The 2,000 members of the American Society of Gene Therapy meet in San Diego this week at an auspicious moment. Scientists from around the globe will present encouraging research on the use of gene therapy for a broad range of illnesses, including a rare hereditary blindness and cancer. But to the north in Seattle, as Xconomy’s Luke Timmerman reported this month, Targeted Genetics, a corporate leader in the field, is struggling to avoid bankruptcy – a clear sign that gene therapy faces significant scientific and business challenges. Xconomy’s Denise Gellene recently talked with David M. Bodine, society president and chief of the National Human Genome Research Institute’s genetics and molecular biology branch.
X: What is gene therapy?
A: Gene therapy is the introduction of genetic material into cells to replace a defective gene, or enhance a desirable property of the target cell. Typically a virus is used as a vector to carry the genetic material into the target cell.
X: Eighteen-year-old Jesse Gelsinger died in a gene therapy experiment 10 years ago this summer. Activity in the field ground to a halt amid deep concerns about safety. What is the state of gene therapy research today?
A: That was obviously a real setback for the field, but progress went on. And now 10 years later there are children in the United States and Europe who would have been dead from immune disorders who are walking around just fine. There are people with inherited blindness who have recovered significant vision, and there are literally hundreds of cancer patients who have been cured. So, there has been tremendous progress, but it comes the way most progress comes, which is very incrementally, from proof of concept to development. It is still hard to get the genes where you want them, and to get them to do what you need them to do.
X: Is the field growing or shrinking? Is it attracting people and investment?
A: My personal feeling is that the field is growing, but it is growing in academia – that is where the trials are going on. The venture capital money that was invested in gene therapy has pretty much dried up. Most of the trials you hear about are funded with grant money from an institution like NIH or Howard Hughes Medical Institute, but it’s not really venture capital money anymore. We have the academic researchers but have kind of lost the biotech ones.
X: Why has venture money abandoned the field?
A: I think it is the economy more