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Delivering Protein Therapeutics to the CNS for Gaucher Disease

Private pre-clinical drug company.

We create novel biological agents to treat Orphan Diseases with an initial focus on Gaucher Disease and Cystic Fibrosis.

Gaucher Disease, other lysosomal storage diseases and Cystic Fibrosis are orphan designated diseases.

Tremendous unmet clinical need

  • No therapeutic options for the neurological consequences of Gaucher Disease Type 3
  • Recently a link has been established between Gaucher Type 1 and Parkinsonism
  • Existing Type 1 therapies do not address the neurological aspects of Gaucher Disease
  • The company is poised to address the neurological and non-neurological manifestations of Gaucher Disease 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

  • 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.