Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial.

Authors:
MacLaren RE; Fischer MD; Gow JA; Lam BL; Sankila EK and 5 more

Journal:
Nat Med

Publication Year: 2023

DOI:
10.1038/s41591-023-02520-3

PMCID:
PMC10579095

PMID:
37814062

Journal Information

Full Title: Nat Med

Abbreviation: Nat Med

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Molecular Biology

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

Transparency Score
4/6
66.7% Transparent
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Core Indicators
Evidence found in paper:

"data availability trial results are publicly accessible at the eudract website ( https://www clinicaltrialsregister eu/ctr-search/trial/2015-003958"

Code Sharing
Evidence found in paper:

"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)."

Evidence found in paper:

"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."

Protocol Registration
Open Access
Paper is freely available to read
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Tool: rtransparent

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Last Updated: Aug 05, 2025