Unique Drug Delivery System
- Is delivered to all cells in the body with the ability to salvage mis-folded, but functional proteins.
- Is delivered across the blood brain barrier and should address untreatable manifestations of Tay-Sachs and Gaucher Disease
- Based on the Endoplasmic Reticulum Associated Degradation (ERAD) pathway
- Portfolio of patent protected, related but distinct compounds
- Supported by data in animals and cell lines
Initial focus on:
- Tay-Sachs Disease with applicability for crossing the blood-brain-barrier and accessing neuronal tissues
- Gaucher Disease with applicability for both Type 1 and Type 3 disease
- In-Licensed from the Hospital for Sick Children, Toronto, Canada
- World-wide exclusive rights
- Key patents issued and pending covering use of mCT.
- Entering manufacturing development stage for parenteral formulation.