Introduction Executive Summary Primary ciliary dyskinesia: 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 Primary ciliary dyskinesia – DelveInsight’s Analytical Perspective In-depth Commercial Assessment • Primary ciliary dyskinesia companies’ collaborations, Licensing, Acquisition -Deal Value Trends Primary ciliary dyskinesia Collaboration Deals • Company-Company Collaborations (Licensing / Partnering) Analysis • Company-University Collaborations (Licensing / Partnering) Analysis Early Stage Products (Phase I/II) • Comparative Analysis Lonodelestat: Santhera Pharmaceuticals • Product Description • Research and Development • Product Development Activities Drug profiles in the detailed report….. Preclinical Stage Products • Comparative Analysis Research programme: RNA therapeutics: ReCode Therapeutics • Product Description • Research and Development • Product Development Activities Drug profiles in the detailed report….. Discovery Stage Products • Comparative Analysis Research programme: mRNA therapeutics: Translate Bio • Product Description • Research and Development • Product Development Activities Drug profiles in the detailed report….. Inactive Products • Comparative Analysis Primary ciliary dyskinesia Key Companies Primary ciliary dyskinesia Key Products Primary ciliary dyskinesia- Unmet Needs Primary ciliary dyskinesia- Market Drivers and Barriers Primary ciliary dyskinesia- Future Perspectives and Conclusion Primary ciliary dyskinesia Analyst Views Primary ciliary dyskinesia Key Companies Appendix

Table 1 Total Products for Primary ciliary dyskinesia 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

• Translate Bio • ReCode Therapeutics • Santhera Pharmaceuticals