Recruiting

Gene editing therapy for sickle cell disease and beta-thalassemia

Plain-English translation of NCT06647979 on ClinicalTrials.gov ↗ · Source last updated February 2026 · Translation generated June 2026 · How we translate trials

Read our Sickle Cell Disease research guide →

What is this trial?

This trial is testing a treatment called BCL11A enhancer gene editing, which modifies your own blood-forming stem cells to treat severe sickle cell disease or beta-thalassemia. Doctors will collect some of your stem cells, edit them in the laboratory to help your body make healthier blood cells, and then return the edited cells to your body. The goal is to reduce or eliminate the need for blood transfusions and decrease pain and other serious complications from these blood disorders.

Current treatments like hydroxyurea and blood transfusions help many patients, but they don't work for everyone or may cause serious side effects. This medication aims to offer a new option for people with severe disease who have failed or cannot tolerate standard treatments.

Do you qualify?

You likely qualify if…

you have been diagnosed with sickle cell disease (HbSS, HbS/β0-thalassemia, HbSD, or HbSO) or transfusion-dependent beta-thalassemia confirmed by genetic testing
you are between 13 and 40 years old
you have severe disease—for sickle cell, this means at least two episodes of acute chest syndrome or three or more severe pain episodes requiring hospital treatment in the past 2 years; for beta-thalassemia, at least 100 mL/kg or 10 units of blood transfusions per year
you have failed, cannot tolerate, or don't benefit from hydroxyurea therapy (standard treatment)
your blood counts, kidney function, liver function, and heart and lung function are adequate for the procedure
you do not have an identical bone marrow donor available in your family

You likely don't qualify if…

you have an active uncontrolled infection or current fever
you have been diagnosed with active cancer
you have had major surgery in the past 30 days
you have serious medical, psychiatric, or social conditions that would make it difficult to follow the study requirements
you have received a previous bone marrow or stem cell transplant

What participation looks like

If you have sickle cell disease, you will first receive blood transfusions for 3 months to prepare your body. Then, your stem cells will be collected from your blood through a procedure called apheresis (similar to donating blood). Your cells will be edited in a lab, and you will receive chemotherapy (busulfan) to prepare your body. Finally, your edited cells will be infused back into your body through an IV. You will be followed closely for 2 years after the infusion, with long-term follow-up continuing for 15 years total.

Gene editing therapy for sickle cell disease and beta-thalassemia

What is this trial?

Do you qualify?

What participation looks like

Trial locations(1 site)