Betibeglogene Autotemcel Gene Therapy for Non-β

Adolescent Adult Biological Products / adverse effects Busulfan / therapeutic use Child Erythrocyte Transfusion / adverse effects Erythropoiesis Female Genetic Therapy / methods Genetic Vectors Genotype Hemoglobins / analysis Humans Iron Overload / prevention & control Lentivirus / genetics Male Middle Aged Myeloablative Agonists / therapeutic use beta-Globins / genetics beta-Thalassemia / blood

Journal

The New England journal of medicine
ISSN: 1533-4406
Titre abrégé: N Engl J Med
Pays: United States
ID NLM: 0255562

Informations de publication

Date de publication:
03 02 2022
Historique:
pubmed: 11 12 2021
medline: 17 2 2022
entrez: 10 12 2021
Statut: ppublish

Résumé

Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with the BB305 lentiviral vector encoding the β-globin (β In this open-label, phase 3 study, we evaluated the efficacy and safety of beti-cel in adult and pediatric patients with transfusion-dependent β-thalassemia and a non-β A total of 23 patients were enrolled and received treatment, with a median follow-up of 29.5 months (range, 13.0 to 48.2). Transfusion independence occurred in 20 of 22 patients who could be evaluated (91%), including 6 of 7 patients (86%) who were younger than 12 years of age. The average hemoglobin level during transfusion independence was 11.7 g per deciliter (range, 9.5 to 12.8). Twelve months after beti-cel infusion, the median level of gene therapy-derived adult hemoglobin (HbA) with a T87Q amino acid substitution (HbA Treatment with beti-cel resulted in a sustained HbA

Sections du résumé

BACKGROUND
Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with the BB305 lentiviral vector encoding the β-globin (β
METHODS
In this open-label, phase 3 study, we evaluated the efficacy and safety of beti-cel in adult and pediatric patients with transfusion-dependent β-thalassemia and a non-β
RESULTS
A total of 23 patients were enrolled and received treatment, with a median follow-up of 29.5 months (range, 13.0 to 48.2). Transfusion independence occurred in 20 of 22 patients who could be evaluated (91%), including 6 of 7 patients (86%) who were younger than 12 years of age. The average hemoglobin level during transfusion independence was 11.7 g per deciliter (range, 9.5 to 12.8). Twelve months after beti-cel infusion, the median level of gene therapy-derived adult hemoglobin (HbA) with a T87Q amino acid substitution (HbA
CONCLUSIONS
Treatment with beti-cel resulted in a sustained HbA

Identifiants

DOI: 10.1056/NEJMoa2113206 PMID: 34891223
pubmed: 34891223
doi: 10.1056/NEJMoa2113206
doi:

Substances chimiques

Biological Products 0
Hemoglobins 0
Myeloablative Agonists 0
beta-Globins 0
betibeglogene autotemcel 0
Busulfan G1LN9045DK

Banques de données

ClinicalTrials.gov
['NCT02906202']

Types de publication

Clinical Trial, Phase III Journal Article Multicenter Study Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

415-427

Commentaires et corrections

Type : CommentIn

Informations de copyright

Copyright © 2021 Massachusetts Medical Society.

Auteurs

Franco Locatelli (F)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Alexis A Thompson (AA)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Janet L Kwiatkowski (JL)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

John B Porter (JB)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Adrian J Thrasher (AJ)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Suradej Hongeng (S)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Martin G Sauer (MG)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Isabelle Thuret (I)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Ashutosh Lal (A)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Mattia Algeri (M)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Jennifer Schneiderman (J)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Timothy S Olson (TS)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Ben Carpenter (B)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Persis J Amrolia (PJ)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Usanarat Anurathapan (U)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Axel Schambach (A)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Christian Chabannon (C)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Manfred Schmidt (M)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Ivan Labik (I)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Heidi Elliot (H)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Ruiting Guo (R)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Mohammed Asmal (M)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Richard A Colvin (RA)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.

Mark C Walters (MC)

From IRCCS Ospedale Pediatrico Bambino Gesù, Sapienza, University of Rome, Rome (F.L., M. Algeri); Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago (A.A.T., J.S.); Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia (J.L.K., T.S.O.); University College London Hospital (J.B.P., B.C.) and University College London Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust (A.J.T., P.J.A.) - all in London; Ramathibodi Hospital, Mahidol University, Bangkok, Thailand (S.H., U.A.); the Department of Pediatric Hematology, Oncology, and Stem Cell Transplantation in Children (M.G.S.) and the Institute of Experimental Hematology (A.S.), Hannover Medical School, Hannover, and GeneWerk, Heidelberg (M.S., I.L.) - both in Germany; Hôpital de la Timone (I.T.) and Institut Paoli-Calmettes Comprehensive Cancer Center (C.C.) - both in Marseille, France; the University of California, San Francisco, Benioff Children's Hospital, Oakland (A.L., M.C.W.); and the Division of Hematology-Oncology, Boston Children's Hospital, Harvard Medical School, Boston (A.S.), and Bluebird Bio, Cambridge (H.E., R.G., M. Asmal, R.A.C.) - all in Massachusetts.
ORCID: 0000-0002-6515-4559

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Adolescent › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Personnes › Tranches d'âge › Adulte › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Mélanges complexes › Produits biologiques › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Composés chimiques organiques › Composés du soufre › Acides du soufre › Acides sulfoniques › Acides alcanesulfoniques › Alcanesulfonates › Méthanesulfonates › Busulfan › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Thérapeutique › Biothérapie › Transfusion sanguine › Transfusion de composants du sang › Transfusion d'érythrocytes › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Phénomènes physiologiques respiratoires et circulatoires › Phénomènes physiogiques du sang › Hématopoïèse › Érythropoïèse › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Vecteurs génétiques › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Phénomènes génétiques › Génotype › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Hémoprotéines › Globines › Hémoglobines › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Betibeglogene Autotemcel Gene Therapy for Non-β
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questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Hémoprotéines › Globines › Hémoglobines › Sous-unités de l'hémoglobine › Globines bêta › Betibeglogene Autotemcel Gene Therapy for Non-β
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Hémoglobinopathies › Thalassémie › bêta-Thalassémie › Betibeglogene Autotemcel Gene Therapy for Non-β