ReCode Therapeutics Reports Breakthrough in Primary Ciliary Dyskinesia Treatment
ReCode Therapeutics Achieves a First: Proof of Activity in Primary Ciliary Dyskinesia Patients
Businesswire
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ReCode Therapeutics has announced successful clinical data from its RCT1100 inhaled mRNA program for treating primary ciliary dyskinesia (PCD), a rare genetic disorder. The Phase 1b trial showed that 57% of patients experienced significant improvement in mucociliary clearance, marking a significant step towards a new treatment for PCD.
- 01The RCT1100-103 Phase 1b trial involved sites in Denmark, Germany, and the United States, confirming the delivery and activity of mRNA therapeutics in patients.
- 02Bronchoscopy results indicated restoration of protein and ciliary activity in the airways, correlating with improved mucociliary clearance.
- 03The drug was reported to be safe and well-tolerated, with no serious adverse events recorded during the study.
- 04ReCode’s RCT1100 program builds on previous studies, including RCT1100-101 and RCT1100-102, which established safety and tolerability.
- 05PCD affects approximately 1 in 7,500 people, with an estimated 45,000 patients in the U.S., and many remain undiagnosed due to lack of screening.
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ReCode Therapeutics has made a significant breakthrough in the treatment of primary ciliary dyskinesia (PCD) by presenting clinical data from its RCT1100 inhaled mRNA program at the American Thoracic Society (ATS) 2026 International Conference. This marks the first clinical evidence of biological activity for a genetic medicine in PCD, a rare and progressive genetic disorder characterized by impaired mucociliary clearance and chronic respiratory issues. The Phase 1b trial, conducted across sites in Denmark, Germany, and the United States, demonstrated that 57% of participants achieved meaningful improvements in mucociliary clearance after 12 weeks. Additionally, bronchoscopy confirmed restoration of protein and ciliary activity in the airways, correlating positively with improvements in mucociliary function. The treatment was safe and well-tolerated, with no serious adverse events reported. Chief Executive Officer Shehnaaz Suliman highlighted this achievement as a validation of ReCode’s SORT lipid nanoparticle platform, paving the way for new therapeutic options for PCD patients. The company’s research indicates that PCD affects at least 45,000 individuals in the U.S., with many remaining undiagnosed due to insufficient screening.
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This breakthrough offers hope for improved treatment options for PCD patients, potentially enhancing their quality of life and health outcomes.
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