areas where we are developing programs.
Protecting and rescuing damaged neurons by selectively degrading toxic protein aggregates via autophagy
Fixing the dysregulation of the immune system by targeting specific mutations associated with defective autophagy1
Clearing pathological accumulation of proteins and lipids
Preventing muscle necrosis and inflammation by enhancing lysosome mediated membrane repair in muscular dystrophy