Disease indications

Asthma

Uncontrolled asthma is a significant burden for patients as ~50% of moderate and sever asthma patients are classified as uncontrolled despite current medication. In the US these are 4.4M patients suffering from asthma attacks leading to 1 million annual emergency department visits & 100 thousand inpatien stays. 80% of acute asthma attacks are associated with virus infections with ETH47 being a first-in class therapy correcting low interferon lambda levels ins asthmatics to prevent asthma attacks driven by viruses.

Protein Replacement Therapies

Primary Ciliary Dyskinesia (PCD)

Primary Ciliary Dyskinesia (PCD) is a rare disease that arises from structural defects or the absence of the cilia lining our respiratory tract. With the cilia unable to function, mucus loaded with trapped microbes, dust, and other debris cannot be cleared from the airways efficiently and can often lead to permanent lung damage. With our SNIM®RNA technology we can deliver a corrected mRNA that is designed to produce ciliary proteins in the respiratory tract thereby reconstituting cilia function.

Pulmonary Alveolar Proteinosis (PAP)

Pulmonary alveolar proteinosis (PAP) is a rare autoimmune disease that results in difficulties in breathing due to reduced gas exchange in the lung. The underlying cause of disease in~90%   of patients is the formation of autoantibodies against GM-CSF, which results in dysfunction of local macrophages and subsequently causes the lung’s tiny air sacs, called alveoli, to fill up with an oily substance called surfactant. There is currently no approved curative pharmacotherapy available. Inhalation of recombinant GM-CSF (rGM-CSF) has limited clinical efficacy due to high levels of endogenous anti-GM-CSF antibodies and the potential for further induction of anti-drug-antibody following treatment with recombinant protein. Our SNIM® RNA-carrier complex can be delivered directly to the lung to restore local macrophage function.