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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS for Gaucher Disease

Our unique drug platform employs inactivated Cholera toxin that has had its cell cytotoxicity neutered by genetic manipulation of the DNA and hence the protein. This modified Cholera toxin (mCT) is delivered to all cells in the body, and crosses the blood brain barrier.

Modified Cholera toxin is composed of an A and B subunit. The B-subunit is the delivery vehicle which can be modified to carry a variety of other proteins to the CNS. We plan to employ this approach for treatment of Gaucher Disease.

Platform technology

  • 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
    • Gaucher Disease with focus on the neurological complications of Type 1 and Type 3 Gaucher Disease
  • In-License from the Hospital for Sick Children
  • World-wide exclusive rights
  • Key patents issued and pending covering both use and specific diseases.
  • Entering manufacturing development stage for parenteral formulation preliminary to additional trials.