BETA-THALASSEMIA

This study is testing if adding a new antibody drug called briquilimab (JSP191) to a blood stem cell transplant makes the treatment more successful for people with sickle cell disease or beta-thalassemia. The transplant is a potential cure, and researchers want to see if the drug…

This early-stage study is testing a one-time treatment called CS-101 for people with beta-thalassemia, a serious inherited blood disorder. Doctors will collect a patient's own blood stem cells, edit a specific gene in the lab to boost healthy fetal hemoglobin production, and then…

This study is testing a one-time gene therapy called CTX001 in children with severe transfusion-dependent beta-thalassemia. Doctors use CRISPR gene-editing technology to modify a patient's own blood stem cells, then infuse them back, aiming to eliminate the need for lifelong bloo…

This study tracks patients for up to 15 years after they received an experimental gene therapy for beta-thalassemia, a serious inherited blood disorder. Researchers want to understand the long-term safety and effectiveness of the treatment, which uses the patient's own geneticall…

This early-stage study is testing a single infusion of a patient's own genetically modified stem cells to treat severe beta-thalassemia. The therapy uses CRISPR gene editing technology with the goal of helping the body produce healthy red blood cells. If successful, it could redu…

This study tracks the long-term safety and effectiveness of a one-time CRISPR gene therapy called CTX001 in people with sickle cell disease or beta-thalassemia. It follows 160 children and adults who have already received the therapy in earlier studies. The main goal is to monito…

This study is tracking the long-term safety and effectiveness of a gene therapy called CS-101 for people with beta-thalassemia, a serious inherited blood disorder. It will follow 8 participants who received the therapy in a previous trial for up to 15 years. The main goals are to…