Trial death spurs questions about gene therapy

Earlier this week [1] we reported that Targeted Genetics [2] put its lead drug trial on hold after one of the subjects in the study became seriously ill. Now the patient in question has died and FDA is investigating the death to see if the gene therapy trial--and others like it--poses serious risks to patients.

Targeted Genetics was conducting Phase I trials of tgAAC94, a potential treatment for inflammatory arthritis that is injected directly into the joints. The patient suffered undisclosed symptoms after receiving a second injection of tgAAC94, though it is as yet unknown whether the illness was a result of the therapy or a coincidence. Gene therapy works by replacing the faulty genes that cause disease with healthy ones. Many researchers believe the therapy holds great promise for treating otherwise incurable diseases such as HIV, Parkinson's, cancer and numerous immune disorders.

- read the FDA's statement [3] on the death
- see the release [4] from Targeted Genetics
- read the New York Times report [5] for more

Related Articles:
Gene therapy makes a big comeback. Report [6]
New advances made in gene therapy for HIV. Report [7]
First gene therapy for eye disease may offer a cure. Report [8]
Gene therapy cures two of advanced melanoma. Report [9]
Targeted Genetics cuts staff. Report [2]