Lysosomaltx unique approach to discovering new drugs for neurodegenerative diseases is based on research performed in the lab of Dr. Dimitri Krainc, one of the company’s founders, at Massachusetts General Hospital (MGH), a founding member of Partners HealthCare and an affiliate of Harvard Medical School.
The work completed at Lysosomaltx scientific co-founder and a former postdoctoral fellow in the lab, builds on the association between Gaucher disease, a rare lysosomal storage disorder (LSD) caused by mutations in the gene for glucocerebrosidase (GCase), and a predisposition to Parkinson’s disease.
They demonstrated that increasing GCase activity in human neurons of Gaucher disease and Parkinson’s disease patients can normalize lysosomal function and improve neuronal survival. This research continues at Northwestern University, where Dr. is now Ward Professor and chairman of the department of neurology, and Dr. is an assistant professor of neurology.