Plain-English translation of NCT07432867 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle Cell Disease research guide →Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing DREPAMIR, a gene therapy that uses your own blood stem cells. Doctors remove these cells, modify them in the lab to correct the genetic problem that causes sickle cell disease, and then return the modified cells to your body. The goal is to help your body produce healthy red blood cells and reduce the painful crises and other serious complications of sickle cell disease.
Most people with sickle cell disease manage their condition with blood transfusions and medications like hydroxyurea, but these treatments don't work for everyone and come with their own risks. The only truly curative option—a bone marrow transplant from a matched sibling—is only possible for about 25% of patients. This trial exists to offer a new cure-focused option for people with severe disease who haven't responded well to standard treatments.
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If enrolled, you will have your blood stem cells collected through a process similar to donating blood. Your cells will be sent to a laboratory where they are genetically modified to produce healthy hemoglobin and correct the sickle cell defect. You will then receive chemotherapy to prepare your bone marrow, and the modified cells will be returned to your body where they should establish themselves and begin producing healthy red blood cells. You will need to visit the hospital for the initial procedures and then return for regular follow-up appointments and blood tests to monitor how well the treatment is working and watch for any side effects.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
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