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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS for Gaucher Disease

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

Platform technology

  • 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