Hemoglobinopathy clinical trials at UCSF
2 in progress, 1 open to eligible people
Hemoglobinopathy includes genetic conditions that affect hemoglobin in the blood. At UCSF, a clinical trial is underway to explore stem cell transplants for those with transfusion-dependent beta thalassemia. Researchers examine if this approach is safe and works well.
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
open to eligible people ages 18-35
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
Oakland, California and other locations
EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
Oakland, California and other locations
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