According to ReCode Therapeutics, the FDA has cleared the 3rd part of an ongoing Phase 2 trial of nebulized RCT2100 CFTR mRNA therapy in cystic fibrosis patients, and enrollment has opened at US sites. The new portion of the study will evaluate the safety and tolerability of RCT2100 plus ivacaftor over 6 weeks. Enrollment at UK and EU sites is expected to begin in early 2026.
In March 2025, the FDA granted orphan drug designation to RCT2100 for the treatment of CF in patients who do not benefit from CFTR modulators. The company recently announced that Cystic Fibrosis Foundation would provide an additional $3 million to support the Phase 2 trial.
ReCode Therapeutics CEO Shehnaaz Suliman commented, “Advancing the study of the combination of RCT2100 with ivacaftor represents an important milestone for the program and for patients. This study will provide further insight into the potential additive benefits of RCT2100 when combined with ivacaftor on clinically meaningful measures such as lung function and patient-reported outcomes. Together with data generated from the other cohorts in this study, we are building a comprehensive dataset to inform our future development strategy.”
Read the ReCode Therapeutics press release
