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Targeted Genetics targets AIDS: Testing under way on single-shot vaccine

By Luke Timmerman
Seattle Times business reporter

	H. Stewart Parker
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AIDS kills 300 people an hour, but in the past two decades, only one biotech company has come close to developing a preventive vaccine.

But another biotech — Seattle-based Targeted Genetics — plus an academic research center and a nonprofit institution backed by the Bill & Melinda Gates Foundation are announcing today they are trying another technique.

They have begun human tests of an AIDS vaccine they hope could become the only one to protect millions with a single shot.

Fifty volunteers at a hospital in Belgium are being tested to see whether the vaccine is safe and whether it stimulates the immune system in a variety of doses.

Patients will get a shot in the arm loaded with a signature DNA snippet of HIV, the virus that causes AIDS. The vaccine is supposed to spur both major types of killer cells produced by the immune system — not just one type, as others have done — to recognize and fight HIV.

Targeted Genetics Located: Seattle, Denny Triangle Founded: 1992 Employees: 80 Chief executive: H. Stewart Parker Mission: To develop an AIDS vaccine and experimental therapies for cystic fibrosis and rheumatoid arthritis.

Animal tests have shown the vaccine can stimulate double-barreled immunity, which scientists believe is essential. Healthy animals were vaccinated then exposed to a virulent strain of HIV. They remained free of AIDS-like symptoms for three years.

More than a dozen other experimental vaccines are in development; others have failed in people after showing promise in animals.

But a leader of the International AIDS Vaccine Initiative, a Gates Foundation-backed nonprofit institution, said researchers are optimistic enough to push the single-shot method ahead. The efficiency of the approach has unusual appeal in the developing world, where a lack of basic health services makes it difficult to organize people to get a series of shots.

"This is the only AIDS vaccine that can be administered in a single shot," said Dr. Wayne Koff, senior vice president of research and development for the vaccine initiative. "We are excited about this. But this is the first step in the clinic, and it's a long run."

If the vaccine passes the safety test within a year, it will be given to hundreds, then thousands, of people in high-risk groups — such as prostitutes in sub-Saharan Africa — to judge its effectiveness. The earliest the vaccine could be widely available would be in five years, Koff said.

Many biotech and drug companies have avoided vaccine development because profits are slim compared with drugs for cancer or heart disease. But H. Stewart Parker, chief executive of Targeted Genetics, said this business model works because the nonprofit initiative is committed to financing further trials if they show promise.

Under the deal, Targeted Genetics maintains intellectual-property rights to the vaccine, agrees to make it inexpensive to the developing world and keeps rights in the potentially profitable U.S. and European markets.

The financial deal and its scientific validation are particularly important for the company, which is recovering this year after running low on cash last year.

"There obviously is increased interest in the global community on solving this problem and growing recognition from the private sector that we've got to contribute," Parker said. "We were a bit ahead of the ballgame."

Dr. Phil Johnson, president of the Ohio-based Columbus Children's Research Institute, a pioneer of the technique and the third partner in the vaccine program, said critics will wonder if the delivery virus is safe. But he said he's not worried, because Targeted Genetics has found a component of the vaccine to be safe in tests of 100 people with other diseases in the past seven years.

He's more curious to see if it can produce strong human immunity like it did in animals.

Johnson and his team are glad to see their career's work on a vaccine finally reach human testing. But ideally, he said, he'd like a more globally organized, multibillion-dollar vaccine effort, an idea the Gates Foundation proposed in Science magazine in June.

"We are really in a fix here," Johnson said. "We really need to develop a safe and effective vaccine, because every year millions of people are dying."

Luke Timmerman: 206-515-5644 or ltimmerman@seattletimes.com

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