BETA-THALASSEMIA

This study tests whether adding a new antibody drug (briquilimab, JSP191) to a standard stem cell transplant can help more people with sickle cell disease or beta-thalassemia achieve a full donor blood system, potentially curing their condition. About 40 participants aged 13 and …

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 …

This study checks the long-term safety and effectiveness of a gene therapy called CS-101 in 8 people with beta-thalassemia who already received the treatment. Researchers will watch for side effects and see if patients can remain transfusion-free for at least a year. No new treat…

This early-stage study tests a new gene-edited stem cell treatment (CS-101) for people with beta-thalassemia, a severe blood disorder requiring frequent transfusions. Ten participants aged 6 to 35 will receive their own genetically modified stem cells to see if it can safely redu…

This study tests a one-time gene-editing treatment (CTX001) in 16 children with transfusion-dependent beta-thalassemia, a severe blood disorder. The therapy uses the child's own stem cells, modified with CRISPR-Cas9, to produce healthy red blood cells. The main goal is to see if …

This 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). Researchers will monitor participants for years to check for side effects, new cancers, and how well the thera…

This study tests a one-time gene therapy called BRL-101 for people with transfusion-dependent β-thalassemia, a severe blood disorder requiring regular transfusions. The therapy uses the patient's own blood stem cells, modified with CRISPR-Cas9, to help the body produce healthy re…