A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects with WAS.

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• • End-organ dysfunction, severe active infection not responsive to treatment, or other severe disease or clinical condition which, in the judgment of the investigator, would make the subject inappropriate for entry into this study. • Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome (detected via medical history check during screening). Subjects with a prior successfully treated malignancy and a sufficient follow-up to exclude recurrence (based on oncologist opinion) can be included after discussion and approval by the Medical Monitor. • Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukemia, or other serious hematological disorders. • Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin. • Previous gene therapy. • Documented human immunodeficiency virus (HIV) infection (positive HIV ribonucleic acid and/or anti-p24 antibodies).