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Delivering Protein Therapeutics to the CNS for Gaucher Disease

Private clinical stage drug company.

We create novel biological agents to treat Orphan Diseases with an initial focus on Tay-Sachs Disease and Gaucher Disease..

Tay-Sachs Disease, Gaucher Disease, other lysosomal storage diseases are orphan designated diseases.

Tremendous unmet clinical need

  • No therapeutic options for Tay-Sachs Disease .
  • No therapeutic options for the neurological complications Gaucher Disease Type 1 and 3.
  • Recently a link has been established between Gaucher Type 1 and Parkinsonism
  • The company is poised to address the neurological and non-neurological manifestations of ERAD diseases with a drug delivery system that will cross the Blood-Brain-Barrier and deliver the enzyme to all cells in the human body

Large & profitable market opportunity

  • No current treatment for Tay-Sachs Disease
  • Type 1 Gaucher Disease market currently exceeds $1.6 billion per year and is growing
  • Ability to address neurological aspects of Gaucher disease may significantly expand this market

Experienced management team and Board of Directors.