Maria Fareri Children’s Hospital, Boston Children’s Health Physicians and New York Medical College Partner to Administer Novel Inhaled mRNA Therapy to Treat Cystic Fibrosis
VALHALLA, NY – January 23, 2026 – Westchester Medical Center Health Network’s (WMCHealth’s) Maria Fareri Children’s Hospital, Boston Children’s Health Physicians (BCHP) and New York Medical College (NYMC) have joined forces to pioneer a novel inhaled messenger RNA (mRNA) therapy for cystic fibrosis. This partnership brings together world-class expertise in clinical research, pediatric care, and innovative treatment development, offering new hope to the cystic fibrosis community—especially the 10 percent of patients who do not benefit from existing therapies.
The novel, investigational therapy, RCT2100, developed by ReCode Therapeutics, is currently being evaluated in a global Phase 2 clinical trial to test its safety and tolerability across multiple ascending doses. Unlike traditional treatments that primarily manage symptoms, RCT2100 is designed to address the underlying genetic cause of cystic fibrosis by delivering mRNA directly to lung cells, enabling them to produce functional cystic fibrosis transmembrane conductance regulator (CFTR) proteins.
“This partnership exemplifies our shared commitment to advancing innovative, science-driven care for children with complex diseases,” said Dr. Allen J. Dozor, President of Boston Children’s Health Physicians, Professor of Pediatrics and Clinical Public Health at New York Medical College, and Associate Physician-in-Chief of Maria Fareri Children’s Hospital.
Dr. Erika Berman Rosenzweig, William Russell McCurdy Physician-in-Chief & Director of the Department of Pediatrics at WMCHealth; Chair of the Department of Pediatrics at NYMC School of Medicine, added, “The constructive collaboration between our institutions allows us to push the boundaries of what’s possible in gene therapy. Our collective expertise and dedication are opening new doors for those who have long awaited more effective treatment options.”
If the therapy proves safe and effective, it could become a lifeline for individuals with cystic fibrosis who are unable to tolerate or respond to current medications, as well as many others living with the disease.
An Inhaled Therapy for Cystic Fibrosis
The innovative inhaled therapy RCT2100, developed by ReCode Therapeutics, is currently being evaluated through a collaborative clinical trial led by Maria Fareri Children’s Hospital, BCHP and NYMC. This partnership brings together leading experts in pediatric care, clinical research, and gene therapy to address the root cause of cystic fibrosis, rather than just managing its symptoms.
By harnessing mRNA technology, RCT2100 delivers genetic instructions to lung cells so they can generate functional CFTR proteins. This innovative method targets the root cause of cystic fibrosis at the genetic level and marks a notable step forward beyond therapies that only address symptoms. Including the research being conducted at BCHP and NYMC, the RCT2100 Phase 2 clinical trial is being conducted at multiple sites worldwide, including the United States, France, the Netherlands, New Zealand, and the United Kingdom, with 13 clinical trial sites across the U.S. participating in this important research.
About Cystic Fibrosis
Cystic fibrosis is a progressive hereditary disorder characterized by persistent pulmonary infections and respiratory insufficiency. The condition arises due to mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. According to the Cystic Fibrosis Foundation, approximately 105,000 individuals have been diagnosed with cystic fibrosis in 94 countries, with nearly 40,000 affected in the United States. The absence or malfunction of the CFTR protein leads to impaired airway hydration, resulting in increased mucus accumulation within the lungs. This defect compromises mucociliary clearance and contributes to recurrent infections, chronic inflammation, respiratory failure, and various related complications.