Alpha-1 Antitrypsin Deficiency

The Diseases:

  • Genetic disease that causes defective production of the Alpha-1 Antitrypsin (A1AT) protein, which leads to lung and liver disease
  • Affects about one in 1,500 to 3,500 individuals with European ancestry
  • Current standards of care are weekly intravenous infusions of functional A1AT protein (obtained from human donor plasma) and lung or liver transplant for severe cases

The Editas Approach

Our genome editing approach starts by deleting the gene in the liver in order to prevent liver disease, followed by gene correction in the liver to address both liver and lung disease.