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ReCode Therapeutics Initiates Enrollment of Phase 2 Clinical Trial of RCT2100 in Combination with Ivacaftor for the Treatment of Cystic Fibrosis

via Business Wire

– RCT2100 co-administered with ivacaftor will assess the safety and tolerability of inhaled CFTR mRNA therapy over 6 weeks in participants with CF –

– U.S. sites now enrolling, with U.K. and EU sites expected to begin enrollment in Q1 2026 –

ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted clearance for the initiation of Part 3 of its ongoing Phase 2 clinical trial evaluating RCT2100, an investigational inhaled CFTR mRNA therapy, in combination with ivacaftor in people with cystic fibrosis (CF). Clinical sites in the United States are now open for enrollment. The company plans to begin enrolling participants at sites in the United Kingdom and the European Union in the first quarter of 2026. For more information, please visit CF-Clinical-Studies.com.

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 and 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.

“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. “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.”

Formulated using ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform, RCT2100 is designed to deliver CFTR mRNA directly to target cells in the lungs, instructing them to produce a functional version of the CFTR protein that is missing in some individuals with CF. By providing this CFTR mRNA, the therapy aims to address the root cause of CF, potentially restoring CFTR protein function in the lungs.

About Cystic Fibrosis

Cystic fibrosis (CF) is a progressive, serious genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the Cystic Fibrosis Foundation. The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs and other organs. It also results in a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of people in the CF community have genetic mutations that do not enable them to benefit from these life-changing therapeutics.

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 delivers genetic medicines with high precision to organs, tissues, and cells implicated in disease, improving efficacy and potency.

ReCode’s lead programs include RCT2100, which is being developed for the treatment of the 10% to 15% of people with cystic fibrosis who have genetic mutations in the CFTR gene that do not respond to or are intolerant of currently approved CFTR modulators. RCT1100 is being developed for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated with the SORT LNP delivery platform.

For more information, visit www.recodetx.com and follow us on LinkedIn and Instagram.

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