Pfizer's Rare Disease team is focusing on highly specialized, potential gene therapy treatments, and clinical trials are underway to explore the potential of gene therapy. By targeting the underlying cause of a genetic disease, Pfizer hopes 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 Pfizer is successful, imagine the possibilities.

How do you translate disease biology into potential gene therapies? Scientists from Pfizer's Rare Disease Research Unit explain how they are developing custom-made viral vectors that may be deployed to different parts of the body to treat genetic diseases.

Who are the players involved in bringing gene therapies from concept to patient? Leaders from Pfizer's rare disease research unit discuss the multi-faceted collaboration needed to realize the promise of gene therapy.

How do you make a gene therapy? Pfizer's bioprocess and manufacturing teams in Research Triangle Park and Sanford, NC, give an inside look at what it takes to produce potential gene therapies from research to clinical to commercial scales.

To learn more about our work in gene therapy visit: Pfizer.com/RareDisease.