Spark Therapeutics will assume control over two clinical trials that originated at the prominent teaching hospital -- one in which patients with a rare form of blindness already have regained some vision, the other an early-stage effort to treat hemophilia B.
The move marks a coming-of-age moment for gene therapy -- the concept of treating disease by replacing or correcting faulty genes. Confined to the realm of research for decades, it now appears headed to the clinic on multiple fronts.
A gene-therapy treatment called Glybera has been approved in
"It's a bold move," Kurtzman said of the hospital's investment. "Based on the technology and the assets and the expertise that Dr. High and other people there have, I think it's a . . . very smart move."
Marrazzo said the hospital's investment was designed to support the company until it has the ability to generate revenue, a period he estimated would last "roughly a couple of years."
He said the hospital was a good partner because, unlike some private investors, its goal was not a short-term gain.
"We felt that a capital partner that had a long-term view . . . was appropriate," said Marrazzo, who has previously held top positions at biotech companies such as
Still unclear is how much gene-therapy treatments would cost for patients or their insurers. Unlike most drug treatments, gene therapy typically consists of just one injection.
"What will be interesting here is this notion of one and done," Altschuler said. "That's not something that the insurance companies have generally had experience with."
Even if such an injection were priced at tens of thousands of dollars, that could be offset easily by the savings from avoiding traditional treatments for the diseases Spark has in its sights.
With improved vision, blind children would be less likely to need a classroom aide, for example. And patients with hemophilia B, if successfully treated, would no longer need to buy synthetic blood-clotting factor, because their bodies could make it themselves.
High said an adult male with hemophilia B commonly needs to treat himself two to three times a week, at a cost of up to
Researchers have not yet disclosed final results for the handful of human patients who have received the hemophilia treatment, but High said dogs with hemophilia have been given such treatments that have lasted for up to 10 years.
Among others on Spark's scientific advisory team is
Several dozen people with mutations in a gene called RPE65 have been enrolled in that trial, now in its final phase. High estimated that in all, 1,000 people in
She stressed that her estimate was an educated guess.
"What often happens with genetic disease is, it's underdiagnosed until there's a treatment," High said. "Because people don't ordinarily seek DNA diagnosis if there's no treatment."
Now, she and her associates at Spark are betting there will be.
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