You are here
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: Pfizer.com/RareDisease.