erad picture subpage

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.

Platform technology

  • Based on reversibly blocking 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 particular applicability to CNS manifestations
  • In-Licensed from the Hospital for Sick Children Toronto, Canada
  • World-wide exclusive rights
  • Key patents issued and pending covering both use and diseases.
  • Entering manufacturing and clinical development stage for parenteral formulation