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Authors
Lessard, Samuel
Rimmelé, Pauline
Ling, Hui
Moran, Kevin
Vieira, Benjamin
Lin, Yi-Dong
Rajani, Gaurav Manohar
Hong, Vu
Reik, Andreas
Boismenu, Richard
Hsu, Ben
Chen, Michael
Cockroft, Bettina M.
Uchida, Naoya
Tisdale, John
Alavi, Asif
Krishnamurti, Lakshmanan
Abedi, Mehrdad
Galeon, Isobelle
Reiner, David
Wang, Lin
Ramezi, Anne
Rendo, Pablo
Walters, Mark C.
Levasseur, Dana
Peters, Robert
Harris, Timothy
Hicks, Alexandra
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Funding
Funding for this research was provided by:
Sanofi
Biogen
Sangamo Therapeutics
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Article History
Received: 13 November 2023
Accepted: 27 September 2024
First Online: 16 October 2024
Declarations
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: AH and SL are employees of Sanofi and may hold shares and/or stock options in the company. HL, KM, BV, DL, DR, PR, RP, TH, YL, GMR, IG and VH were employees of Sanofi at the time of the study. AR, MC, and BH are employees of Sangamo and may hold shares and/or stock options in the company. RB and BC were employees of Sangamo at the time of study. NU and JT have no conflict of interest. MA receives honorarium from Bristol Myers Squibb, hold stock from Cytodine Inc, and participates in speaker bureaus for Bristol Myers Squibb, Gilead, and Abbvie. MCW has served as consultant for Ensoma, Inc., Vertex pharmaceuticals, AllCells, Inc, and BioLabs, Inc.
: The study (NCT03653247) was performed in accordance with the Declaration of Helsinki and local regulations. The study was approved by an independent ethics committee or institutional review board at each trial site (UCSF Benioff Children’s Hospital Oakland 2018–041; National Institutes of Health 526,145; Western Institutional Review Board 1–1,134,061-1/20,181,878; UC Davis Clinical Committee 1,363,599–4). All participants provided written informed consent.
