Libmeldy 2-10 x 10^6 cells/mL dispersion for infusion

A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy

An open label, non-randomized trial to evaluate the safety and efficacy of a single infusion of OTL-200 in patients with Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD)

A single arm, open label, clinical study of cryopreserved autologous CD34+ cells transduced with lentiviral vector containing human ARSA cDNA, for the treatment of early onset Metachromatic Leukodystrophy (MLD)

Sample Collection Study to Monitor the Risk of Malignancy Due to Insertional Oncogenesis in Early Onset Patients with Metachromatic Leukodystrophy Treated with OTL-200 in the Clinical Development Program