for people ages 18-35 (full criteria)
at Oakland, California and other locations
study started
completion around



The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia

Official Title

A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)


This is a Phase 1/2 single-arm, open-label, multicenter study evaluating the safety, tolerability, and efficacy of a single unit dose of EDIT-301 for autologous hematopoietic stem cell transplant in adult participants with TDT, age 18 to 35 years, inclusive


Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia, Beta-Thalassemia, CRISPR-Cas 12a, Autologous CD34+, Thalassemia, EDIT-301


You can join if…

Open to people ages 18-35

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

You CAN'T join if...

  • 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 109/L or a platelet count < 100 x 109/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


  • UCSF accepting new patients
    Oakland California 94609 United States
  • University of Minnesota accepting new patients
    Minneapolis Minnesota 55410 United States


accepting new patients
Start Date
Completion Date
Editas Medicine, Inc.
Phase 1/2 research study
Study Type
Expecting 9 study participants
Last Updated