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Delivering Protein Therapeutics to the CNS for Gaucher Disease

Our unique drug platform is based on the finding that an inactive component of a bacterial toxin (cholera toxin CT) called the B sub-unit (CTB) can be engineered to deliver enzymes and proteins to all cells throughout the body—including to brain cells, across the “blood brain barrier.” We have modified the CTB subunit to optimize it for our drug delivery strategies. We refer to this as our CTB Delivery System and it is the basis for our Platform Technology.

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.
  • We are in advanced discussions around a unique production method that should result in a formulation suitable for oral delivery.