atidarsagene autotemcel

Atidarsagene autotemcel (formerly known as OTL-200) is an ex vivo autologous hematopoietic stem cell gene therapy developed for the treatment of metachromatic leukodystrophy (MLD), a rare and fatal inherited metabolic disorder. The therapy involves harvesting the patient’s own hematopoietic stem cells, which are then genetically modified outside the body using a lentiviral vector to insert functional copies of the ARSA gene. These corrected cells are infused back into the patient after myeloablative conditioning. The introduced ARSA gene enables production of functional arylsulfatase A enzyme, addressing the underlying enzymatic deficiency in MLD and aiming to halt or slow disease progression. Atidarsagene autotemcel has demonstrated preservation of motor function and cognitive development in most treated patients compared to natural history cohorts, with long-term follow-up showing sustained benefits and a favorable safety profile[2][4][7].

Brand names

LenmeldyLibmeldy

Other names

atidarsagene autotemcel