ReCode Therapeutics Doses First Patient in Phase 1 Clinical Study of Novel mRNA-based Therapy for the Treatment of Primary Ciliary Dyskinesia
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
Excerpt from the Press Release:
MENLO PARK, Calif.–(BUSINESS WIRE)–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.
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