Trial Information This study is the first in-human clinical trial to explore the safety, tolerability, and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA patients. This...
ABO-101 Gene Therapy Transfer | Type IIIB | Phase I-II | Abeona
March 9, 2020
Adeno-associated virus serotype 9 carrying the human NAGLU gene under the control of a CMV enhancer/promoter (rAAV9.CMV.hNAGLU) will be delivered one time through a venous catheter inserted into a patient’s peripheral limb vein. A tapering course of prophylactic enteral prednisone or prednisolone will be administered for a period of at least two months. As of July 2020, patients are still being recruited. There are four trial locations globally, including one in the United States (Ohio). The others are in France, Germany, and Spain. Read this clinical trial’s information on ClinicalTrials.gov, which includes enrollment criteria, trial locations, and study contact information.