ETH42

ETH42 is a first-in-class, lipid nanoparticle (LNP)-formulated mRNA therapeutic developed by Ethris for the treatment of primary ciliary dyskinesia (PCD) caused by mutations in the CCDC40 gene. The drug delivers mRNA encoding the structural protein CCDC40, which is essential for proper function and structure of motile cilia in respiratory epithelial cells. By replacing the missing or defective CCDC40 protein in patients with relevant genetic mutations, ETH42 aims to restore ciliary structure and function, thereby improving mucociliary clearance and respiratory health. ETH42 is administered via inhalation to target airway epithelial cells directly. It is currently in preclinical development with plans for a Phase 1 clinical trial focused on safety, tolerability, target protein production, and improvement of ciliary function[1][5][7].