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, or CFF, the median age at death for patients with CF in the United States was 29.6 years in 2016. 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 1,000 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.
OTC deficiency is a metabolic liver enzyme disorder that results from a mutation in the OTC gene, and is the most common urea cycle disorder. The OTC enzyme is necessary for preventing the accumulation of ammonia, a normal byproduct of protein breakdown. When the enzyme is defective or absent, high levels of ammonia accumulate in the blood, which can cause serious and irreversible neurological damage.
Based on published research, the incidence of OTC deficiency is estimated to be 1 in 56,500 live births in the United States. OTC deficiency is an X-chromosome-linked disease, and females are typically less severely affected than males.
We are developing an mRNA therapeutic designed to deliver fully functional OTC enzyme to the liver to enable hepatocytes, the predominant type of liver cell, to produce normal OTC enzyme.
Rare Liver, Lung and CNS Diseases
We believe that our MRT 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 lung, liver, eye, CNS and lymphatic tissues. All of these programs are in the discovery stage.