Spark’s novel gene therapy for blindness is racing to a historic date with the FDA

The $1 million medicine is coming.

On Thursday, a committee of experts invited by the FDA will meet outside Washington D.C. to review an experimental gene therapy from Spark Therapeutics (ONCE). Barring unexpected surprises, the experts will advise the FDA to approve the one-time shot, which will be used to treat a rare, inherited form of childhood blindness.

An FDA approval would be historic for both scientific and financial reasons. It would be the first true gene therapy — meant to replace or repair a defective gene — approved in the U.S.  That would end an 18-year interregnum for the gene therapy field that began, tragically, with the death of a patient in a clinical trial.

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