Re-dosable, titratable genetic medicines

Glycogen storage disease type 1a (GSD1a) is the most common genetically inherited glycogen storage disease. GSD1a is caused by the deficiency of glucose-6-phosphatase (G6Pase) which results in the inability to regulate blood sugar. GSD1a affects approximately 6,000 people worldwide.

Hemophilia A is a rare, inherited bleeding disorder resulting from a deficiency in factor VIII, a protein crucial for normal clotting. More than 150,000 people worldwide are affected by hemophilia A.

Progressive familial intrahepatic cholestasis (PFIC) is a rare inherited liver disease in which liver cells are less able to secrete bile, causing progressive liver disease in affected people. There are about one in 50,000 to one in 100,000 people living with PFIC worldwide.

Phenylketonuria, or PKU, is a rare inherited metabolic disorder caused by a defect in the gene called PAH, which helps create the enzyme needed to break down phenylalanine. This results in the accumulation of phenylalanine to levels that are toxic to the central nervous system. PKU impacts about 16,500 people in the U.S., while prevalence outside the U.S. varies.