Lantu Biopharma

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 study is observing how well a gene therapy called Vesemnogene works for spinal muscular atrophy (SMA) in real-world settings in lower-income countries. Researchers will follow 15 patients with SMA who receive this one-time treatment to monitor their safety and see if they ac…