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- SAA that is refractory to or has relapsed while on standard of care IST, as defined in the protocol
- Hematopoietic stem cell transplantation (HSCT) is not available or suitable as a treatment option or has been refused by the patient
- Adequate hepatic and renal function as defined in the protocol
- Diagnosis of Fanconi anemia as defined in the protocol
- Evidence of myelodysplastic syndrome as defined in the protocol
- Paroxysmal nocturnal hemoglobinuria (PNH) with evidence of significant hemolysis or history of PNH-associated thrombosis
- Treatment with a T cell-depleting agent (eg, ATG or alemtuzumab) within 6 months prior to dosing
- Treatment with a calcineurin inhibitor (eg, cyclosporine) within 4 weeks prior to dosing
- Treatment with eltrombopag or investigational thrombopoietin receptor agonist, Granulocyte Colony-Stimulating Factor (G-CSF), or an androgen (eg, danazol), within 2 weeks prior to dosing
- HIV, hepatitis B or hepatitis C positive by serological testing at the screening visit
- Active tuberculosis, latent tuberculosis infection (LTBI) or history incompletely-treated tuberculosis or LTBI
Note: Other protocol-defined inclusion/ exclusion criteria apply.