First Patient Receives mRNA Therapy for Primary Ciliary Dyskinesia in Phase 1 Study

A clinical-stage genetic medicine company has embarked on a pioneering endeavor with the commencement of a Phase 1 study. The focus is on RCT1100, an innovative mRNA-based therapy, which is being assessed for its safety and efficacy in treating primary ciliary dyskinesia (PCD). This rare genetic disorder impairs the function of cilia, minuscule hair-like structures in the respiratory tract that are vital for moving mucus out of the body. Dysfunctional cilia lead to chronic infections and a decline in respiratory function, with DNAI1 gene mutations accounting for approximately 10% of PCD cases.

ReCode Therapeutics, leveraging its proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, has developed RCT1100 as an inhaled treatment. The therapy is designed to deliver and express DNAI1 mRNA in target cells, aiming to restore normal ciliary function by addressing the root cause of PCD. Encouraging results from preclinical studies have demonstrated the potential of this mRNA to produce the DNAI1 protein and revive ciliary function in relevant PCD models.

The Phase 1 study marks a significant step in the search for effective treatments for PCD, a condition that often goes undiagnosed due to its similarity to more common, less severe disorders. Currently, there are no FDA-approved therapies that directly address the impaired cilia characteristic of PCD. Existing treatments are primarily focused on improving lung function and slowing disease progression.

In August 2023, ReCode completed the first part of the Phase 1 study, a double-blind, placebo-controlled trial involving 40 healthy adult volunteers. This segment of the study showed no serious side effects associated with RCT1100. The second part of the study is now underway, an open-label trial designed to evaluate the safety and tolerability of a single ascending dose of the inhaled therapy in PCD patients with DNAI1 gene mutations. The company plans to enroll up to nine patients at sites across the U.S. and internationally.

The development of RCT1100 represents a crucial advancement in the field of genetic medicine, particularly for a disease like PCD, which lacks approved therapeutics that target its underlying cause. The study's progress is closely watched by the patient community and the medical field, as it could potentially lead to improved respiratory health and quality of life for those living with PCD.

ReCode Therapeutics is dedicated to the advancement of mRNA and gene correction therapeutics, with a focus on precision delivery to ensure that treatments reach the organs and tissues most affected by disease. The company's pipeline includes other promising therapies for a range of genetic conditions, reflecting its commitment to transforming the treatment landscape for rare and common genetic diseases.