A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Inclusion criteria

• ✓. an older sibling affected by MLD (index case), whose age of symptom onset was \<=6 years of age (i.e., had not celebrated 7th birthday). Subjects will be classified as LI, EJ or intermediate LI/EJ based on age of symptom onset in the index case and their ARSA genotype; LI: symptom onset in index case \<=30 months of age and genotype typically 0/0; EJ: symptom onset in index case \>30 months and \<= 6 years of age with genotype typically 0/R; Intermediate LI/EJ: symptom onset in index case \<= 6 years of age but unable to unambiguously characterize index case as LI or EJ OR
• ✓. if MLD is diagnosed in a pre-symptomatic child without an older affected sibling, (e.g.) incidentally or via newborn screening) and the totality of the data available to the investigator strongly suggest that the subject has an early onset variant of MLD likely to benefit from gene therapy, and the subject is \<=6 years of age (i.e., has not celebrated 7th birthday), the subject may be considered eligible after discussion and approval by the Orchard medical monitor.