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HEMOGLOBINOPATHIES (TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE)

Clinical trials for HEMOGLOBINOPATHIES (TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE) explained in plain language.

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Not yet recruiting 1

First-in-Human gene therapy trial aims to free kids from blood transfusions

Disease control Not yet recruiting

This is a very early, small study to test the safety of a single dose of an experimental gene therapy called YOLT-204. It will enroll up to 18 children and teenagers with severe, transfusion-dependent forms of sickle cell disease or beta-thalassemia. The main goals are to see if …

Matched conditions: HEMOGLOBINOPATHIES (TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE)

Phase: EARLY_PHASE1 • Sponsor: Guangzhou Women and Children's Medical Center • Aim: Disease control

Last updated Mar 17, 2026 13:08 UTC

HEMOGLOBINOPATHIES (TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE)

Get alerted when new HEMOGLOBINOPATHIES (TRANSFUSION-DEPENDENT Β-THALASSEMIA AND SICKLE CELL DISEASE) trials appear

First-in-Human gene therapy trial aims to free kids from blood transfusions