OTL-200 + busulfan

**OTL-200 + busulfan** is a combination therapy regimen for the treatment of metachromatic leukodystrophy (MLD), consisting of OTL-200, an autologous gene therapy product (atidarsagene autotemcel), and busulfan, an alkylating agent used as a conditioning regimen. OTL-200 contains autologous CD34+ hematopoietic stem and progenitor cells, transduced ex vivo with a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. After the patient’s own stem cells are collected and genetically modified to express functional ARSA, the patient undergoes myeloablative conditioning with busulfan to ablate the bone marrow and facilitate engraftment of the gene-modified cells. The modified cells are then infused intravenously. The therapy aims to provide a durable source of functional ARSA enzyme, addressing the underlying cause of MLD. Busulfan is included specifically to enable efficient engraftment of gene-corrected cells. OTL-200 has shown significant preservation of motor and cognitive function in early-onset MLD patients and is classified as an Advanced Therapy Medicinal Product (ATMP) in the EU[1][2][3][4][5].

Other names

atidarsagene autotemcel + busulfanOTL200 + busulfan