Table of Content

1. Executive Summary
• Introduction
2. Gaucher Disease: Overview
• Introduction
• Causes
• Pathophysiology
• Signs and Symptoms
• Diagnosis
• Treatment
3. Pipeline Analysis
• Assessment by Phase
o Phase I Clinical Trials
o Phase II Clinical Trials
o Phase III Clinical Trials
• Assessment by Mechanism of Action
o Enzyme Replacement Therapy (ERT)
o Substrate Reduction Therapy (SRT)
o Gene Therapy
• Assessment by Route of Administration
o Intravenous
o Oral
4. List of Key Companies Involved
• CANbridge Life Sciences Ltd. *
o Drug Profile
• Spur Therapeutics
• Prevail Therapeutics
• Sanofi
• Lingyi Biotech Co., Ltd.
• Yuhan Corporation
5. Regulatory Designations
• Orphan Drug Designation
• Fastrack Designation
• Breakthrough Therapy Designation
• Priority Review
• PRIME Designation
• RMAT Designation
• ATMP Designation
6. Clinical Data Readouts
• Trial Initiation
• Trial Completion
• Safety/Efficacy- Data Readouts
• Discontinued Products
7. Estimated Approvals
8. Merger and Acquisitions
9. Future Perspectives and Conclusion