Therapeutic Focus

Casma Therapeutics is working to accelerate the clinical translation of autophagy insights into new treatments for patients with rare genetic diseases and broader pathologies where restoration of cellular balance may arrest or reverse disease progression. A range of preclinical data supports the idea that boosting autophagy may play an important role in improving cellular clearance in genetic diseases, including lysosomal storage disorders, systemic diseases and CNS disorders.

Casma is advancing preclinical programs in four broad areas –


Preventing muscular dystrophy associated with storage disorders by promoting autophagy and lysosomal flux


Clearing pathological accumulation of proteins and lipids


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


Protecting and rescuing damaged neurons by selectively boosting autophagy

1) Image attribution: Calicut Medical College [CC BY-SA 4.0 (], via Wikimedia Commons