Treatment of Lysosomal Storage Diseases

Technology Overview

Researchers at McMaster University have discovered that a repurposed FDA-approved drug is an effective treatment against Sandhoff disease in mice models. Treated mice had an increased life span and reduced muscle atrophy compared to control.

Benefits

• Targets a unique pathway compared to other therapeutic methods
• Can be paired with additional substrate reduction drugs or enzyme replacement treatments

Applications

• Treating Tay Sachs and Sandhoff disease

Opportunity

McMaster University is seeking for technology licensing opportunities and research collaboration.