Phase 1 study aims to evaluate safety and tolerability of a single ascending dose of RCT1100 in patients with PCD caused by mutations in the DNAI1 gene

RCT1100 is the first clinical evaluation of an inhaled mRNA-based therapy designed to restore ciliary function in people with PCD

ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced that a patient was treated with RCT1100, a novel mRNA-based therapy currently being evaluated in a Phase 1 study in patients with primary ciliary dyskinesia (PCD) caused by pathogenic mutations in the DNAI1 gene.

“This is the first time a patient with PCD has received a targeted mRNA-based therapeutic,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “This study will evaluate RCT1100 as a potential novel treatment option for patients who do not have any approved therapies. We are especially grateful to the PCD community for the continued support and collaboration as we continue our efforts to develop a treatment for PCD patients.”

PCD is a rare and progressive genetic disease that causes cilia, the microscopic hairlike structures that line the respiratory tract, to be dysfunctional and, therefore, unable to move mucus out of the body. Bacteria and viruses become trapped in the airways, which leads to chronic infections and impaired respiratory function. Approximately 10% of PCD cases result from mutations in the DNAI1 gene.

ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform was used to develop RCT1100, an inhaled mRNA therapy designed to deliver and express DNAI1 mRNA in target cells. The DNAI1 mRNA is intended to establish or restore proper ciliary function by addressing the underlying cause of the disease. In preclinical studies, the company’s DNAI1 mRNA led to efficient production of the DNAI1 protein and rescue of ciliary function in a relevant model of PCD.

“As the presenting symptoms of PCD can imitate common and less serious disorders, an accurate diagnosis is critical but often elusive for patients. There are currently no FDA-approved therapies that help directly rescue impaired cilia, so current treatment for PCD typically focuses on improving lung function and delaying disease progression,” said Michele Manion, president and executive director of the PCD Foundation. “The initiation of the Phase 1 study of RCT1100 marks an important milestone for our patient community and will support our ongoing efforts to change the course of treatment for PCD.”

In August 2023, ReCode completed Part A of the Phase 1 study, a double-blind, placebo-controlled study that evaluated the safety and tolerability of RCT1100 in 40 healthy adult volunteers. No serious side effects were observed in that study. Part B of the study is open-label and designed to assess the safety and tolerability of a single ascending dose of inhaled RCT1100 administered with a nebulizer in patients with PCD caused by pathogenic mutations in the DNAI1 gene. ReCode anticipates enrolling up to nine PCD patients across sites in the U.S. and internationally. For more information, please visit clinicaltrials.gov.

“With no approved therapeutics to treat the underlying cause of PCD, we are excited to bring our inhaled mRNA-based therapy forward into the clinic and to patients,” said David Lockhart, Ph.D., president and chief scientific officer of ReCode Therapeutics. “RCT1100 is currently the only treatment in clinical development for PCD. This study brings us one step closer to improving the respiratory health and quality of life of people living with this disease.”

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 50 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 or disease-modifying treatments currently exist. PCD is estimated to affect at least 1 in 7,500 people across diverse ethnicities, with approximately 45,000 patients in the U.S. Due to the lack of widespread screening, approximately 80% of people with PCD are thought to be undiagnosed.

ReCode has launched ThinkPCD.com, a program that provides genetic testing to PCD patients that qualify at no cost. Patients can enroll directly or through a healthcare professional who can go to the website to determine if their patients are eligible for genetic testing through the program.

About ReCode Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues and cells implicated in disease, enabling improved efficacy and potency. ReCode’s lead programs include RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and RCT2100 for the treatment of the 10-13% of cystic fibrosis patients who have Class I mutations in the CFTR gene and do not respond to currently approved CFTR modulators. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including musculoskeletal, central nervous system, liver and infectious disease indications.

ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn.

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