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 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 P 1037: Parion Sciences • Product Description • Research and Development • Product Development Activities Drug profiles in the detailed report….. Preclinical stage products • Comparative Analysis Research programme: RNA and gene therapeutics - ReCode Therapeutics • 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

• Parion Sciences • ReCode Therapeutics • Translate Bio • Santhera Pharmaceuticals • ethris GmbH