How Inhaled mRNA May Help Rare Disease Patients Breathe Easier

RARECast

Primary ciliary dyskinesia is a rare, genetic disease that arises from structural defects or the absence of the cilia lining of respiratory tract. This leads to mucus littered with trapped microbes, dust, and other debris getting caught in the airways, which can lead to permanent lung damage. Ethris is developing an inhaled mRNA therapy to get the body to produce a needed structural protein to restore normal cilia structure and function. We spoke to Thomas Langenickel, chief medical officer of Ethris, about how the company’s technology overcomes existing challenges for the therapeutic use of mRNA, its ability to deliver treatments directly to the lung, and its pipeline of therapies in development.