Lead Sponsor

Editas Medicine, Inc.

Age

18 - 35 years

Sex

ALL

Healthy Volunteers

No

•Diagnosis of Transfusion Dependent B-Thalassemia as defined by:
•Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and
•History of at least 100 mL/kg/year or 10 U/year of packed red blood cell (RBC) transfusions in the 2 years prior to signing informed consent
•Clinically stable and eligible to undergo autologous HSCT
•Karnofsky Performance Status ≥ 70

•Available 10/10 human leukocyte antigen (HLA)-matched related donor
•Prior HSCT or contraindications to autologous HSCT
•Participants with associated a history of α-thalassemia and \> 1 alpha chain deletion, or alpha multiplications as documented in medical records
•Participants with a history of other inherited hemoglobinopathy or thalassemic mutation (Hb S, C, D or other) as documented in medical records
•Prior receipt of gene therapy
•Inadequate bone marrow function, as defined by white blood cell count of \< 3 x 10\^9/L or a platelet count \< 100 x 10\^9/L (without hypersplenism), per investigator judgement
•Inadequate organ function
•Advanced liver disease
•Any prior or current malignancy, or immunodeficiency disorder,
•Immediate family member with a known or suspected Familial Cancer Syndrome
•Clinically significant and active bacterial, viral, fungal, or parasitic infection

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