According to ReCode Therapeutics, the FDA has granted orphan drug designation to the company’s RCT2100 inhaled mRNA therapy for the treatment of cystic fibrosis in patients who do not benefit from CFTR modulators. In February 2024, ReCode announced the initiation of a Phase 1 trial of RCT2100 in healthy adults. The company says that a Phase 1b study is now recruiting CF patients in the US, UK, France, and the Netherlands.
In addition to RCT2100, ReCode’s pipeline includes RCT1100 inhaled mRNA therapy for the treatment of primary ciliary dyskinesia, which entered Phase 1 development in 2023. RCT1100 received orphan drug designation in 2024, and a Phase 1b study is ongoing. Both formulations are based on the company’s SORT lipid nanoparticle delivery platform.
ReCode CEO Shehnaaz Suliman commented, “While there have been tremendous advancements in the development of novel CF therapies in the past two decades, we are focused on the subset of CF patients who are not eligible for current treatments and who have been waiting for a treatment. We are currently enrolling patients in a Phase 1b study evaluating RCT2100 in people with CF who do not respond to or are intolerant of current modulator therapies. We are grateful to the cystic fibrosis community and physicians for the continued support of the RCT2100 clinical program.”
Read the ReCode Therapeutics press release
