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Spark Therapeutics: Calculating The Price Of A Cure

Executive Summary

John Furey, chief operating officer of Spark Therapeutics, discusses how the company has created a value framework for the first US-approved gene therapy to treat an incurable, inherited genetic condition: Luxturna  (voretegene neparvovec-ryzl).  He also highlights the remaining access and reimbursement challenges for this novel gene therapy, which is approved specifically for treatment of inherited retinal blindness from a rare RPE65 gene mutation. 
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