ReCode Therapeutics, a clinical-stage genetic medicines company using lipid nanoparticle delivery to power the next wave of mRNA and gene correction therapeutics, today announced the presentation of clinical data from its RCT1100 PCD inhaled mRNA program at the American Thoracic Society (ATS) 2026 International Conference, held May 15-20, 2026, at the Orange County Convention Center in Orlando, FL.
The data represent the first clinical evidence of biological activity for a genetic medicine in primary ciliary dyskinesia (PCD), a rare, inherited disease characterized by progressive lung destruction for which no treatments exist. This is the first indication of successful delivery and translation of an mRNA therapeutic in the human airway.
Data from ReCode’s RCT1100-103 Phase 1b trial across sites in Denmark, Germany, and the United States showed that 57% of patients achieved meaningful improvement in mucociliary clearance at 12 weeks. Bronchoscopy confirmed restoration of protein and ciliary activity in the airway. Restoration of protein was correlated with positive changes in mucociliary clearance. The drug was safe and well-tolerated. There were no serious adverse events.
“For the first time, we demonstrated the promise of inhaled genetic medicine delivery to the lung, and downstream biologic activity in PCD patients,” said Shehnaaz Suliman, chief executive officer of ReCode. “This is substantial validation of ReCode’s SORT LNP platform and opens the door to a new treatment paradigm for PCD patients.”
“These data represent a foundational advancement for PCD patients,” said John Matthews, chief medical officer of ReCode. “This has been a huge collaborative effort with the PCD community, and we are grateful to the patients and investigators who participated in this study.”
RCT1100-103 was supported by data from two earlier trials: a single-dose Phase 1a study (RCT1100-101) in healthy volunteers and patients with PCD, and a multiple-dose Phase 1b study (RCT1100-102) in patients with PCD, which established the safety and tolerability of RCT1100 across dose levels up to 5 mg administered three times weekly, with no serious or treatment-emergent adverse events of Grade 3 or higher. A separate longitudinal observational study of 25 adults with confirmed PCD found that MCC values were consistently low regardless of underlying genotype, consistent with the baseline MCC values in the RCT1100-103 study. Together, these data support MCC as a reliable measure for evaluating disease-modifying therapies in this population in future studies.
The company was also featured during an oral showcase at the ATS 2026 Respiratory Innovation Summit (RIS), held May 15-16 in Orlando.
About Primary Ciliary Dyskinesia
Primary ciliary dyskinesia (PCD) is a rare genetic disease characterized by deficient mucociliary clearance (MCC), chronic respiratory tract infections, bronchiectasis, and declining respiratory function. Mutations in more than 50 genes result in dysfunctional cilia and loss of MCC. PCD is a progressive disorder, meaning it will get worse over time, for which no cure or disease-modifying treatments currently exist. PCD is estimated to affect at least 1 in 7,500 people across diverse ethnicities, with approximately 45,000 patients in the U.S. Due to the lack of widespread screening, approximately 80% of people with PCD are thought to be undiagnosed.
About ReCode Therapeutics
ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease, enabling improved efficacy and potency.
For more information, visit www.recodetx.com and follow us on LinkedIn and Instagram.
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