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Spark set to file eye disease gene therapy next year

SPK-RPE65 shows significant benefit in functional vision for patients with retinal dystropies
Spark Therapeutics

A gene therapy for the eye disease retinal dystrophy has cleared a phase III trial, setting developer Spark Therapeutics on course to file for approval in 2016.

The late-stage study of SPK-RPE65 was able to provide a significant benefit in functional vision compared to placebo in patients with retinal dystrophies caused by a mutation in the RPE65 gene, which codes for a pigment found in the epithelial cells lining the retina.

It is estimated that these inheritable mutations account for around 11% of patients who develop early-onset retinal degeneration, a broad group of disorders that result in progressive loss of vision and in some cases can lead to blindness. If approved, SPK-RPE65 could eventually achieve sales in excess of $1.5bn, according to analysts.

The trial enrolled 31 patients with confirmed RPE65 mutations, with 21 patients receiving an injection of Spark's gene therapy into the eye - in a bid to insert a functional copy of the RPE65 gene into retinal cells - and 10 patients acting as a control group.

Subjects enrolled into the trial were evaluated over the course of a year for their ability to navigate around a mobility course under various light conditions, a task which can be challenging for retinal dystrophy patients. Other measures included visual acuity and light-sensitivity threshold testing.

SPK-RPE65 achieved a significant improvement in the ability of patients to navigate the course, as well as improvements on secondary endpoints including light sensitivity. However, the therapy was unable to show significant improvement on visual acuity.

Shares in Spark rocketed up more than 50% yesterday on the results, which put the company in pole position in the race to bring the first gene therapy through to commercialisation in the US. In Europe that milestone was passed in 2012 when UniQure's $1m therapy Glybera was approved to treat lipoprotein lipase deficiency (LPLD).

The eye has been an early target for gene therapies as delivery sidesteps systemic administration so is relatively straightforward, and Spark is one of several companies taking this route to market for various ophthalmic indications.

Others include Bristol-Myers Squibb-backed Eyevensys, GenSight Biologics (supported by Novartis), Oxford Biomedica, Avalanche Biotechnologies, Applied Genetic Technologies, and ReGenxbio. 

Article by
Phil Taylor

6th October 2015

From: Research



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