We don’t follow footsteps. We create the path.
Our mission at Spark Therapeutics is to challenge the inevitability of genetic disease by discovering, developing and delivering potential treatments in ways unimaginable—until now. We have built a fully integrated company, combining our proprietary adeno-associated viral (AAV) gene therapy platform with excellence in R&D, manufacturing and commercial operations. Find out more about how we strive to turn genes into medicines for patients with inherited diseases, including inherited retinal diseases (IRDs), liver-directed diseases such as hemophilia, and neurodegenerative diseases.