Cystic fibrosis (CF) is the most common fatal inherited disease in the United States. CF results in mucus buildup in the lungs, pancreas and other organs, and mortality is primarily driven by a progressive decline in lung function. There is no cure for CF.
According to the Cystic Fibrosis Foundation (CFF), the median age at death for patients with CF in the United States was 30.6 years in 2017. This means that half of those deaths occurred before the age of 31 — meaning too many people with CF miss the opportunity to finish school, begin careers, start families, and reach other milestones.
According to the CFF, more than 30,000 patients in the United States and more than 70,000 patients worldwide are living with CF and approximately 900 new cases of CF are diagnosed each year.
CF is caused by dysfunctional or missing CFTR protein. We have developed an mRNA therapeutic designed to deliver mRNA encoding fully functional CFTR protein to the lung, resulting in the expression of the functional CFTR protein. This therapeutic has the potential to treat all patients with CF, regardless of the underlying genetic mutation.
Additional Pulmonary Diseases
Beyond our CF clinical program, the primary focus of our research efforts is the evaluation of targets in additional pulmonary diseases utilizing our proprietary lung delivery platform. Preclinical research efforts include discovery-stage programs in primary ciliary dyskinesia (PCD), pulmonary arterial hypertension (PAH) and idiopathic pulmonary fibrosis (IPF).
Other Rare Diseases
We believe that our MRTTM platform may be applied across a broad array of diseases and target tissues via multiple routes of administration. We are currently undergoing lead identification activities seeking to identify additional potential mRNA therapeutics in the liver, eye and central nervous system. Additionally, our MRTTM platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. All of these programs are in the discovery stage.