ReCode Therapeutics Announces First Participants Dosed in a Healthy Volunteer Clinical Trial for the Treatment of Primary Ciliary Dyskinesia

ReCode Therapeutics, a private, clinical-stage genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics, announced today that the first healthy volunteer participants have been dosed in a Phase 1 clinical trial of RCT1100, a first-in-class, mRNA-based genetic medicine for the treatment of people with primary ciliary dyskinesia (PCD) caused by pathogenic mutations in the DNAI1 gene.

“Initiating this clinical study of RCT1100, which was developed using our novel Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, represents a major milestone for ReCode and delivering on the promise of extra-hepatic delivery of genetic medicines,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “Our platform enables precise delivery of genetic medicines like RCT1100 to cells impacted by disease, and we are one step closer to initiating a clinical trial in PCD patients.”

There are currently no approved treatments for PCD, a rare genetic disease caused by inherited mutations leading to loss of ciliary activity (and therefore the loss of normal mucus clearance) in the airways. Patients with PCD have a high burden of morbidity with chronic respiratory infections and bronchiectasis, and often develop respiratory failure. RCT1100 is designed to be a disease-modifying mRNA-based therapeutic for PCD caused by pathogenic mutations in DNAI1, a gene that encodes a protein essential for ciliary movement. Formulated using ReCode’s proprietary SORT LNP delivery platform, RCT1100 is nebulized and delivered as an aerosol directly into the airway using an optimized eFlow® Nebulizer System (PARI). The intent is for the mRNA delivery to lead to DNAI1 protein production in target cells and thereby rescue ciliary function. Preclinical results supporting the mechanism of action of RCT1100 in vitro and in vivo were presented at the American Thoracic Society (ATS) 2022 International Conference.

“PCD is a genetic disease with a serious impact on respiratory health that significantly affects quality of life and currently has no approved treatments. We are excited to take this next step toward a first-in-class, precision genetic medicine for the treatment of people living with PCD,” stated David Lockhart, Ph.D., chief scientific officer and president, ReCode Therapeutics. “This program was the result of many years of work, beginning with the design and optimization of the DNAI1 mRNA, and continuing all the way through pharmacology and toxicology studies to support dosing in humans. We believe that it serves as validation for our SORT LNP delivery platform that will enable us to translate the potential of genetic medicines into clinical outcomes, ultimately altering the course of disease for many people living with rare genetic disorders.”