With One Infusion, a Path Toward Transforming Patient Lives With Gene Therapy Begins

Gene therapy offers the prospect of using one-time treatments to address an array of devastating diseases caused by genetic mutations. In July 2018, we initiated a late-stage clinical trial to help determine if one infusion of an investigational gene therapy will free hemophilia B patients from the regular injections they currently need to prevent spontaneous bleeding. And in March 2018, the first patient was dosed in a Phase 1b clinical trial for Pfizer's mini-dystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD).

Although gene therapy holds tremendous promise, the science behind it is extraordinarily complex and presents many challenges. For example, using engineered viral vectors to introduce corrected copies of missing or defective genes into a patient's body requires expertise in translating the biology of a disease into meaningful therapeutic targets, expertise in engineering the vectors for the therapeutic genes, and expertise in manufacturing. We are addressing those challenges by advancing in-house resources and expertise, creating end-to-end capabilities from research and development to manufacturing and commercialization, and collaborating with leading biotech companies in the field. While the science is undoubtedly high-tech, the most powerful testimony to its potential may one day come from a 10-year old boy who can play a Little League season without once thinking about or being sidelined by his hemophilia.