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Disease
beta-Thalassemia
Late-stage therapeutic developmentEmerging research
2
Publications
18
Clinical trials
1
Related conditions
2024
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Research2024-03-01Medical science monitor : international medical journal of experimental and clinical research
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Research2020-12-05The New England journal of medicine
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 3 clinical trials expected to report results, the earliest in Q4 2026.
Upcoming Milestones
- Q4 2026A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIA
- Q4 2027A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
- Q2 2032Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-Thalassemia
Major developments
Major trial resultsHigh impact
Results posted2026-06-23
Major trial resultsHigh impact
Results posted2026-06-23
Clinical MilestonesViewHide
Results posted
- 2026-06-23A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)Results posted
- 2026-06-23A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)Results posted
Results expected
- 2026-07-13Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-ThalassemiaResults expected Q2 2032
- 2026-07-01A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIAResults expected Q4 2026
- 2026-07-01A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-ThalassemiaResults expected Q4 2027
Recently completed
- 2026-02-09Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-ThalassemiaCompleted
- 2025-11-13A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-ThalassemiaCompleted
Activity timeline7
- 2026-07-13ClinicalMatched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-ThalassemiaResults expected Q2 2032
- 2026-07-01ClinicalA PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIAResults expected Q4 2026
- 2026-07-01ClinicalA Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-ThalassemiaResults expected Q4 2027
- 2026-06-23ClinicalA Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)Results posted
- 2026-06-23ClinicalA Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)Results posted
- 2026-02-09ClinicalEffects of Hydroxyurea and Metformin in Transfusion Dependent Beta-ThalassemiaCompleted
- 2025-11-13ClinicalA Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-ThalassemiaCompleted
Clinical trials
The current development programme across all trial phases.
Clinical programme
18
6
9
5
Late-stage studies
Recently completed
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Publications over time
20212024
Most influential
Major themes4
- Anemia, Sickle Cell1
- beta-Thalassemia1
- CRISPR-Cas Systems1
- Genetic Therapy1
Leading journals2
- Medical science monitor : international medical journal of experimental and clinical research1
- The New England journal of medicine1
Leading researchers8
- Altshuler D1
- Cappellini MD1
- Chen YS1
- Corbacioglu S1
- de la Fuente J1
- de Montalembert M1
- Domm J1
- Eustace BK1
Affiliations (unnormalised)1
- From the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial1
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A disorder characterized by reduced synthesis of the beta chains of hemoglobin. There is retardation of hemoglobin A synthesis in the heterozygous form (thalassemia minor), which is asymptomatic, while in the homozygous form (thalassemia major, Cooley's anemia, Mediterranean anemia, erythroblastic anemia), which can result in severe complications and even death, hemoglobin A synthesis is absent.
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.