โ Abstract selected for oral presentation at the American Thoracic Society International Conference 2026ย โ
May 23, 2026 โ The international PCD Clinical Trial Network (PCD CTN) played a central role in the clinical execution of a groundbreaking study investigating inhaled mRNA therapy in patients with primary ciliary dyskinesia (PCD), presented at the ATS 2026 International Conference in Orlando, USA (May 15โ20, 2026).
Two highly specialized PCD CTN investigator sites were deeply involved in the study:
- Danish PCD Centre, Copenhagen, Rigshospitalet (Caroline Nygaard, Tavs Qvist and Kim G. Nielsen)
- University Childrenโs Hospital Muenster (Johanna Raidt and Heymut Omran)
In addition, a U.S. site at the University of North Carolina (UNC), Chapel Hill contributed to the trial.
The data represent the first clinical evidence of biological activity of a genetic medicine in PCD, a rare inherited disease characterized by progressive lung damage and the absence of disease-modifying treatments. These findings provide the first indication of successful delivery and translation of an inhaled mRNA therapeutic in the human airway.
Results from the Phase 1b trial (RCT1100-103), conducted across sites in Denmark, Germany, and the United States, showed that 57% of patients achieved a clinically meaningful improvement in mucociliary clearance (MCC) at 12 weeks. Bronchoscopy confirmed restoration of protein expression and ciliary function in the airways. This biological activity correlated with improvements in MCC. The therapy was safe and well tolerated, with no serious adverse events reported.
The study builds on earlier Phase 1 trials, which established the safety and tolerability of repeated dosing. In parallel, longitudinal observational data have demonstrated consistently reduced MCC across PCD patients regardless of genotype, supporting MCC as a robust endpoint for evaluating disease-modifying therapies in future studies.
These findings represent an important milestone for the PCD field and reflect a strong collaboration between international expert centers, patients, and industry partners. The investigational therapy was developed by ReCode Therapeutics, which contributed the underlying mRNA and lipid nanoparticle delivery technology.
About Primary Ciliary Dyskinesia (PCD)
Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterized by impaired mucociliary clearance (MCC), chronic respiratory infections, bronchiectasis, and progressive loss of lung function. Mutations in more than 50 genes lead to dysfunctional cilia and loss of MCC. PCD is a progressive disease with no curative or disease-modifying treatments currently available. It is estimated to affect at least 1 in 7,500 individuals, although up to 80% of patients are thought to remain undiagnosed.
About PCD CTN
The PCD Clinical Trial Network (PCD CTN) is an international network of specialized centers dedicated to advancing clinical research and therapeutic development for patients with primary ciliary dyskinesia. The network enables high-quality multicenter trials through standardized protocols, harmonized outcome measures, and close collaboration between clinicians, researchers, and patient organizations.
About ReCode Therapeutics
ReCode Therapeutics is a clinical-stage genetic medicines company developing mRNA and gene correction therapies using lipid nanoparticle-based delivery technologies for targeted treatment of disease.
