First gene therapy approved for children with metachromatic leukodystrophy

FDA has approved atidarsagene autotemcel (Lenmeldy), the first gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD). MLD is caused by a deficiency of the enzyme arylsulfatase A (ARSA), which leads to a buildup of sulfatides (fatty substances) in cells. Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic stem cells, which have been genetically modified to include functional copies of the ARSA gene.