Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial.
Journal Information
Full Title: Nat Med
Abbreviation: Nat Med
Country: Unknown
Publisher: Unknown
Language: N/A
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Subject Category: Molecular Biology
Available in Europe PMC: Yes
Available in PMC: Yes
PDF Available: No
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"data availability trial results are publicly accessible at the eudract website ( https://www clinicaltrialsregister eu/ctr-search/trial/2015-003958"
"Competing interests R.E.M. and M.D.F. have received grant support, clinical trial support and consultancy fees from Biogen. R.E.M. is a named inventor on the BIIB111 patent licensed by the University of Oxford. B.L.L. has received grant support and consultancy fees from Biogen. E.-M.K.S. has received clinical trial support from Biogen. A.G. was an employee of Nightstar at the time of the study. J.A.G., S.P., D.Y. and G.Z. were employees of Biogen at the time of the study and may hold stock in the company. M.E.P. has received consultancy fees from Biogen and grant funding from Research to Prevent Blindness (New York) to Casey Eye Institute (unrestricted grant)."
"This study was funded by Biogen. Biogen acquired Nightstar Therapeutics in 2019. Nightstar Therapeutics designed the trial with the academic authors before the acquisition. Biogen provided timrepigene emparvovec to trial sites, analyzed the data and funded medical writing support for manuscript development. The authors thank the STAR study investigators who participated in the conduct of the study. The full list of STAR study investigators and their affiliations is included in Supplementary Table 1. The authors also thank C. Lu, E. Meunier and J. Liu for their contributions to this study. Writing and editorial assistance was provided under the direction of the authors by M. Das of MedThink SciCom and funded by Biogen."
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Last Updated: Aug 05, 2025