GENETIC DISEASES, INBORN
Clinical trials for GENETIC DISEASES, INBORN explained in plain language.
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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 17, 2026 04:55 UTC
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CRISPR gene therapy could free kids with thalassemia from lifelong blood transfusions
Disease control OngoingThis study tests a one-time gene therapy (CTX001) in 16 children with transfusion-dependent beta-thalassemia, a severe blood disorder requiring regular transfusions. The treatment uses CRISPR to modify the child's own blood stem cells so they produce healthy red blood cells. The …
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 10, 2026 13:24 UTC
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New cell therapy shows promise in slowing duchenne muscle decline
Disease control OngoingThis phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receiv…
Matched conditions: GENETIC DISEASES, INBORN
Phase: PHASE3 • Sponsor: Capricor Inc. • Aim: Disease control
Last updated Jun 09, 2026 12:10 UTC