Thalassaemia: who works on it

One card per institution. Filter by target, by country, or show only institutions in EU-13 (Horizon Europe widening) countries.

• All targets
• G6PD 0
• IDH 0
• TP53 0
• CD19 2
• IL-17 0
• JAK 0
• CTLA-4 0
• FGFR 0
• GLP-1 0
• HDAC 1
• HIF-1 0
• HMGB1 0
• IL-23 0
• IL-6 0
• LAG-3 0
• NLRP3 0
• TIM-3 0

EU-13 is a filter, not a ranking: it only narrows the list.

24 of 492 institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Terminated

  Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

  NCT06219239· Institute of Hematology & Blood Diseases Hospital, China· Updated 4 Nov 2026

  “Suboptimal treatment response”Registry text, quoted unchanged. Source

• Terminated

  Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

  NCT06041620· Institute of Hematology & Blood Diseases Hospital, China· Updated 4 Nov 2026

  “Suboptimal treatment response”Registry text, quoted unchanged. Source

• Terminated

  A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia

  NCT07681440· Bristol-Myers Squibb· Updated 2 Jul 2026

  “Insufficient enrollment”Registry text, quoted unchanged. Source

• Terminated

  Siplizumab for Sickle Cell Disease Transplant

  NCT06078696· Columbia University· Updated 10 Jun 2026

  “Closed by the industry collaborator”Registry text, quoted unchanged. Source

• Terminated

  A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia

  NCT04925206· EdiGene (GuangZhou) Inc.· Updated 30 Mar 2025

  “Development plan change”Registry text, quoted unchanged. Source

• Terminated

  A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.

  NCT05752123· EdiGene (GuangZhou) Inc.· Updated 30 Mar 2025

  “Development plan change”Registry text, quoted unchanged. Source

• Terminated

  Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

  NCT02179359· Masonic Cancer Center, University of Minnesota· Updated 3 Mar 2025

  “Replaced by another study”Registry text, quoted unchanged. Source

• Terminated

  In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

  NCT02986698· University of California, San Francisco· Updated 27 Jan 2025

  “Enrollment ended early due to interim data suggesting a lack of efficacy. Interim data did demonstrate that IUHSCT was safe and well tolerated, but the PI and DSMB agreed that enrollment should not continue under the current treatment protocol.”Registry text, quoted unchanged. Source

• Suspended

  Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells

  NCT05442346· Bioray Laboratories· Updated 28 Feb 2024

  “Sponsor decision”Registry text, quoted unchanged. Source

• Withdrawn

  Isoquercetin in Sickle Cell Anemia

  NCT04474626· Jeffrey Zwicker, MD· Updated 11 Jan 2021

  “Trial did not activate”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia

  NCT07884773· Khyber Medical University Peshawar· Source

• A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety of Luspatercept in subjects who have participated in other Luspatercept (ACE-536) clinical trials.

  2022-502498-40-00· Celgene Corp.· Source

• A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

  NCT06609226· Novo Nordisk A/S· Source

• A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

  NCT05664737· Bristol-Myers Squibb· Source

• Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

  NCT00061568· National Heart, Lung, and Blood Institute (NHLBI)· Source

• A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

  NCT05891249· Bristol-Myers Squibb· Source

• Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

  NCT02105766· National Heart, Lung, and Blood Institute (NHLBI)· Source

• Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia

  NCT07721480· Prince of Wales Hospital, Shatin, Hong Kong· Source

• Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

  NCT04143724· Celgene· Source

• A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

  NCT06421636· Regeneron Pharmaceuticals· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Single-Cell Analysis 1
• CRISPR-Cas Systems 18
• Gene Expression Profiling 3
• Whole Genome Sequencing 3
• Exome Sequencing 12
• Proteomics 7
• Metabolomics 3
• Mass Spectrometry 1
• Flow Cytometry 1
• Immunohistochemistry 1
• Magnetic Resonance Imaging 30
• Machine Learning 11
• Deep Learning 2
• Cohort Studies 17

• ILF3 regulates erythroid differentiation: A single-cell transcriptomic analysis and cellular experiment.

  PLoS One, Sept 2026

• Multiomics analysis of red blood cells reveals thalassemia severity beyond globin gene mutations.

  Blood Adv, May 2026

• The induction effect of hydroxyurea and metformin on fetal globin in the K562 cell line.

  Mol Med, Apr 2025

See which targets of this disease your abstract mentions, and go to the people working on them.