BETA-THALASSEMIA

This early-stage study is testing a new, one-time gene therapy for people with severe beta-thalassemia, a blood disorder that requires lifelong blood transfusions. Doctors will collect a patient's own blood stem cells, add a healthy gene in the lab, and then return them. The goal…

This 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…

This 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…

This early-stage trial is testing a 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 to boost healthy fetal hemoglobin, and return the cells after chemotherapy. The …

This 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…

This 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 dose and see how the drug affects the body, with the goal of reducing the need for blood transfusions and improving hemog…

This 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 …