– Phase 1 trial to evaluate the safety and tolerability of single-dose RCT1100, an inhaled mRNA-based genetic drug
– First clinical phase candidate of ReCode’s SORT LNP delivery platform designed to restore ciliary function in the lungs of people with PCD caused by pathogenic mutations in DNAI1 gene; IND filing is planned for 2H23
MENLO PARK, California, March 15, 2023–(Antara/BUSINESS WIRE)– ReCode Therapeutics, a private clinical-stage genetic pharmaceuticals company that uses superior delivery to power the next wave of mRNA and gene correction therapy, announced today that its first healthy volunteer participants have been dosed in a Phase 1 trial. RCT1100, a first-in-class mRNA-based genetic drug for the treatment of people with primary ciliary dyskinesia (PCD) caused by pathogenic mutations in DNAI1 gene.
“Starting this clinical study of RCT1100, which was developed using our new Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, is a major milestone for ReCode and fulfills the promise of extra-hepatic delivery of genetic drugs,” said Shehnaaz Suliman, MD. , MBA, M. Phil., CEO of ReCode Therapeutics. “Our platform enables the delivery of precise genetic drugs such as RCT1100 to diseased cells, and we are one step closer to starting clinical trials in PCD patients.”
There is currently no approved treatment for PCD, a rare genetic disease caused by an inherited mutation that causes loss of ciliary activity (and therefore loss of normal mucus clearance) in the airways. Patients with PCD have a high morbidity burden with chronic respiratory infections and bronchiectasis, and often experience respiratory failure. RCT1100 is designed to be a disease-modifying mRNA-based therapy for PCD caused by pathogenic mutations in DNAI1, a gene that codes for a protein essential for ciliary movement. Formulated using ReCode’s SORT LNP delivery platform, RCT1100 is nebulized and delivered as an aerosol directly into the airway using the optimized eFlow® Nebulizer System (PARI). The goal is for mRNA delivery to lead to DNAI1 protein production in the target cells and thus save the function of the cilia. Preclinical results support the mechanism of action of RCT1100 in vitro And life presented at the 2022 American Thoracic Society (ATS) International Conference.
“PCD is a genetic disease with serious impact on respiratory health that significantly affects quality of life for which there is currently no approved treatment. We are excited to take the next step towards a first-in-class precision genetic drug for the treatment of people living with PCD,” said David Lockhart, Ph.D., chief scientific officer and president, ReCode Therapeutics. “This program is the result of many years of work, starting with design and optimization DNAI1 mRNA, and continues through pharmacological and toxicological studies to support human dosing. We believe that this serves as validation for our LNP SORT delivery platform that will allow us to translate genetic drug potential into clinical outcomes, ultimately changing the disease course for many people living with rare genetic disorders.”
The first double-blind, placebo-controlled, Phase 1 human RCT1100 study designed to assess the safety and tolerability of a single inhaled dose of RCT1100 administered via a nebulizer. The study was conducted in New Zealand and ReCode anticipates enrolling approximately 32 healthy adults who will receive a single dose of placebo or RCT1100. For more information, please visit clinicaltrials.gov.
ReCode plans to submit a New Drug Investigation (IND) application to the US Food and Drug Administration for a Phase 1 RCT1100 trial in PCD patients in the second half of 2023, and another IND for CFTR Therapeutic mRNA for the treatment of cystic fibrosis, a life-threatening inherited genetic disorder that affects around 100,000 people worldwide.
About Primary Ciliary Dyskinesia
Primary ciliary dyskinesia (PCD) is a rare genetic disease characterized by mucociliary clearance (MCC) deficiency, chronic respiratory tract infections, bronchiectasis, and decreased respiratory function. Mutations in more than 40 different genes result in dysfunctional cilia and loss of MCC. PCD is a progressive disorder, meaning it gets worse over time, for which there is currently no cure. It is estimated that there are more than 87,000 people affected by PCD across North America and Europe, with many more worldwide.
About ReCode Therapeutics
ReCode Therapeutics is a genetic pharmaceuticals company that uses superior delivery to power the next wave of messenger RNA (mRNA) and gene correction therapies. The ReCode lipid nanoparticle (LNP) selective organ targeting platform (SORT) is a next-generation genetic drug technology that enables precise delivery to target organs and cells outside the liver. The LNP SORT platform is the foundation for the ReCode channel for disease-modifying mRNA and gene-correction therapy. ReCode’s main program is focused on primary ciliary dyskinesia and cystic fibrosis. ReCode leverages LNP’s SORT platform and its nucleic acid technology to expand its network with therapies that employ mRNA-mediated replacement and gene correction in target organs by precision targeting 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 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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