ETH42 is our lead therapeutic candidate currently under development for the treatment of Primary Ciliary Dyskinesia (PCD), a severe genetic pulmonary disease affecting around 1 in every 15,000 people worldwide.
Current PCD treatments are extremely limited and can only help to manage the symptoms of the disease and deal with complications that arise from it. Regular physical therapy and the inhalation of hypertonic saline are often prescribed, but there is a high unmet need for therapies to treat PCD’s underlying causes.
ETH42 is based on our ground-breaking proprietary SNIM®RNA technology platform and provides corrected mRNA instructions for the CCDC40 gene, one of the most prevalent causative genes behind the onset of PCD. CCDC40 plays a pivotal role in assembly and function of the dynein regulatory complex, a structure of motile cilia that regulates movement. CCDC40 patients are in the most severely affected populations of PCD sufferers as measured by BMI and lung function, making it an ideal target for our lead PCD therapeutic.
Weekly treatment with ETH42 by nebulization, is designed to enable maturing cells in the airways to grow normal functional cilia, leading to a restoration of mucus clearance, reduced inflammation, prevention of infections that lead to long term structural damage, and improved overall lung function.
ETH42 is in pre-clinical development. Find out more about the development status of ETH42 here.