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Gene Therapy
EDIT-301 for Beta Thalassemia
Recruiting1 awardPhase 1 & 2
New York, New York
This trial tests a new treatment called EDIT-301, which modifies a patient's own stem cells to treat severe beta Thalassemia. It targets adults who need regular blood transfusions. The goal is to fix their cells so they can produce healthy blood cells and reduce the need for transfusions.
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