Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia.

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:
24 Apr 2024
Historique:
medline: 24 4 2024
pubmed: 24 4 2024
entrez: 24 4 2024
Statut: aheadofprint

Résumé

Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of the erythroid-specific enhancer region of We conducted an open-label, single-group, phase 3 study of exa-cel in patients 12 to 35 years of age with transfusion-dependent β-thalassemia and a β A total of 52 patients with transfusion-dependent β-thalassemia received exa-cel and were included in this prespecified interim analysis; the median follow-up was 20.4 months (range, 2.1 to 48.1). Neutrophils and platelets engrafted in each patient. Among the 35 patients with sufficient follow-up data for evaluation, transfusion independence occurred in 32 (91%; 95% confidence interval, 77 to 98; P<0.001 against the null hypothesis of a 50% response). During transfusion independence, the mean total hemoglobin level was 13.1 g per deciliter and the mean fetal hemoglobin level was 11.9 g per deciliter, and fetal hemoglobin had a pancellular distribution (≥94% of red cells). The safety profile of exa-cel was generally consistent with that of myeloablative busulfan conditioning and autologous HSPC transplantation. No deaths or cancers occurred. Treatment with exa-cel, preceded by myeloablation, resulted in transfusion independence in 91% of patients with transfusion-dependent β-thalassemia. (Supported by Vertex Pharmaceuticals and CRISPR Therapeutics; CLIMB THAL-111 ClinicalTrials.gov number, NCT03655678.).

Sections du résumé

BACKGROUND BACKGROUND
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of the erythroid-specific enhancer region of
METHODS METHODS
We conducted an open-label, single-group, phase 3 study of exa-cel in patients 12 to 35 years of age with transfusion-dependent β-thalassemia and a β
RESULTS RESULTS
A total of 52 patients with transfusion-dependent β-thalassemia received exa-cel and were included in this prespecified interim analysis; the median follow-up was 20.4 months (range, 2.1 to 48.1). Neutrophils and platelets engrafted in each patient. Among the 35 patients with sufficient follow-up data for evaluation, transfusion independence occurred in 32 (91%; 95% confidence interval, 77 to 98; P<0.001 against the null hypothesis of a 50% response). During transfusion independence, the mean total hemoglobin level was 13.1 g per deciliter and the mean fetal hemoglobin level was 11.9 g per deciliter, and fetal hemoglobin had a pancellular distribution (≥94% of red cells). The safety profile of exa-cel was generally consistent with that of myeloablative busulfan conditioning and autologous HSPC transplantation. No deaths or cancers occurred.
CONCLUSIONS CONCLUSIONS
Treatment with exa-cel, preceded by myeloablation, resulted in transfusion independence in 91% of patients with transfusion-dependent β-thalassemia. (Supported by Vertex Pharmaceuticals and CRISPR Therapeutics; CLIMB THAL-111 ClinicalTrials.gov number, NCT03655678.).

Identifiants

DOI: 10.1056/NEJMoa2309673 PMID: 38657265
pubmed: 38657265
doi: 10.1056/NEJMoa2309673
doi:

Banques de données

ClinicalTrials.gov
['NCT03655678']

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

Copyright © 2024 Massachusetts Medical Society.

Auteurs

Franco Locatelli (F)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).
ORCID: 0000-0002-7976-3654

Peter Lang (P)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Donna Wall (D)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Roland Meisel (R)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Selim Corbacioglu (S)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).
ORCID: 0000-0003-1070-8486

Amanda M Li (AM)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Josu de la Fuente (J)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Ami J Shah (AJ)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Ben Carpenter (B)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Janet L Kwiatkowski (JL)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Markus Mapara (M)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Robert I Liem (RI)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Maria Domenica Cappellini (MD)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Mattia Algeri (M)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Antonis Kattamis (A)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Sujit Sheth (S)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Stephan Grupp (S)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Rupert Handgretinger (R)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Puja Kohli (P)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Daoyuan Shi (D)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Leorah Ross (L)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Yael Bobruff (Y)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Christopher Simard (C)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Lanju Zhang (L)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Phuong Khanh Morrow (PK)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

William E Hobbs (WE)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).

Haydar Frangoul (H)

From IRCCS Ospedale Pediatrico Bambino Gesù (F.L., M.A.) and Catholic University of the Sacred Heart (F.L.), Rome, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan (M.D.C.), and the Department of Health Sciences, Magna Graecia University, Catanzaro (M.A.) - all in Italy; University Children's Hospital Tübingen (R.H.), and the Cluster of Excellence iFIT (EXC 2180) "Image-guided and Functionally Instructed Tumor Therapies" and the German Cancer Consortium, Partner Site Tübingen, University of Tübingen (P.L.), Tübingen, the Division of Pediatric Stem Cell Therapy, Department of Pediatric Oncology, Hematology, and Clinical Immunology, Medical Faculty, Heinrich Heine University, Düsseldorf (R.M.), and the University of Regensburg, Regensburg (S.C.) - all in Germany; the Hospital for Sick Children and University of Toronto, Toronto (D.W.), and BC Children's Hospital, University of British Columbia, Vancouver (A.M.L.) - all in Canada; Imperial College Healthcare NHS Trust, St. Mary's Hospital (J.F.), and University College London Hospitals NHS Foundation Trust (B.C.) - both in London; Stanford University, Palo Alto, CA (A.J.S.); Children's Hospital of Philadelphia and Perlman School of Medicine, University of Pennsylvania, Philadelphia (J.L.K., S.G.); Herbert Irving Comprehensive Cancer Center, Columbia University (M.M.), and Joan and Sanford I. Weill Medical College of Cornell University (S.S.) - both in New York; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (R.I.L.); National and Kapodistrian University of Athens, Athens (A.K.); Vertex Pharmaceuticals, Boston (P.K., D.S., L.R., Y.B., C.S., L.Z., W.E.H.), and CRISPR Therapeutics, Cambridge (P.K.M.) - both in Massachusetts; and Sarah Cannon Research Institute at the Children's Hospital at TriStar Centennial, Nashville (H.F.).
ORCID: 0000-0002-0590-2826

Classifications MeSH