Gene therapy study for sickle cell disease and thalassemia
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This study tests an experimental gene therapy called YOLT-204 for people with sickle cell disease or thalassemia. It aims to see if the treatment is safe and can help reduce symptoms, and participants will be followed long-term.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 14 and 35 years old.
- You must have a confirmed diagnosis of sickle cell disease (types HbSS, HbSβ0, or HbSβ+) or thalassemia that requires regular blood transfusions.
- You need to have medical records showing your transfusion history for the past 2 years.
- Your major organs (heart, lungs, liver, kidneys) must be functioning normally enough for the study.
- You must agree to use effective contraception during the study and be willing to participate in long-term follow-up.
- If you have sickle cell disease, you must have had at least 2 severe pain crises or other serious complications in the past year despite standard care.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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