BETA-THALASSEMIA
Clinical trials for BETA-THALASSEMIA explained in plain language.
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CRISPR gene therapy aims to free kids from lifelong blood transfusions
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 02, 2026 02:00 UTC
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Gene therapy could free Beta-Thalassemia patients from lifelong transfusions
Disease control OngoingThis early-stage study tests a gene therapy for people with severe beta-thalassemia who need regular blood transfusions. The treatment modifies the patient's own blood stem cells to produce healthy hemoglobin, aiming to reduce or eliminate the need for transfusions. Six participa…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Lantu Biopharma • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Gene-Editing hope for thalassemia patients: early trial launched
Disease control ENROLLING_BY_INVITATIONThis early-phase trial is testing a new gene therapy called CS-101 for people with beta-thalassemia, a blood disorder that often requires lifelong transfusions. The treatment uses the patient's own stem cells, which are modified in a lab to produce fetal hemoglobin, potentially r…
Matched conditions: BETA-THALASSEMIA
Phase: EARLY_PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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New stem cell transplant could help sickle cell patients without a perfect donor match
Disease control OngoingThis study tests a stem cell transplant using cells from a half-matched family donor, combined with low-dose radiation and drugs to prevent rejection, for adults with severe sickle cell disease or beta-thalassemia. The goal is to see if this gentler approach can replace the disea…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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CRISPR gene therapy: a Long-Term safety check for blood disease patients
Disease control ENROLLING_BY_INVITATIONThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New antibody could boost stem cell transplant success for blood disorders
Disease control OngoingThis study tests whether adding an antibody called briquilimab (JSP191) to a standard stem cell transplant can help more donor cells take root in people with sickle cell disease or beta-thalassemia. About 40 patients aged 13 and older will receive the antibody along with low-dose…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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Gene-edited cell therapy for beta-thalassemia: long-term safety check begins
Disease control ENROLLING_BY_INVITATIONThis study follows 5 people with beta-thalassemia who already received an experimental gene-edited cell therapy called CS-101. Researchers will monitor them for years to see if the treatment remains safe and whether they can stay free from blood transfusions for at least 12 month…
Matched conditions: BETA-THALASSEMIA
Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC
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8 patients with β-thalassemia tracked for years after one-time cell therapy
Disease control ENROLLING_BY_INVITATIONThis study follows 8 people with β-thalassemia who already received a one-time treatment called CS-101, which modifies their own blood cells to produce fetal hemoglobin. No new drug is given. Researchers will monitor them for years to see if they stay free of blood transfusions a…
Matched conditions: BETA-THALASSEMIA
Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC
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One-Shot gene fix for blood disorder enters human testing
Disease control ENROLLING_BY_INVITATIONThis early-stage trial tests a one-time gene therapy called BRL-101 for people with transfusion-dependent beta-thalassemia, a severe blood disorder. The treatment uses the patient's own stem cells, edited with CRISPR to boost healthy hemoglobin production. The goal is to see if a…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Bioray Laboratories • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: BETA-THALASSEMIA
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 02:00 UTC