ReCode Therapeutics, a leader in genetic medicines, is pioneering a promising avenue in the treatment of primary ciliary dyskinesia (PCD) with its latest clinical trial involving RCT1100, a groundbreaking mRNA-based therapy. This novel approach is the first of its kind to be clinically evaluated for restoring ciliary function in individuals affected by PCD, particularly those with mutations in the DNAI1 gene.
Utilizing ReCode's proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, this inhaled mRNA therapy is designed to deliver and express DNAI1 mRNA in target cells. The aim is to establish or restore proper ciliary function by addressing the disease at its genetic root. Preclinical studies have shown promising results, with the DNAI1 mRNA effectively producing the DNAI1 protein and rescuing ciliary function in relevant PCD models.
The Phase 1 study of RCT1100 is set to evaluate the safety and tolerability of the therapy in patients with PCD caused by mutations in the DNAI1 gene. Earlier in August 2023, Part A of the study, involving 40 healthy adult volunteers, showed no serious side effects, marking a positive progression in the research. The ongoing Part B of the study is an open-label trial assessing the safety and tolerability of a single ascending dose of RCT1100, administered through a nebulizer.
The PCD Challenge and RCT1100’s Potential Impact
PCD, a rare genetic disorder, is characterized by deficient mucociliary clearance, leading to chronic respiratory tract infections and progressive respiratory impairment. With mutations in over 50 genes contributing to PCD, the disease affects at least 1 in 7,500 people across various ethnicities. Currently, there are no FDA-approved therapies that directly rescue impaired cilia, making RCT1100 a beacon of hope for about 45,000 patients in the U.S. alone.
Recognizing the challenges in diagnosing and treating PCD, ReCode has initiated ThinkPCD.com, a program offering no-cost genetic testing to eligible PCD patients. This initiative not only aids in diagnosis but also fosters a deeper understanding of the disease.