Tag: #gene-therapy

Two 2025 Pediatric Deaths in China Linked to Experimental Gene-Therapy Trials Raise Transparency Questions

Two children died in separate experimental gene-therapy trials in China in 2025; disclosures in 2026 have raised questions about safety oversight and reporting.

#gene-therapy, #china, #clinical-trials, #research-ethics

FDA expands Casgevy gene therapy to children as young as 2 for sickle cell and beta-thalassemia

FDA clears Vertex’s Casgevy for children aged 2+ with sickle cell disease or transfusion-dependent beta-thalassemia, widening access to a one-time gene therapy.

$VRTX, #sicklecell, #gene-therapy, #casgevy, #vertex, #fda