Introduction Executive Summary Gaucher’s Disease: Overview ● Causes ● Mechanism of Action ● Signs and Symptoms ● Diagnosis ● Disease Management Pipeline Therapeutics ● Comparative Analysis Therapeutic Assessment ● Assessment by Product Type ● Assessment by Stage and Product Type ● Assessment by Route of Administration ● Assessment by Stage and Route of Administration ● Assessment by Molecule Type ● Assessment by Stage and Molecule Type Gaucher’s Disease – DelveInsight’s Analytical Perspective In-depth Commercial Assessment ● Gaucher’s Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends Gaucher’s Disease Collaboration Deals ● Company-Company Collaborations (Licensing / Partnering) Analysis ● Company-University Collaborations (Licensing / Partnering) Analysis Late Stage Products (Phase III) ● Comparative Analysis Drug name: Company name ● Product Description ● Research and Development ● Product Development Activities Drug profiles in the detailed report….. Mid Stage Products (Phase II) ● Comparative Analysis Arimoclomol: Orphazyme ● Product Description ● Research and Development ● Product Development Activities Drug profiles in the detailed report….. Early Stage Products (Phase I/II) ● Comparative Analysis AVR-RD-02: AVROBIO ● Product Description ● Research and Development ● Product Development Activities Drug profiles in the detailed report….. Inactive Products ● Comparative Analysis Gaucher’s Disease Key Companies Gaucher’s Disease Key Products Gaucher’s Disease- Unmet Needs Gaucher’s Disease- Market Drivers and Barriers Gaucher’s Disease- Future Perspectives and Conclusion Gaucher’s Disease Analyst Views Gaucher’s Disease Key Companies Appendix

Table 1 Total Products for Gaucher’s Disease Table 2 Late Stage Products Table 3 Mid Stage Products Table 4 Early Stage Products Table 5 Pre-clinical & Discovery Stage Products Table 6 Assessment by Product Type Table 7 Assessment by Stage and Product Type Table 8 Assessment by Route of Administration Table 9 Assessment by Stage and Route of Administration Table 10 Assessment by Molecule Type Table 11 Assessment by Stage and Molecule Type Table 12 Inactive Products

• Orphazyme • AVROBIO • Prevail Therapeutics