BETA-THALASSEMIA
Clinical trials for BETA-THALASSEMIA explained in plain language.
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Scientists track patients after revolutionary Gene-Editing treatment for blood disorder
⭐️ CURE ⭐️ Recruiting nowThis study follows five patients with beta-thalassemia for two years after they received an experimental gene therapy called CS-101. The goal is to monitor the long-term safety of the treatment and see if patients remain free from needing blood transfusions. Researchers will trac…
Matched conditions: BETA-THALASSEMIA
Phase: NA • Sponsor: Children's Hospital of Fudan University • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:34 UTC
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One-Time treatment aims to free patients from lifelong blood transfusions
⭐️ CURE ⭐️ Recruiting nowThis trial is testing a one-time gene therapy for people with severe beta-thalassemia who need regular blood transfusions to survive. Doctors take a patient's own blood stem cells, add a corrected gene in the lab, and then return them to the patient. The goal is to see if this tr…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Lantu Biopharma • Aim: ⭐️ CURE ⭐️
Last updated Mar 30, 2026 14:32 UTC
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One-Shot CRISPR cure trial for devastating blood diseases
⭐️ CURE ⭐️ Recruiting nowThis study is testing a single treatment using the patient's own stem cells that have been genetically modified with CRISPR technology. The goal is to see if this one-time therapy can eliminate the need for lifelong blood transfusions in beta-thalassemia patients and stop painful…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 25, 2026 14:08 UTC
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New drug trial aims to help kids with blood disorder need fewer transfusions
Disease control Recruiting nowThis study is testing a drug called luspatercept in children aged 6 to 18 with beta-thalassemia, a genetic blood disorder. Researchers want to find the safest and most effective dose and see if it can help children who need regular blood transfusions require fewer of them, or hel…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE2 • Sponsor: Celgene • Aim: Disease control
Last updated Apr 03, 2026 21:10 UTC
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Scientists test One-Time gene fix for devastating blood diseases
Disease control Recruiting nowThis early-stage study is testing the safety of a new, one-time gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. Doctors take a patient's own blood stem cells, edit a specific gene in the lab to boost healthy fetal hemoglobin, and then return…
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1 • Sponsor: Daniel Bauer • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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One-Time gene therapy aims to free patients from lifelong blood transfusions
Disease control Recruiting nowThis early-stage trial is testing a one-time gene therapy for people with severe beta-thalassemia, a blood disorder that requires lifelong regular blood transfusions. Doctors will collect a patient's own blood stem cells, add a healthy gene in the lab, and then return them after …
Matched conditions: BETA-THALASSEMIA
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Apr 03, 2026 14:41 UTC
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Extended safety watch for blood disorder drug
Disease control Recruiting nowThis study aims to understand the long-term safety of the drug luspatercept in people with certain blood disorders. It enrolls about 665 adults who have already participated in other luspatercept trials, either to continue treatment or to be monitored in long-term follow-up. The …
Matched conditions: BETA-THALASSEMIA
Phase: PHASE3 • Sponsor: Celgene • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC