Optimer Pharmaceuticals

Spark Therapeutics receives FDA Breakthrough Therapy designation for SPK-RPE65

Friday, November 7, 2014

Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, has announced that its lead product candidate, SPK-RPE65, has received Breakthrough Therapy designation from the FDA. Breakthrough Therapy designation is intended to expedite the development and review of investigational therapies for serious or life-threatening conditions where initial clinical evidence demonstrates a significant improvement over existing therapies. SPK-RPE65 targets a group of blinding conditions known as inherited retinal dystrophies (IRDs) caused by autosomal recessive mutations in the RPE65 gene, and for which there currently is no pharmacologic treatment available.

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