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`Holy Grail' Seen as Gene Drug for Blind Produces Lasting Effect

A gene therapy maker showed this week it could make blind children and adults see. But the big question left is how long the effect will last.

Much depends on the answer, including how much the company can charge for the drug -- a price tag some say could be more than $1 million a patient.

On Saturday, the Philadelphia biotech firm, Spark Therapeutics Inc., reported encouraging data on that front. Spark’s gene therapy, which treats certain rare inherited eye disorders that cause blindness, has maintained for three years the improved vision of patients who completed an early-stage trial, the company said.

In a widely anticipated presentation on Saturday at a conference in Paris, Spark also revealed more details of a final-stage trial, showing how it helped improve vision in 31 people. Together, the two data sets may help Spark’s treatment become the first gene therapy to market in the U.S.

After years of setbacks, a resurgence of gene therapy -- curing diseases by injecting new genes into the body -- has come this year from improved technology and promising early trials. Spark, along with biotech companies like UniQure NV, Bluebird Bio Inc. and Applied Genetic Technologies Corp., are all developing treatments for disorders caused by mutations in a patient’s genetic code -- inherited eye disorders, hemophilia and sickle cell anemia are leading candidates.

"It’s a really cool moment," said Jeff Marrazzo, Spark’s chief executive officer, by telephone Friday. Earlier in the week, Spark had announced that the final-stage trial was positive, without providing details. “I think the amount of e-mails I got from other gene therapy CEOs indicates everyone was very happy."

Marrazzo, 37, was part of the group that founded Spark, which was a built on work from a team of researchers at the University of Pennsylvania and the Children’s Hospital of Philadelphia. The company went public on Jan. 30, and shares are up more than double their IPO price, valuing the company at $1.21 billion.

Both pieces of data Spark has released this week show that “with lots of expertise, if you pick the correct target, and run a well controlled study, and get all the little details right, you can do this," he said on his way to the airport to attend the Retina Society Annual Scientific Meeting in Paris, where the three-year data was revealed.

Spark’s gene therapy “is the hottest thing in medicine right now," said Julia Haller, ophthalmologist-in-chief at the Wills Eye Hospital and president of the Retina Society. Correcting genetic defects that make people sick “has been the holy grail for a long time and now we are a huge step closer." Haller, who was an unpaid consultant for the final-stage trial, spoke in an interview before the new results were presented.

Moonless Night Walk

Spark’s treatment, SPK-RPE65, is injected directly into the eye...