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Biogen’s inherited eye disease gene therapy misses the mark in late-stage trial

June 15, 2021

The Phase III STAR study enrolled 169 adult males with a genetically confirmed diagnosis of choroideremia. It evaluated the safety and efficacy of a single subretinal injection of the investigational gene therapy.

The primary endpoint was the proportion of patients with an improvement of at least 15 letters from baseline in best corrected visual acuity at 12 months post-treatment, as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart.

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