The idea behind gene therapy is simple : a person is born with a version of a gene that causes a disease, so you deliver a new gene to resolve the issue. More than 80% of rare diseases have a known genetic component. At Pfizer Rare Disease, we are exploring the potential of gene therapy to treat these rare genetic diseases through potentially life-changing innovations, trusted partnerships, and relentless passion. it has become clear that genetics may not have to determine the future of a person living with a rare disease. Gene therapy is a potentially transformative treatment approach that, if successful, may help reduce the burden of ongoing treatments for nearly 320 million people worldwide who are affected by rare genetic diseases...