Trial reportNature medicine2022
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.
3 registry-linked trialsAbstract readClinical Trial, Phase IClinical Trial, Phase II
In one paragraph
Trial report in Nature medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Cited by 55 papers.
0numbers the graph read from it
0cells of the map it votes in
55citing papers in PubMed
14.8field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral β-A-T87Q Globin Vector (LentiGlobin BB305 Drug Product)
NCT07432867 phase1 / phase2recruitingnot on this mapstarted 2026, after this paper: background citation
A Phase 1/2 Open Label Cohort Study Evaluating the Efficacy and Safety of Gene Therapy of the Sickle Cell Disease (SCD) by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains Autologous CD34+ Cells Transduced ex Vivo by the Bifunctional βAS3m/miR7m Lentiviral Vector Expressing the Therapeutical Beta-globin βAS3m and a Micro-RNA (miRNA) Targeting Specifically the Endogenous βS-globin mRNA.
Standard of Care Comparative Arm of Phase 1/2 Gene Therapy Trial DREPAMIR" in Severe Sickle Cell Disease Patients
Who cites it
55 citing papers in PubMed, 99 citations in OpenAlex.
- Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.Nature communications · 2025Trial
- Beyond Precision: A Multidimensional Framework for Selecting Genetic Medicine Platforms.Cells · 2026Review
- Thalassaemia.Nature reviews. Disease primers · 2026Review
- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- Article
- Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review.Molecular therapy. Advances · 2026Review
- Emerging Considerations in Transfusion Medicine: Hematopoietic Stem and Progenitor Cell Collection for Gene Therapy for Sickle Cell Disease and Transfusion-Dependent Thalassemia.Clinics in laboratory medicine · 2026Review
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- The Molecular Pathology of Non-Malignant Haematological Disease.British journal of biomedical science · 2026Review
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- G2B: an optimized lentiviral vector with enhanced titer and β-globin expression for improved β-thalassemia gene therapy.Blood science (Baltimore, Md.) · 2025Article
- Emerging Therapies for Sickle Cell Disease: From Symptom Management to Curative Gene Therapy.Cureus · 2025Review
- Hematopoietic stem cell therapy with gene modification to treat sickle cell disease.Stem cells translational medicine · 2025Review
- Clinical hematopoietic stem cell-based gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Two Nonmyeloablative HLA-Matched Related Donor Allogeneic Hematopoietic Cell Transplantation Regimens in Patients with Severe Sickle Cell Disease.Transplantation and cellular therapy · 2025Article
- Emerging need for a hepato-hematology program for patients with sickle cell disease in Saudi Arabia.Saudi journal of gastroenterology : official journal of the Saudi Gastroenterology Association · 2025Article
- Therapeutic gene correction of HBB frameshift CD41-42 (-TCTT) deletion in human hematopoietic stem cells.Advanced biotechnology · 2025Article
- Preclinical efficacy and safety evaluation of BD211 autologous CD34Frontiers in cell and developmental biology · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
43 authors at 10 institutions in 3 countries.
Elisa Magrin *Biotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Michaela Semeraro *Centre d'Investigation Clinique-Unité de Recherche Clinique, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Nicolas Hebert *Univ Paris Est Creteil, INSERM, EFS, IMRB, Créteil, France.ORCID http://orcid.org/0000-0003-0232-831X
Laure JosephBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Alessandra MagnaniBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Anne ChalumeauIMAGINE Institute, Université de Paris, Sorbonne Paris Cité, Paris, France.
Aurélie GabrionBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.ORCID http://orcid.org/0000-0002-1885-2071
Cécile RoudautBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Jouda MaroueneCentre d'Investigation Clinique-Unité de Recherche Clinique, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Francois LefrereBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Jean-Sebastien DianaBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.ORCID http://orcid.org/0000-0001-8806-011X
Adeline DenisIMAGINE Institute, Université de Paris, Sorbonne Paris Cité, Paris, France.
Bénédicte NevenPediatric Immunology and Hematology Department, Hôpital Necker Enfants-Malades, Paris, France.
Isabelle Funck-BrentanoPediatric Immunology and Hematology Department, Hôpital Necker Enfants-Malades, Paris, France.
Olivier NegreCEA, INSERM, Université Paris-Saclay, Division of Innovative Therapies, Institut François Jacob, Fontenay aux Roses, France.
Sylvain RenolleauPediatric Intensive Care Unit, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.ORCID http://orcid.org/0000-0002-0638-5613
Valentine BrousseDepartment of General Pediatrics and Pediatric Infectious Diseases, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.ORCID http://orcid.org/0000-0002-4984-754X
Laurent KigerUniv Paris Est Creteil, INSERM, EFS, IMRB, Créteil, France.
Fabien TouzotBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Catherine PoirotDepartment of Hematology, Fertility Preservation, Hôpital Saint Louis, Paris, France.
Philippe BourgetPharmacy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Wassim El NemerInstitut National de la Transfusion Sanguine (INTS), Paris, France.ORCID http://orcid.org/0000-0001-8184-427X
Stéphane BlanchePediatric Immunology and Hematology Department, Hôpital Necker Enfants-Malades, Paris, France.
Jean-Marc TréluyerCentre d'Investigation Clinique-Unité de Recherche Clinique, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Mohammed AsmalBluebird Bio, Inc., Cambridge, MA, USA.
Courtney WallsBluebird Bio, Inc., Cambridge, MA, USA.
Yves BeuzardUniv Paris Est Creteil, INSERM, EFS, IMRB, Créteil, France.
Manfred SchmidtGeneWerk GmbH, Heidelberg, Germany.
Salima Hacein-Bey-AbinaBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Vahid AsnafiUniversité de Paris, Institut Necker-Enfants Malades, INSERM U1151, Assistance Publique-Hôpitaux de Paris, Hôpital Necker Enfants-Malades, Paris, France.
Isabelle GuichardService de Médecine Interne, Hôpital Nord, CHU de Saint-Étienne, Saint-Étienne, Paris, France.
Maryline PoiréeDepartment of Pediatric Hematology-Oncology, Centre Hospitalier Universitaire Lenval, Nice, France.
Fabrice MonpouxUnité d'Hémato-Oncologie Infantile. Hôpital de l'Archet 2, Nice, France.
Philippe TouraineDepartment of Endocrinology and Reproductive Medicine, Assistance Publique-Hopitaux de Paris, La Pitié-Salpêtrière, and Sorbonne University, Pierre et Marie Curie School of Medicine, Paris, France.
Chantal BrouzesLaboratory of Onco-hematology, Hôpital Necker-Enfants Malades, Paris, France.
Mariane de MontalembertDepartment of General Pediatrics and Pediatric Infectious Diseases, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Emmanuel PayenCEA, INSERM, Université Paris-Saclay, Division of Innovative Therapies, Institut François Jacob, Fontenay aux Roses, France.ORCID http://orcid.org/0000-0002-1329-2327
Emmanuelle SixIMAGINE Institute, Université de Paris, Sorbonne Paris Cité, Paris, France.ORCID http://orcid.org/0000-0001-7806-0968
Jean-Antoine RibeilBiotherapy Department, Hôpital Universitaire Necker Enfants-Malades, GH Paris Centre, Paris, France.
Annarita MiccioIMAGINE Institute, Université de Paris, Sorbonne Paris Cité, Paris, France.ORCID http://orcid.org/0000-0002-3409-9665
Pablo BartolucciUniv Paris Est Creteil, INSERM, EFS, IMRB, Créteil, France.
Philippe LeboulchCEA, INSERM, Université Paris-Saclay, Division of Innovative Therapies, Institut François Jacob, Fontenay aux Roses, France. pleboulch@rics.bwh.harvard.edu.ORCID http://orcid.org/0000-0002-9196-0255
Marina CavazzanaUniversité de Paris, Paris, France. m.cavazzana@aphp.fr.ORCID http://orcid.org/0000-0002-0264-0891
Hôpital Necker-Enfants Malades · FRInserm · FRUniversité Paris Cité · FRBluebird Bio (United States) · USSorbonne Université · FRBrigham and Women's Hospital · USGene Bridges (Germany) · DEHôpital l'Archet · FRHôpital Nord · FRInstitut National de la Transfusion Sanguine · FR
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are the most prevalent monogenic disorders worldwide. Trial HGB-205 ( NCT02151526 ) aimed at evaluating gene therapy by autologous CD34
Indexed as
Genetic TherapyAdolescentAnemia, Sickle Cellbeta-ThalassemiaFemaleHumansLentivirusMaleTreatment OutcomeYoung Adult
Identifiers
What OpenQuestion holds
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Read underepoch 390
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.