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INNOVATIVE SCIENCE

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.