Genetic Medicine

Gene therapies

After decades of research, a variety of gene therapy approaches have made it to market. Gene therapies aim to provide a permanent cure rather than just managing a disease. Specifically, the goal is to fix an inherited genetic defect. 

Medical experts believe that the new gene therapy approaches will be useful in treating a growing number of diseases. Since tumor growth often has genetic causes, approximately two-thirds of all gene therapies are being tested for use in cancer medicine, according to recent estimates of the Journal of Gene Medicine. Other therapeutic areas are monogenic disorders caused by a single gene defect (>11%), infectious diseases (>5%), cardiovascular diseases (>5%) and neurological disorders (>1%). Many inherited diseases have a prevalence of 1 000 to 10 000 patients.

Three gene therapies are currently approved in the USA and four in the EU. Approximately 2 800 such therapies are undergoing clinical trials worldwide. More than half of these candidates are in the first phase of clinical development, with only a few being tested in pivotal regulatory studies.