Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

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Title: Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.
Authors: Kwiatkowski JL; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA.; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Thompson AA; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA.; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Schneiderman J; Division of Hematology Oncology NeuroOncology and Stem Cell Transplantation, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL., Thuret I; Hospital Timone, Marseille, France., Kulozik AE; Department of Hematology, Oncology and Immunology, University of Heidelberg and German Cancer Research Center (DKFZ), Heidelberg, Germany., Yannaki E; G. Papanikolaou General Hospital, Thessaloniki, Greece., Cavazzana M; Biotherapy Clinical Investigation Center, INSERM Assistance Publique-Hôpitaux de Paris, Paris, France.; Pediatric Immunology, Imagine Institute, Paris, France.; Department of Biotherapy, Hospital Necker, Université Paris Cité, Paris, France., Hongeng S; Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Olson TS; Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA.; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA., Sauer MG; Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany., Thrasher AJ; Section of Molecular and Cellular Immunology, University College London Great Ormond Street Institute of Child Health, Great Ormond Street Hospital NHS Trust, London, United Kingdom., Lal A; University of California San Francisco Benioff Children's Hospital, Oakland, CA., Rasko JEJ; Royal Prince Alfred Hospital, Camperdown, Australia., Kunz JB; Heidelberg University Hospital, Heidelberg, Germany., Kinney MA; Genetix Biotherapeutics, Somerville, MA., Chawla A; Genetix Biotherapeutics, Somerville, MA., Ali S; Genetix Biotherapeutics, Somerville, MA., Tao G; Genetix Biotherapeutics, Somerville, MA., Thakar HL; Genetix Biotherapeutics, Somerville, MA., Paramore C; Genetix Biotherapeutics, Somerville, MA., Witthuhn N; Genetix Biotherapeutics, Somerville, MA., Walters MC; University of California San Francisco Benioff Children's Hospital, Oakland, CA., Locatelli F; IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.; Department of Pediatrics, Catholic University of the Sacred Heart, Rome, Italy.
Source: Blood [Blood] 2026 May 07; Vol. 147 (19), pp. 2203-2214.
Publication Type: Journal Article; Clinical Trial, Phase II; Clinical Trial, Phase I; Clinical Trial, Phase III; Multicenter Study
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1528-0020
DOI:10.1182/blood.2025029196