Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Sickle-cell disease

The number of subjects with immune-related AEs (e.g., autoimmune disorders, graft-versus-host disease, opportunistic infections, HIV), The number of subjects with new or worsening hematologic disorders, The number of subjects with new or worsening neurologic disorders, The number of subjects with malignancies

Vaso-Occlusive Events Endpoints: The proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15, The proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15, Annualized number of severe VOEs over time through Year 15, Annualized number of VOEs over time through Year 15, Change from parent study baseline in annualized number of severe VOEs over time through Year 15, Hematologic Endpoints: Assessment of the following over time post-drug product infusion through Year 15: − total Hb − non-transfused total Hb − HbS percentage of non-transfused total Hb − HbAT87Q percentage of non-transfused total Hb − non-HbS percentage of non-transfused total Hb, Change from parent study baseline through Year 15 in the following hemolysis markers: absolute reticulocyte count, % reticulocytes/erythrocytes, total bilirubin, indirect bilirubin, haptoglobin, and lactate dehydrogenase, Change from parent study baseline through Year 15 in the following markers of iron stores: serum ferritin and liver iron content

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