GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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CRISPR gene therapy aims to free kids from lifelong blood transfusions
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 01, 2026 23:00 UTC
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Heart cell therapy shows promise for duchenne MD in major trial
Disease control OngoingThis Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to …
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC
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CRISPR gene therapy: a Long-Term safety check for blood disease patients
Disease control ENROLLING_BY_INVITATIONThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC