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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS

  • Market opportunity may expand to $2.3 billion for Type 1 Gaucher Disease patients
  • Expanding market opportunity to treat Type 1 patients based on oral dosage in an existing large and profitable market

Type 1 Gaucher Disease Market Opportunity

  • Current $1.5 billion U.S. market for intravenous administered ERT therapy;
  • Only 50-60% of Type 1 patients are treated
  • ERAD plans to introduce an oral formulation of our drug
    • We believe newly diagnosed Type 1 patients will be preferentially started on our therapy and existing Type 1 patients may switch
  • $2.35 billion U.S. market opportunity in Type 1
  • 8,000 total addressable U.S. patient population (100%)
  • Potentially applicable in treating the Parkinson-like symptoms associated with both Gaucher Disease patients and disease carriers