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The purpose of this Phase 1/2 study is to evaluate the safety, tolerability and efficacy of EDIT-301 (renizgamglogene autogedtemcel, reni-cel) in adults with transfusion-dependent beta thalassemia. The therapy is given in the setting of an autologous hematopoietic stem cell transplant using the participant's own cells. Key eligibility includes documented homozygous or compound heterozygous β-thalassemia, including β-thalassemia/hemoglobin E (HbE), in the medical record, plus a history of transfusion of at least 100 mL/kg per year.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: 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 Key Exclusion Criteria: * 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