Targeted Genetics Survives Brush With Death, Sells Gene Therapy IP to Genzyme for $7M

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were a lot of patents and proprietary know-how on manufacturing of adeno-associated viruses (AAV). These are tiny viruses that don’t cause illness in humans, like the more common adenovirus. For this reason, AAVs are thought by many researchers to be the ideal delivery vehicle to efficiently transmit genes into cells. By purchasing intellectual property around these AAVs, Genzyme could apply these techniques to its own pipeline of experimental gene therapies, which includes one early-stage treatment for Parkinson’s disease.

“Genzyme has a substantial commitment to advancing gene therapy as a solution to many complex diseases,” said David Meeker, executive vice president for Genzyme’s therapeutics, biosurgery and transplant businesses, in a statement.. “The acquisition of the intellectual property from Targeted Genetics will add depth to our program and overall gene therapy capabilities.”

The deal also isn’t putting Targeted Genetics out of business by forcing it to hand over the store. Targeted Genetics will retain the development rights to its own pipeline of gene therapies, which includes treatments for a rare form of blindness known as Leber’s Congenital Amaurosis, Huntington’s disease, and inflammatory arthritis. The blindness treatment wowed gene therapy researchers and landed ink in the New England Journal of Medicine based on the first few patients.

The results were so impressive that when researchers gathered in San Diego at the American Society of Gene Therapy in June, they had sober talks about what would happen to the drug if Targeted Genetics ran out of cash; ultimately researchers concluded it would still go on. But because the company has raised money from Genzyme, Targeted Genetics will now be around long enough to see results from another clinical trial at University College London that will generate more results in 2010, Robinson says.

Robinson knew all about Genzyme’s interest in gene therapy, having once worked there previously as the director of business development for its molecular oncology business before she joined Targeted Genetics. She took over as Targeted Genetics’ CEO when founder H. Stewart Parker resigned in November of last year. Within weeks, Genzyme was one of the companies Robinson talked to first about a possible partnership or deal to keep her company afloat.

Those talks ended up dragging on for about nine months, right when Targeted Genetics’ back was up against the wall. She sounded relieved on the other end of the phone last night.

“For me, the important thing is to be out of crisis mode,” Robinson says. “Now we can say all of this intellectual capital and know-how isn’t going to be lost.”

That’s good news for gene therapy researchers, and may give Genzyme an edge it needs to sustain an interest in gene therapy amid all the failures. For Targeted Genetics as a company, $7 million isn’t nearly enough to develop a new drug. What matters is that it now has a chance to really get back on its feet. “We can now look forward,” Robinson says.

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