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A 6-year-old girl died seven days after receiving a personalised base-editing therapy for a rare genetic disorder in an unregistered Chinese trial, prompting international concern about oversight.
Gene therapy for cardiomyopathy is moving from concept to clinic, but durable benefit will depend on solving delivery, immunogenicity, cargo, and safety barriers in the failing human heart.
A single‑arm trial of AAV‑OTOF (Anc80L65) in 10 patients (1.5–23.9 years) with autosomal recessive deafness 9 showed good tolerability and rapid, clinically meaningful hearing gains, with optimal outcomes in children aged 5–8 years; longer
A single AAV gene therapy infusion for severe hemophilia B led to sustained factor IX expression, substantial reduction in bleeding and factor use, and no late safety concerns over 13 years.
