ReCode Therapeutics has announced the initiation of a Phase 1 safety and tolerability study of RCT2100 inhaled CFTR mRNA-based therapy, which the company is developing for cystic fibrosis patients who do not benefit from CFTR modulators. The study is expected to enroll 32 healthy adults and will evaluate a single dose of nebulized RCT2100 against placebo. According to the announcement, ReCode intends to submit an IND for a Phase 1 trial of the CFTR mRNA therapy in CF patients by mid-2024.
In September 2023, ReCode announced that it had raised $50 million for development of its inhaled mRNA therapies. In addition to RCT2100, the company is developing RCT1100 for the treatment of primary ciliary dyskinesia (PCD) caused by certain genetic mutations. A Phase 1 trial of RCT1100 in PCD patients was initiated in March 2023 and is still recruiting at this time.
ReCode President and Chief Scientific Officer David Lockhart commented, “The initiation of the clinical study of RCT2100, which is the second candidate developed using our novel Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, represents an important milestone for the CF community. While progress has been made for many people with CF, those who do not benefit from existing modulators report feeling left behind and continue to face profound physical and emotional hardship from the disease. RCT2100 is designed to target the underlying cause of CF so that more people with CF may benefit, including those with rare mutations.”
Read the ReCode Therapeutics press release.
