TRANSFUSION DEPENDENT BETA-THALASSAEMIA

Clinical trials for TRANSFUSION DEPENDENT BETA-THALASSAEMIA explained in plain language.

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Scientists test One-Time gene fix for devastating blood diseases

Disease control Recruiting now

This early-stage study is testing the safety of a new, one-time gene-editing treatment for people with severe sickle cell disease or beta-thalassemia. Doctors take a patient's own blood stem cells, edit a specific gene in the lab to boost healthy fetal hemoglobin, and then return…

Matched conditions: TRANSFUSION DEPENDENT BETA-THALASSAEMIA

Phase: PHASE1 • Sponsor: Daniel Bauer • Aim: Disease control

Last updated Apr 03, 2026 14:43 UTC

TRANSFUSION DEPENDENT BETA-THALASSAEMIA

Get alerted when new TRANSFUSION DEPENDENT BETA-THALASSAEMIA trials appear

Scientists test One-Time gene fix for devastating blood diseases