ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Trial of Novel Disease-Modifying Genetic Medicine, RCT1100 for the Treatment of Primary Ciliary Dyskinesia

– Phase 1 trial to evaluate safety and tolerability of a single ascending dose of RCT1100, an inhaled mRNA-based genetic medicine

– First clinical-stage candidate from ReCode’s proprietary SORT LNP delivery platform designed to restore ciliary function in the lungs of people with PCD caused by pathogenic mutations in the DNAI1 gene; IND submission planned for 2H23

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.”

The Phase 1 double-blind, placebo-controlled, first-in-human study of RCT1100 is designed to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered via nebulizer. The study is being conducted in New Zealand and ReCode anticipates enrolling approximately 32 healthy adults who will receive a single dose of either placebo or RCT1100. For more information, please visit clinicaltrials.gov.

ReCode plans to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration for a Phase 1 trial of RCT1100 in PCD patients in the second half of 2023, and another IND for its CFTR mRNA therapeutic for the treatment of cystic fibrosis, an inherited life-threatening genetic disorder that affects approximately 100,000 people worldwide.

About Primary Ciliary Dyskinesia

Primary ciliary dyskinesia (PCD) is a rare genetic disease characterized by deficient mucociliary clearance (MCC), chronic respiratory tract infections, bronchiectasis, and declining respiratory function. Mutations in more than 40 different genes result in dysfunctional cilia and loss of MCC. PCD is a progressive disorder, meaning it will get worse over time, for which no cure currently exists. There are estimated to be more than 87,000 people affected with PCD across North America and Europe, with more worldwide.

About ReCode Therapeutics

ReCode Therapeutics is a genetic medicines company using superior delivery to power the next wave of messenger RNA (mRNA) and gene correction therapeutics. ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation, genetic medicines technology that enables precise delivery to target organs and cells beyond the liver. The SORT LNP platform is the foundation for ReCode’s pipeline of disease-modifying mRNA and gene correction therapeutics. ReCode’s lead programs are focused on primary ciliary dyskinesia and cystic fibrosis. ReCode is leveraging its SORT LNP platform and nucleic acid technologies to expand its pipeline with therapeutics that use mRNA-mediated replacement and gene correction in target organs with precision targeting of disease-relevant cells. In 2022, ReCode was named among Fierce Biotech’s “Fierce 15” as one of the most promising early-stage biotechnology companies in the industry, and was described by Nature as one of the “Seven Technologies to Watch in 2022” for its SORT LNP platform. For more information, visit www.recodetx.com and follow us on LinkedIn.

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