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Biogen Inc.: Biogen Announces Topline Results from Phase 3 Gene Therapy Study in Choroideremia Choroideremia is a rare inherited retinal disease that results in progressive vision loss, ultimately leading to blindness CAMBRIDGE, Mass., June 14, 2021(Nasdaq: BIIB) today announced topline results from the Phase 3 STAR study of timrepigene emparvovec (BIIB111/AAV2-REP1), an investigational gene therapy for the potential treatment of choroideremia. The STAR study did not meet its primary endpoint of proportion of participants with a =15 letter improvement from baseline in best corrected visual acuity (BCVA) at Month 12, in the interventional group in comparison to the non-interventional control group, as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart. In addition, the study did not demonstrate efficacy on key secondary endpoints. Safety results from the Phase 3 STAR study were consistent with previous studies.

United kingdomKatherine dawsonMike henckeWalter gilbertCharles weissmannHeinz schallerKenneth murrayAllison parksExchange commissionTherapeutics development unit at biogenEarly treatmentDiabetic retinopathy studyTherapeutics development unitNobel prizeToday biogenPrivate securities litigation reform act