Transfusion-dependent β-thalassemia (TDT, also known as β-thalassemia major) - Healing Genes

Transfusion-dependent β-thalassemia (TDT, also known as β-thalassemia major)

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A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

A Phase 1/2, Open-label, Single-arm Study to Assess the Safety, Tolerability, and Efficacy of ST-400 Autologous Hematopoietic Stem Cell Transplant for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Phase 1/2


Researchers in CA, GA, MA, and MN are recruiting patients with Transfusion-Dependent Beta-thalassemia to undergo a single dose treatment with gene-edited stem cells that will produce fetal hemoglobin, which is lacking due to the presence of BCL11A gene. The stem cells will be drawn from the patient, modified in the lab and then infused back to the patient after a chemotherapy treatment.

This investigatory treatment aims to assure the safety of the treatment, and to assess its impact on hemoglobin levels and transfusion requirements. Participants will be followed for up to 3 years after the treatment.


  • Be ≥18 and ≤40 years of age
  • Genetic confirmation of the beta-TDT diagnosis
  • Able and willing to use contraception during the treatment and for one year following
  • Not be HIV-positive, pregnant or breastfeeding, malignancy in the past 5 years
  • Not have received previous gene therapy


  1. Prescreening at the study site to confirm eligibility of the patient to participate
  2. Apheresis, or collection of the patient’s blood stem cells, will be conducted at the study site
  3. The gene-editing phase of the study will not require patient involvement
  4. Chemotherapy treatment will reduce the patient’s bone marrow
  5. One-time Infusion of the investigational drug made from the patient’s cells, with observation period
  6. Follow up at the study site location at a regular schedule


Primary contact: Medical Monitor | [email protected] | 510-307-7266


The following locations are participating in the trial:

University of California, Los Angeles | Los Angeles, CA

UCSF Benioff Children’s Hospital Oakland | Oakland, CA

Children’s Healthcare of Atlanta | Atlanta, GA

Dana-Farber Boston Children’s Cancer and Blood Disorders Center | Boston, MA

University of Minnesota | Minneapolis, MN


Sangamo Therapeutics & Bioverativ Therapeutics Inc.


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