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The Luxturna Model: Spark’s Approach To Retinal Disease Endpoint Embraced In Gene Therapy Guidance

Executive Summary

Established efficacy endpoints may not be suited to assessing gene therapies’ clinically meaningful effects in some rare retinal disorders, US FDA says in draft guidance that gives a shout-out to Spark’s development of a functional vision endpoint for voretigene; guidance also includes recommendations on appropriate control groups and use of sham injections.

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