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Article History

Received: 22 January 2024

Accepted: 19 September 2024

First Online: 18 October 2024

Competing interests

: Dr. C Lee reports advisory board participation for Sanofi, Kite, Kadmon, BMS, and Incyte; research funding from Incyte; steering board committee participation for Incyte; speakers panel participation for Kite; and consulting for Fresenius Kabi, Sanofi, and Mallinckrodt. Dr. Sharma reports Grant funds from CRISPR Therapeutics; Consulting fees from Spotlight Therapeutics, Medexus Inc., Vertex Pharmaceuticals, Sangamo Therapeutics and Editas Medicine; and is the St. Jude Children’s Research Hospital site principal investigator of clinical trials for genome editing of sickle cell disease sponsored by Vertex Pharmaceuticals/CRISPR Therapeutics (NCT03745287), Novartis Pharmaceuticals (NCT04443907) and Beam Therapeutics (NCT05456880). The industry sponsors provide funding for the clinical trial, which includes salary support paid to Dr. Sharma’s institution. Dr. Sharma has no direct financial interest in these therapies. Dr. Nishihori reports significant payments (research support) to the institution for clinical trial (Novartis), research support (drug supply only) to the institution for clinical trial (Karyopharm) and advisory board participation for Medexus. Dr. Kitko reports compensation for ad board for Horizon Therapeutics and Incyte and compensation for participation in CME lectures about GVHD for Sanofi, Physicians Education Resource and i3Health. Dr. Pidala reports consulting and advisory board membership for Syndax, CTI Biopharma, Amgen, Regeneron, and Incyte; and clinical trial support for Novartis, Amgen, Takeda, Janssen, Johnson and Johnson, Pharmacyclics, Abbvie, CTI Biopharma, and BMS. Dr. S Lee reports compensation (research funding) from Amgen, AstraZeneca, Incyte, Kadmon, Pfizer, Syndax; steering committee participation for Incyte; drug supply for Janssen; consulting for Mallinckrodt, Equillium, Kadmon, Novartis and Sanofi; and National Marrow Donor Program Board of Directors (uncompensated). All other authors declare no competing interests.

: This study was approved the NMDP Institutional Review Board, registration number IRB00001253. All patients provided written informed consent for research participation and data submission, in accordance with the Declaration of Helsinki.