Gene therapy has had enough trouble demonstrating efficacy in the various indications in which it is being applied without running into concerns over safety. Thus the death of a single patient in a Phase I study under a physician IND at the University of Pennsylvania's Institute for Human Gene Therapy could undermine confidence in gene therapy studies in general, especially since the death may have been avoidable under the study's protocol.

While no safety concerns were noted in the study until the final patient, who experienced multiple organ failure and died four days after receiving the gene therapy, the trial's primary purpose was to look for a dose of the adenoviral vector that achieved efficient