Targeted Genetics is a biotechnology company based out of Seattle, Washington. Their primary focus is to develop targeted therapies for human diseases through the application of what is called "gene therapy." Targeted Genetics uses an innocuous virus, called "Adeno-Associated Virus" (AAV), to introduce gene vectors into host cells with the purpose of correcting genetic defects or enhancing the ability of host cells to combat the sources of human disease.
Though gene therapies are the source of considerable controversy due to the scientific and regulatory hurdles that they must overcome, Targeted Genetics is a leader in the application of human gene therapy. They currently have several therapies in early-stage clinical trials, including therapies for an HIV vaccine, as well as gene therapies for treating Inflammatory Arthritis, Congestive Heart Failure, and Huntington's Disease.
Targeted Genetics recently experienced a Severe Adverse Event (SAE) in their promising Inflammatory Arthritis clinical drug trial. In July 2007, a patient died while enrolled in the clinical trial of tgAAC94, the name of the product candidate to treat arthritis. A review of the SAE, by both the National Institutes of Health Recombinant DNA Advisory Committee, or RAC, and the trial’s independent data safety monitoring board, or Safety Board, concluded that the patient’s death was not caused by the tgAAC94 product. However, concerns about the safety of gene therapy poses a serious challenge to Targeted Genetics.