Gene editing therapy for severe beta-thalassemia
Part of Blood & lymphatic clinical trials.
This study tests an experimental gene-editing treatment called CS-101 for people with severe beta-thalassemia who need regular blood transfusions. The goal is to see if it can reduce or eliminate the need for transfusions by helping the body make more healthy red blood cells.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be between 12 and 35 years old.
- You must have a confirmed diagnosis of beta-thalassemia major and have needed at least 8 blood transfusions in the past year.
- Your blood counts before transfusions must be very low (hemoglobin 70 or below).
- You must be generally healthy enough to undergo stem cell collection and the gene-editing procedure.
- If you are of childbearing age, you must agree to use effective birth control during the study.
- You cannot have had a prior stem cell transplant, gene therapy, or certain other treatments like thalidomide, hydroxyurea, or luspatercept recently.
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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