Targeted Genetics' gene therapy trial for arthritis was flagged in 2003 by an advisory committee of the NIH as experts raised questions about its novel approach to the field: the use of an experimental therapy on patients who hadn't received standard therapy and little efficacy data from animal studies. But the Phase I trial involving 32 patients went forward without incident, paving the way to an expanded study of 100 patients. One of those patients became ill and died days after being dosed for the second time. The Seattle-based biotech company's approach involved injecting a huge number of genetically engineered viruses into the joints of patients with arthritis.
Company officials say that they were able to address the early concerns of the NIH advisers. Targeted Genetics is using adeno-associated viruses in the trial. AAV is being used in more than two dozen studies and is widely regarded as safe. But the death of another patient in a gene therapy trial could once again derail a field that has had a troubled history in regards to patient safety. And investigators in the field are acutely aware of the spotlight they are in.
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