ReCode Therapeutics, a biopharmaceutical company powering the next wave of genetic medicines through superior delivery presented new preclinical data from the company’s inhaled mRNA-based molecular therapy program for the treatment of primary ciliary dyskinesia (PCD) in three posters at the American Thoracic Society (ATS) 2022 International Conference, taking place May 13-18, 2022 in San Francisco. The company’s mRNA-based program in PCD uses ReCode’s first-in-class Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform, which enables the targeting of organs and tissues beyond the liver via a variety of administration routes.
Read More: Nutraceuticals: The Diet Add On To Avoid Pharmaceuticals
There are currently no approved treatments for primary ciliary dyskinesia (PCD), a rare genetic disease with inherited mutations that cause the loss of ciliary activity (and therefore the loss of normal mucus clearance) in the airways. Patients with PCD have a high burden of morbidity with chronic respiratory infections, bronchiectasis, and often develop respiratory failure.
ReCode is developing a disease-modifying mRNA-based approach for the treatment of PCD caused by mutations in DNAI1, an essential gene for ciliary movement. Using the company’s proprietary delivery platform, DNAI1 mRNA is formulated in ReCode’s SORT LNPs, nebulized, and delivered as an aerosol directly into the airway. The intent is for the mRNA delivery to lead to DNAI1 protein production in target cells and therefore enable rescue of ciliary function.
“These promising new data demonstrate that our SORT LNP-formulated DNAI1 mRNA can be precisely delivered directly to the intended cells within the lungs, without significant exposure to other tissues. Importantly, treatment was shown to be well-tolerated, allowing for repeat administration. Additional preclinical data show that our DNAI1 mRNA has low immune reactivity, rescues ciliary function, and results in long-lasting functional recovery,” said David Lockhart, Ph.D., Chief Scientific Officer and President, ReCode Therapeutics. “The totality of data presented at ATS today reflects the broad potential of our novel SORT LNP platform to deliver highly targeted mRNA-based therapies and supports the continued development of inhaled mRNA as a promising disease-modifying therapy for primary ciliary dyskinesia, a rare disease with no approved treatments.”