Recruiting

Gene editing treatment for severe sickle cell disease

Plain-English translation of NCT07708350 on ClinicalTrials.gov ↗ · Source last updated October 2026 · Translation generated October 2026 · How we translate trials

Read our Sickle Cell Disease research guide →

What is this trial?

This pilot study is testing a new approach to treating severe sickle cell disease by editing your own bone marrow stem cells using a technology called BCL11A enhancer gene editing. The edited cells are then returned to your body to help produce healthy blood and reduce sickle cell complications. This treatment is being tested in a small group of young people (ages 13–35) whose disease has not improved enough with current medications.

Current treatments like hydroxyurea don't work well enough for everyone with severe sickle cell disease, and not all patients have access to bone marrow transplants from a matched donor. This trial explores whether editing your own cells—rather than relying on a donor transplant—could offer a safer alternative to control the disease.

Do you qualify?

You likely qualify if…

you are between 13 and 35 years old
you have been diagnosed with sickle cell disease (HbSS, HbS/B0 thalassemia, HbSD, or HbSO genotypes)
you have severe disease, defined as at least two acute chest syndrome episodes in the past 2 years, or three or more severe pain crises requiring hospital treatment and opioid medication in the past 2 years
you have tried hydroxyurea for at least 6 months without adequate benefit, or you cannot tolerate it due to side effects
you do not have an HLA-matched related bone marrow donor available
your blood counts, kidney, liver, heart, and lung function meet the study requirements

You likely don't qualify if…

you currently have an active infection or fever
you have been diagnosed with cancer
you have had a major surgery in the past 30 days
you have previously undergone a bone marrow or stem cell transplant
you have medical, psychiatric, or social circumstances that would prevent you from completing the study visits and follow-up

What participation looks like

You will receive blood transfusions for 3 months to prepare your body, then your stem cells will be collected from your blood through a process called apheresis. Your cells will be sent to the lab for gene editing while a backup sample is preserved. After conditioning chemotherapy with busulfan, you will receive an infusion of your edited cells back into your body. You will then be followed closely for 24 months after the infusion, with long-term follow-up visits for 15 years total to monitor your health and how well the treatment works.

Gene editing treatment for severe sickle cell disease

What is this trial?

Do you qualify?

What participation looks like

Trial locations(1 site)