1. Gene therapy for hemoglobinopathiesHemoglobinopathies are genetic disorders that affect the structure or production of the hemoglobin molecule, which is responsible for transporting oxygen in the blood. The most common hemoglobinopathies include sickle cell disease and thalassemia, which affect more than 330,000 children around the world every year, including more than 100,000 patients with sickle cell disease in the U.S.
“The gold standard included a medication that helped prevent. Despite adherence and taking the medication, we still see patients coming in [with complications],” Dr. Rabi Hanna, Cleveland Clinic pediatric hematology department chair, said at the virtual Cleveland Clinic Medical Innovation Summit. “Only a small portion of patients can undergo a transplant from another transplant patient.”
New research in hemoglobinopathies has brought an experimental gene therapy to give the potential ability to make functional hemoglobin molecules and reduce the presence of sickled blood cells or ineffective red blood cells in thalassemia to prevent associated complications.
“Gene therapy will allow a patient to get a transplant for your own self. Take the cells, repair the gene through technology,” Hanna said. “I am very pleased to say there are many pharmaceutical companies that are undergoing clinical trials. This is really the fruit of many decades of technology improvement.”