According to ReCode Therapeutics, studies of its nebulized lipid nanoparticle DNAI1 mRNA therapy for the treatment of primary ciliary dyskinesia in non-human primates demonstrated that a single dose of the mRNA could be delivered successfully for good distribution in the lungs. Along with the in vivo studies, tests in human and mouse cell models confirmed that DNAI1 protein was synthesized after delivery of the mRNA and demonstrated restoration of ciliary function. The company is presenting the data at the 2021 PCD on the Move Virtual Scientific Conference.
ReCode Therapeutics CEO and President David Lockhart said, “The data featured today demonstrate that delivery of our LNP-formulated mRNA leads to production of DNAI1 protein in the target cells and rescue of ciliary function. These results further demonstrate the potential of our novel mRNA approach as a disease-modifying therapy for primary ciliary dyskinesia, a rare lung disease that drastically affects length and quality of life. These data underscore the power of our LNP platform to deliver mRNA-based therapies to treat PCD and are an important step in advancing our program toward human clinical studies.”
Read the ReCode Therapeutics press release.
