The USFDA has approved the first gene therapy, atidarsagene autotemcel, for treating children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). MLD is a rare genetic disease affecting the brain and nervous system. It is caused by arylsulfatase A enzyme deficiency, which leads to the accumulation of sulfatides in the cells.
The building up of sulfatides damages the central and peripheral nervous system, resulting in loss of cognitive and motor function and early death. Atidarsagene autotemcel is made from autologous hematopoietic stem cells with the insertion of one or more functional copies of arylsulfatase A cDNA. Clinical evidence In a clinical trial conducted on 39 children with MLD (age: 8 to 140 months), atidarsagene autotemcel significantly reduced the risk of severe motor impairment or…
