Hemoglobinopathy clinical trials at UCSF
3 in progress, 2 open to eligible people
Hemoglobinopathy is a blood disorder. UCSF is studying how EDIT-301 can help people with severe sickle cell disease. Another trial is looking at how EDIT-301 can be used in stem cell transplants for Beta Thalassemia.
EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
open to eligible people ages 12-50
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
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
In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)
Sorry, accepting new patients by invitation only
The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.
San Francisco, California
Our lead scientists for Hemoglobinopathy research studies include Tippi Mackenzie.
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