David M. Bodine Q&A: Making Progress on Gene Therapy, Despite Targeted Genetics’ Woes

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than anything else. The private sector took a downturn in 2003 and gene therapy companies were hit by that trend. My guess is it is more of a global biotech struggle for financing than anything specific to gene therapy.

X: Has research been lost due to lack of funding?

A: It is not like less is getting done, but it is taking longer and longer to do. There are a whole bunch of projects standing on the doorstep at a critical point waiting for funding from the NIH or foundations. Foundations have been hit by the stock market, so they don’t have extra money lying around. NIH funding has been flat for the last five years at about $264 million, with just $16 million for clinical trials. So that is where we really miss the private sector. The lack of venture capital has really slowed very promising trials from getting to patients. We are grateful for the support we have gotten; we only wish there were more large companies and venture capital firms investing.

X: There are no FDA-approved gene therapies. When will we see one, and what will it be?

A: You will see it in cancer. The FDA is reviewing a gene therapy for head and neck cancer, which is great, because there really is nothing else for those people. I would guess a gene therapy for a kind of lymphoma you see in patients who have the Epstein-Barr virus is ready for FDA approval. You will probably never see an FDA approval in immune deficiencies. Those diseases are so rare, I suspect you are not going to get a drug licensed and on the market.

X: Despite this optimism, Targeted Genetics is teetering on the edge of bankruptcy. This can’t be good for the field.

A: It’s obviously a setback. Targeted was involved in sponsoring trials and providing materials for other trials, including the blindness trials in London. These researchers are excited about developing a gene therapy for glaucoma; somebody will have to make the glaucoma vector. Targeted was filling a huge void because it was one of the last companies to go. So it will be missed. But you miss them all.

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Denise Gellene is a former Los Angeles Times science writer and regular contributor to Xconomy. You can reach her at dgellene@xconomy.com Follow @

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