Thousands of patients have now received experimental gene therapies. Target indications for gene therapies include genetic and metabolic diseases, cancer, acquired diseases such as AIDS, and cardiovascular disease. The field of gene therapy continues to focus its efforts on patients with severe and life-threatening diseases who usually have few treatment options or who have failed all available therapies. Even after a decade of research and clinical development, many of the gene therapy clinical trials that are active today are in an early stage of development. These trials continue to be in early development not because of problems establishing a safety profile, but because researchers are exploring their options with indications, routes of administration, dosing regimes, patient populations, combination therapies and novel vectors.
Gene therapy is subject to greater oversight than virtually all other technology.
BIO recommends that any system of oversight for gene therapy be memorialized in an agreement between the key federal agencies and industry that will provide the agencies with safety data while ensuring that patient confidentiality and trade secret data is protected. For more than a decade, the academic and industrial research communities have observed a voluntary moratorium on gene therapy procedures that would affect the germ line cells the egg and sperm that pass on genetic composition to future generations.