ALS20

ALS20 is a novel lentiviral vector gene therapy designed for the treatment of beta-thalassemia. It works by inserting a healthy beta-globin gene into blood stem cells, enabling them to produce significantly higher levels of hemoglobin compared to other vectors currently used in clinical trials. The therapy involves collecting a patient's blood stem cells, genetically modifying them with the ALS20 vector, and then reinfusing them after chemotherapy to empty the bone marrow. ALS20 was developed at Children's Hospital of Philadelphia (CHOP) and has shown promising results in preclinical studies, producing 40-157% more adult hemoglobin than benchmark vectors at lower viral copy numbers.