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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS for Gaucher Disease

Unique Drug Delivery System

  • Is delivered to all cells in the body with the ability to salvage mis-folded, but functional proteins.
  • Is delivered across the blood brain barrier and should address untreatable manifestations of Tay-Sachs and 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 with applicability for crossing the blood-brain-barrier and accessing neuronal tissues
  • Gaucher Disease with applicability for both Type 1 and Type 3 disease
  • In-Licensed from the Hospital for Sick Children, Toronto, Canada
  • World-wide exclusive rights
  • Key patents issued and pending covering use of mCT.
  • Entering manufacturing development stage for parenteral formulation.