From the Dana–Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School (E.B.E., L.E.L., A.B., C.B., M.F.C., B.M., K.B., S.-Y.P., W.B.L., C.D., M.M.H., D.A.W.), the Harvard Stem Cell Institute, Harvard Medical School (A.B., C.B.), the Gene Therapy Program, Dana–Farber/Boston Children's Cancer and Blood Disorders Center (A.B., M.F.C., B.M., E.M., A.F., S.-Y.P., C.D., D.A.W.), the Division of Hematology, Brigham and Women's Hospital, Harvard Medical School (M. Achebe), the Connell and O’Reilly Families Cell Manipulation Core Facility, Dana–Farber Cancer Institute (H.D., R.K., K.S., H.N., S.N., J.R.), the TransLab, Boston Children's Hospital (D.A., M. Armant), and the Department of Laboratory Medicine, Boston Children's Hospital, Harvard Medical School (J.P.M.) — all in Boston; and Bluebird Bio, Cambridge, MA (O.N.).
Address reprint requests to Dr. Williams at 300 Longwood Ave., Karp 08125.3, Boston, MA 02115, or at [email protected].
Supported by a grant (5R01HL137848) from the National Heart, Lung, and Blood Institute, National Institutes of Health. The GMP vector was produced and furnished by Bluebird Bio as part of a licensing agreement between Boston Children's Hospital and Bluebird Bio, and as part of the agreement, Bluebird Bio prereviewed an earlier version of the manuscript.
Dr. Esrick reports receiving grant support, paid to Boston Children's Hospital, and steering committee fees from bluebird bio, and grant support, paid to Boston Children's Hospital, from Sangamo Therapeutics; Dr. Achebe, receiving advisory board fees from Fulcrum Therapeutics, Global Blood Therapeutics, and Pharmacosmos; Dr. Shaw, receiving consulting fees from Orchard Therapeutics; Dr. O. Negre, being employed by and owning shares in bluebird bio; Dr. Ritz, receiving advisory board fees from Aleta Biotherapeutics, Celgene, Falcon Therapeutics, LifeVaultBio, Rheos Medicines, Talaris Therapeutics, and TScan Therapeutics, grant support from Amgen, Equillium, and Kite, fees for serving on a data and safety monitoring board from AvroBio, and consulting fees from Infinity Pharmaceuticals; Dr. London, receiving fees for serving on a data and safety monitoring board from Jubilant Draximage and Merck; Dr. Heeney, receiving consulting fees, advisory board fees, and clinical trial support from AstraZeneca and Novartis, fees for serving on a data and safety monitoring board from CRISPR Therapeutics and Vertex Pharmaceuticals, advisory board fees and clinical trial support from Cyclerion and Global Blood Therapeutics, advisory board fees from Forma Therapeutics, and consulting fees and clinical trial support from Micelle Biopharma (Sancilio & Co), and Pfizer; Dr. Manis, receiving advisory board fees from Sanofi US Services; Dr. Williams, receiving advisory board fees from Beam Therapeutics and Geneception, grant support and advisory board fees from bluebird bio and Orchard Therapeutics, consulting fees from Emerging Therapy Solutions, serving as an advisor for Novartis, serving as cofounder of Alerion Biosciences, and serving as site investigator for a trial sponsored by Sangamo. No other potential conflict of interest relevant to this article was reported.
Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.
This article was published on December 5, 2020, and updated on December 8, 2020, at NEJM.org.
A data sharing statement by the authors is available with the full text of this article at NEJM.org.
We thank current and previous employees of Bluebird Bio for helpful discussions; Mursal Hassan and Pauleen Faynberg for administrative assistance; Pei-Chi Kao for assistance with statistics and graphics; Stephanie Patriarca for clinical research coordination assistance; Ellen Proeung and the BCH Therapeutic Apheresis Unit for transfusion-related patient care; BCH Hematology and HSCT providers for inpatient and post-transplantation clinical care; the hematology teams at Massachusetts General Hospital, Hasbro Children's Hospital, Phoenix Children's Hospital, and Wyckoff Heights Medical Center for patient care collaboration; and David G. Nathan, Stuart Orkin, Vijay Sankaran, Franklin Bunn, and Orah Platt for critical review of an earlier version of the manuscript and for helpful discussions.
