Β-THALASSEMIA MAJOR

Clinical trials for Β-THALASSEMIA MAJOR explained in plain language.

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Trials to join now! 1 Not yet recruiting 1

Gene therapy could free blood disease patients from transfusions

Disease control Not yet recruiting

This study tests a single dose of CS-101, a gene therapy, in 20 people aged 12 to 35 with β-thalassemia major, a severe blood disorder requiring regular transfusions. The goal is to see if it is safe and can help patients produce enough healthy hemoglobin to stop needing transfus…

Matched conditions: Β-THALASSEMIA MAJOR

Phase: PHASE2 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control

Last updated May 12, 2026 13:41 UTC

Β-THALASSEMIA MAJOR

Get alerted when new Β-THALASSEMIA MAJOR trials appear

Gene therapy could free blood disease patients from transfusions