“You’ve got to believe that every time someone gives a pitch to an investor, the investor will say, ‘What are you doing about manufacturing?’” Dr. Wilson said. The whole development enterprise has become nerve-racking, researchers said. “You don’t know until the end that you have a product that is good enough to be used in a treatment,” said Dr. David Williams of Harvard. Or, as officials at Bluebird Bio can attest, whether you have any product at all. The company was formed in 2010, hoping to show that gene therapy could work in adrenoleukodystrophy, a rare and fatal neurodegenerative disease that strike boys. That was before the virus production logjam had begun, and all seemed well. Bluebird gave a virus manufacturer its recipe for making needed viruses. Then, said Nick Leschly, the company’s chief executive, he got bad news. Using Bluebird’s recipe, the manufacturing company said it was going to cost Bluebird a million dollars to create enough viruses to treat one patient. The company scurried to find ways to improve the efficiency of its recipe. Finally, they were ready to start anew. Manufacturing began, but months later there was nothing to show for it. “We got no virus,” Mr. Leschly said. “It was an Apollo 13 moment,” he added. “We put everyone in a room and said, ‘We have to figure this out. Everything at the company is now stopped. Nothing can be done without virus.’” They finally found the source of the problem — the acidity of the solution used to grow the viruses was slightly off, killing them.
The expanding field of gene therapy is being hampered by a lack of disabled viruses with which to test new treatments, with some companies forced to wait years for a supply. Companies that create bespoke viruses are deluged with orders, but the process of creation is laborious, requires extensive documentation and is prone to setbacks. In an effort to speed up production, scientists at Oxford University have developed a virus called PMLV, or “polymyxin-producing virus”, which they say could be used in clinical trials