Disease Focus

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.

Neurodegeneration

Protecting and rescuing damaged neurons by selectively degrading toxic protein aggregates via autophagy

Inflammatory
disorders

Fixing the dysregulation of the immune system by targeting specific mutations associated with defective autophagy1

Metabolic
disease

Clearing pathological accumulation of proteins and lipids

Muscle
disorders

Preventing muscle necrosis and inflammation by enhancing lysosome mediated membrane repair in muscular dystrophy

1) Image attribution: Calicut Medical College [CC BY-SA 4.0 (https://creativecommons.org/licenses/by-sa/4.0)], via Wikimedia Commons