NEW YORK – ReCode Therapeutics on Wednesday said it has dosed the first cohort of healthy volunteers in a Phase I trial testing an inhaled messenger RNA (mRNA) therapy designed as a potential treatment for cystic fibrosis.
The placebo-controlled and first-in-human trial assesses the safety and tolerability of a single ascending dose of RCT2100, an investigational treatment that aims to treat cystic fibrosis caused by nonsense mutations in the CFTR gene. Nonsense mutations result in patients who have no CFTR proteins and do not respond to approved CFTR modulators.
The mRNA therapy, which is administered using a nebulizer, is designed to deliver CFTR mRNA to target cells, which can instruct them to produce functional versions of the CFTR protein.
ReCode expects to enroll about 32 healthy volunteers into the Phase I trial in New Zealand. The firm plans to submit an investigational new drug application to the US Food and Drug Administration to begin a Phase I trial in the US in the first half of this year.
RCT2100 is the second therapeutic candidate developed using Menlo Park, California-based ReCode's selective organ targeting (SORT) lipid nanoparticle delivery platform after RCT1100, an investigational treatment for primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene.