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Article History

Received: 19 December 2024

Accepted: 27 February 2025

First Online: 27 March 2025

Declarations

:

: Haydar Frangoul has been a consultant for Editas Medicine, Rocket Pharmaceuticals, and Vertex Pharmaceuticals; served on a data monitoring and safety committee for Rocket Pharmaceuticals; served on a steering committee for Vertex Pharmaceuticals; and has received honoraria from Jazz Pharmaceuticals. Franco Locatelli has been a consultant for Amgen, Bellicum Pharmaceuticals, Novartis, Novimmune, and Sobi, Inc; speaker bureau for Bluebird bio and Gilead Sciences; and served on a steering committee for Vertex Pharmaceuticals. Michael Eckrich has been a member of an advisory board for Vertex Pharmaceuticals. Suzan Imren, Nanxin Li, and Fengjuan Xuan are employees of Vertex Pharmaceuticals Incorporated and may hold stock and/or stock options in the company. Stephan Grupp has been a consultant for Novartis, Jazz, Adaptimmune, Allogene, Cabaletta, Verismo, Eureka, and Vertex Pharmaceuticals; received honoraria and research funding from Novartis; served on the advisory boards/committees for Allogene and McKesson.

: The CLIMB SCD-121 trial was conducted in accordance with the Declaration of Helsinki, local applicable laws and regulations, and current Good Clinical Practice Guidelines as described by the International Council for Harmonization. Each patient or patient’s legal guardian provided informed consent, with assent obtained when age appropriate. An independent data-monitoring committee is reviewing safety data throughout the study.