BETA-THALASSEMIA

This early-stage study tests a new gene therapy (CS-101) for people with beta-thalassemia, a severe blood disorder requiring frequent transfusions. The treatment uses the patient's own blood stem cells, edited in a lab to produce healthy hemoglobin. The goal is to see if it is sa…

This study tests whether adding a new antibody drug (briquilimab, JSP191) to a standard blood stem cell transplant can help more people with sickle cell disease or beta-thalassemia achieve a full donor cell takeover, potentially curing their condition. About 40 participants aged …

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who already received an experimental CRISPR gene therapy called CTX001. Researchers will track participants for years to check for long-term side effects, new cancers, and how well the treatme…

This study checks the long-term safety and effectiveness of a treatment called CS-101 in 8 people with beta-thalassemia who already received it in an earlier study. Researchers will monitor serious side effects and see if patients can stay free from blood transfusions for at leas…

This study tests a single dose of CTX001, a gene therapy made from the patient's own blood stem cells edited with CRISPR, in 16 children with transfusion-dependent beta-thalassemia. The goal is to see if it can help them produce enough healthy red blood cells to stop needing regu…

This study tests a single-dose gene therapy (BRL-101) for people with transfusion-dependent beta-thalassemia, a severe blood disorder requiring regular transfusions. The treatment uses the patient's own blood stem cells, modified with CRISPR-Cas9, to help the body produce healthy…

This study follows 5 people with beta-thalassemia who already received CS-101, a gene therapy that modifies their own blood stem cells. Researchers will monitor them for years to check for side effects and see if they remain free from needing blood transfusions. No new treatment …