GENETIC DISEASES, INBORN

Clinical trials for GENETIC DISEASES, INBORN explained in plain language.

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Trials to join now! 1 Not yet recruiting 1

CRISPR gene therapy offers hope for blood disorder patients

Disease control Recruiting now

This study tests a single treatment using CRISPR to modify a person's own blood stem cells, aiming to help them produce healthy red blood cells and reduce or eliminate the need for blood transfusions. It is for adults with transfusion-dependent beta-thalassemia or severe sickle c…

Matched conditions: GENETIC DISEASES, INBORN

Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control

Last updated May 01, 2026 15:57 UTC

GENETIC DISEASES, INBORN

Get alerted when new GENETIC DISEASES, INBORN trials appear

CRISPR gene therapy offers hope for blood disorder patients