ReCode Initiates Phase 1 Clinical Trial of mRNA Inhaled Therapy RCT2100 for Cystic Fibrosis

ReCode Therapeutics, a company specializing in genetic medicine, has initiated a Phase 1 clinical trial for RCT2100, an innovative inhaled treatment for cystic fibrosis (CF). This therapy is specifically tailored for the 10-13% of CF patients with Class I mutations in the CFTR gene, who do not respond to current CFTR modulators or experience side effects from them. RCT2100 is the second clinical-stage candidate using ReCode's SORT LNP delivery platform, which aims to deliver CFTR mRNA to target cells, enabling them to produce a functional CFTR protein.
The study, conducted in New Zealand, is a double-blind, placebo-controlled investigation of the safety and tolerability of a single dose of RCT2100 administered via nebulizer. Approximately 32 healthy adults are expected to participate, receiving either a placebo or the mRNA-based therapy. ReCode anticipates submitting an IND application to the FDA for a Phase 1 trial in CF patients in the first half of 2024.
Cystic fibrosis is a progressive genetic disease affecting around 130,000 individuals globally. It is caused by mutations in the CFTR gene, leading to lung infections and respiratory failure. While CFTR modulator treatments have advanced, no therapies currently restore function for Class I mutations, as these mutations prevent the production of modifiable CFTR protein.
ReCode Therapeutics utilizes its SORT LNP platform for precision delivery of genetic medicines, targeting organs, tissues, and cells affected by disease. Their lead programs include RCT1100 for primary ciliary dyskinesia and RCT2100 for CF patients with Class I mutations. The company is also expanding its pipeline to develop therapies for other genetic conditions, including musculoskeletal, CNS, liver, and infectious diseases. ReCode has been honored as a top workplace by the San Francisco Business Times and Silicon Valley Business Journal. 

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