Page Last Updated Feb. 15, 2021 LYS-SAF302 is a gene therapy by Lysogene which is intended to deliver a working copy of the SGSH gene to deliver the gene therapy to the patient's brain. The normal gene is placed inside the shell of a non-disease-causing virus...
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.