ReCode Therapeutics Advances Phase 2 Trial of Inhaled CFTR mRNA Therapy RCT2100 Combined with Ivacaftor for Cystic Fibrosis
核心洞察
ReCode Therapeutics (搜索) has received FDA clearance to initiate Part 3 of its Phase 2 clinical trial evaluating RCT2100, an inhaled CFTR (搜索) mRNA therapy, in combination with ivacaftor for cystic fibrosis (搜索) patients.
The study will assess safety and tolerability over six weeks in up to 12 CF participants, with secondary endpoints including lung function and quality of life measures.
U.S. clinical sites are now enrolling patients, with UK and EU sites expected to begin enrollment in Q1 2026.
ReCode Therapeutics (搜索) has received FDA clearance to advance Part 3 of its ongoing Phase 2 clinical trial evaluating RCT2100, an investigational inhaled CFTR (搜索) mRNA therapy, in combination with ivacaftor for people with cystic fibrosis (搜索). Clinical sites in the United States are now open for enrollment, with plans to begin enrolling participants at sites in the United Kingdom and European Union in the first quarter of 2026.
Trial Design and Objectives
The multi-part, open-label Phase 2 clinical trial is designed to evaluate the safety, tolerability and biodistribution of RCT2100 when administered by oral inhalation through a nebulizer. Part 1 of the study investigated single ascending doses of RCT2100 in healthy volunteers, while Part 2 explored multiple ascending doses in participants with CF.
Part 3, which is currently enrolling participants, will evaluate the primary endpoints of safety and tolerability of RCT2100 when co-administered with ivacaftor over a six-week treatment period in up to 12 individuals with CF. Secondary endpoints include assessments of lung function and quality of life. The results of this study will inform future development and optimization of the RCT2100 treatment regimen.
Technology Platform and Mechanism
RCT2100 uses ReCode's Selective Organ Targeting (SORT) lipid nanoparticle platform to deliver CFTR (搜索) mRNA to lung cells directly, aiming to restore function of the CFTR protein absent in some CF patients. This approach represents a novel therapeutic strategy for addressing the underlying genetic defect in cystic fibrosis (搜索).
Clinical Significance
"Advancing the study of the combination of RCT2100 with ivacaftor represents an important milestone for the program and for patients," said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics (搜索). "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."
Company Pipeline
In addition to the CF program, ReCode has been advancing other mRNA-based therapies. In January 2024, the company dosed the first subject in a Phase 1 clinical trial of its inhaled mRNA-based therapy, RCT1100, for primary ciliary dyskinesia (搜索) caused by dynein axonemal intermediate chain 1 gene mutations.