Targeted Genetics, Mainstay of Gene Therapy, Faces Likely Shutdown

5/7/09Follow @xconomy

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early on in adeno-associated viruses, which don’t cause any cold or flu like symptoms in nature.

Targeted Genetics has also suffered a couple of painful setbacks in the past few years. Cystic fibrosis, a deadly lung disease caused by a single deficient or malfunctioning gene, was thought to be one of the first and easiest targets for gene therapy. The company invested 15 years of effort, and at least $40 million from investors and partners in that program before pulling the plug in March 2005. A clinical trial of 102 patients showed the treatment failed to help patients breathe better, even for a month.

The company moved on from that setback to rheumatoid arthritis. Targeted Genetics, using intellectual property dating to its original spin-out from Immunex, shifted its resources to a gene therapy that would deliver copies of the TNF receptor, which soaks up excess inflammatory proteins. Amgen and Wyeth currently sell etanercept (Enbrel), the world’s best-selling biotech drug with more than $5 billion in global sales, but this drug circulates throughout the bloodstream, and doesn’t work for everybody with rheumatoid arthritis. Targeted Genetics theorized that a gene therapy to deliver the TNF receptor, given directly into a “stubborn” joint or two, might help many of the millions of patients worldwide with the disease.

This treatment ran into trouble as well in August 2007. Jolee Mohr, a 36-year-old mother of a five-year-old daughter, died after receiving treatment in the gene therapy trial sponsored by Targeted Genetics—a story that was widely publicized by the Washington Post. A later investigation by the FDA, and confirmed by an expert panel of the National Institutes of Health found no reason to say the drug was responsible for Mohr’s death. Mohr, who was taking other immune-suppressing drugs at the time, died from an opportunistic fungal infection, disseminated histoplasmosis, researchers concluded.

But the cause of Mohr’s death wasn’t really settled until December 2007, when the National Institutes of Health held its recombinant DNA advisory committee. The damage had already been done to Targeted Genetics’ stock price, seriously limiting its ability raise new investment capital. The company’s stock price has been consistently below $1 in the last year, and its market capitalization had diminished to under $6 million. The company had 35 employees left on May 1, according to its quarterly report.

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