GenSight Undeterred By Gene Therapy’s Placebo Puzzle
Phase III success at 96 weeks sets up GenSight blindness therapy for EU filing, but puzzle of placebo arm benefits remain
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Company boosted by animal study which helps answer riddle of improvement in untreated eye.
Novartis/Spark’s gene therapy Luxturna has been OKd for NHS use by NICE in record time thanks to early engagement and close collaboration between the two parties.
The company's gene therapy didn't hit the primary endpoints in either of its two Phase III studies in a severe genetic retinal disease reported so far, but a third trial designed through a special protocol assessment with the US FDA is ongoing.