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How To Build An Endpoint: Spark Designed Testing Maze To Study Rare Vision Loss

 
• By Sue Sutter

In an interview, Spark President Katherine High explains that multi-luminance mobility test used in clinical development of Spark's gene therapy voretigene sprang from 2011 FDA meeting on need to study functional vision in patients with retinal degenerative diseases. Experience highlights need for new endpoints when targeting previously untreated diseases, she says.

Spark Therapeutics Inc.'s experience with a new endpoint for testing Luxturna (voretigene neparvovec) in a rare vision disorder highlights the possibility that gene therapies and other novel therapeutics, particularly in the rare disease space, may require the development of equally novel efficacy assays.

Spark developed the multi-luminance mobility test (MLMT) to measure improvements in functional vision because existing efficacy assays did not directly address the visual deficits seen in voretigene's target population –

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