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Spark's Vision Loss Gene Therapy Raises US FDA Questions About Novel Endpoint

Executive Summary

Multi-luminance mobility testing was developed by firm to measure functional vision across a range of lighting levels in patients with RPE65 mutations; advisory committee also will consider minimum patient age and clinical stage of disease appropriate for subretinal injections with voretigene.

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Spark's Early Christmas Present: US FDA Approves Luxturna For Vision Loss

Review and approval of first directly administered gene therapy targeting a disease caused by genetic mutations embodies 'spirit' of agency's forthcoming series of guidance documents on gene therapies, FDA Commissioner Gottlieb says; documents are likely to encourage development of the kind of novel and clinically meaningful endpoint that Spark used in its clinical program.

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

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's Gene Therapy Luxturna Sails Through US FDA Panel

Improvements seen in multi-luminance mobility testing in the voretigene Phase III trial were clinically meaningful, panelists said, endorsing the novel endpoint for the vision loss treatment. They also were persuaded by first-hand accounts from patients.

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