Gene Therapy in Patients With Mucopolysaccharidosis Disease

"Adeno-associated viral vector serotype 8 with liver-specific thyroxinebinding globulin (TBG) promoter driving the expression of the human ARSB gene. Four dose levels are available:~* Starting dose is 6x1011 gc of vector per kg of body weight;~* High dose is 2x1012 gc of vector per kg of body weight and will be administered after at least two subjects at the starting dose have experienced no DLT;~* Very high dose is 6x1012 vector per kg of body weight and will be administered after three subjects at the high dose have experienced no DLT;~* Low dose is 2x1011 gc of vector per kg of body weight. Intermediate doses are also possible. The administration of the IMP will be performed into a peripheral vein. The IMP final volume to be injected is calculated based on the patient's weight (determined on the day of hospital admission), as 3 mL/kg."