Unique Drug Delivery System
- Delivers therapeutic proteins to all cells in the body for treatment of Gaucher Disease, other Lysosomal Storage Diseases and Cystic Fibrosis
- Can deliver drugs across the Blood-Brain Barrier to address previously untreatable neurological manifestations of Gaucher Disease
- Based on the Endoplasmic Reticulum Associated Degradation (ERAD) pathway
- Portfolio of patent protected, related but distinct compounds
- Potential for both intravenous (IV) and oral (pill) formulations
- Supported by data in animals and cell lines
Initial focus on:
- Gaucher Disease with applicability for crossing the blood-brain-barrier and accessing neuronal tissues
- Cystic Fibrosis
- In-License with the Hospital for Sick Children
- World-wide exclusive rights
- Key patents issued and pending covering both use and composition of matter.
- Entering manufacturing development stage for parenteral formulation preliminary to additional trials.
- In advanced discussions around a unique production method that could result in a formulation suitable for oral delivery