Spark strides ahead in race for first genetic disease gene therapy

13 October 2017

A unanimous vote by a US Food and Drug Administration (FDA) advisory committee on Thursday has put a Philadelphia-based biotech in pole position to reach market with the USA’s first gene therapy for a genetic disease.

Spark Therapeutics (Nasdaq: ONCE) halted trading of its common stock as the meeting took place on Thursday, but shares opened 6% up on Friday at $91.60 following the vote of the agency’s Cellular, Tissue and Gene Therapies Advisory Committee.

The clinical program for the company’s investigational drug Luxturna (voretigene neparvovec) includes up to four years of efficacy data. It has been recommended as a one-time gene therapy for patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD) – a progressive form of blindness that usually starts in childhood.

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