Genes play an essential role in determining the function of each cell in the body, making up 30 million codes of DNA. If even one of these codes is damaged, a genetic alteration may occur causing a genetic disease. Gene therapy has the potential to significantly improve the lives of people who have genetic diseases caused by genetic alterations, some of which can be debilitating and life-threatening.

Pfizer Rare Disease is focusing on highly specialized, potentially one-time gene therapy treatments and clinical trials are underway to explore the potential of gene therapy. By targeting the underlying cause of a genetic disease–at the cellular level–we hope to restore normal function in affected tissues or cells, which could potentially enable a patient to manage his or her disease without the need for ongoing treatments. If we are successful, imagine the possibilities.

To learn more about our work in gene therapy visit: