Table of Content

1. Executive Summary
• Introduction
2. Cystic Fibrosis: 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 CFTR Modulators
o Gene Therapy & RNA-Based Therapies
o Anti-Infective Therapies
o Anti-Inflammatory & Immunomodulators
o Others
• Assessment by Route of Administration
o Oral
o Inhalation
o Intravenous
4. List of Key Companies Involved
• Vertex Pharmaceuticals Incorporated*
o Detailed Drug Profile
• Spirovant Sciences, Inc.
• SpliSense Ltd.
• Enterprise Therapeutics Ltd
• Boehringer Ingelheim International GmbH
• 4D Molecular Therapeutics
• Clarametyx Biosciences, Inc.
• Arcturus Therapeutics, Inc.
• Krystal Biotech, Inc.
• Cystetic Medicines, Inc.
• Kither Biotech Srl
• ReCode Therapeutics
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