Living with AATD
Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that can cause severe, progressive lung and liver disease due to a lack of normal alpha-1 antitrypsin protein, or AAT, with varying intensity based on patient genotype and environmental factors. Patients often develop chronic obstructive pulmonary disorder, or COPD, in the lungs and cirrhosis of the liver, which can result in liver failure or death. There are an estimated 3.4 million individuals with deficiency allele combinations worldwide.
How RNA editing can help alphas
Benefits of our approach
Our proprietary RNA editing approach repairs endogenous AAT protein, which has the potential to clear protein aggregates from within liver cells to create a potentially disease-modifying, clinically differentiated benefit for liver function and to preserve lung function by providing an adequate amount of normal AAT protein.