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Fatal Outcome in Single-Patient Gene Editing Trial Raises Safety Concerns
Medical Newsgene editingbase editing

Fatal Outcome in Single-Patient Gene Editing Trial Raises Safety Concerns

By MedXY|Jul 26, 2026

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.

Cardiomyopathy Gene Therapy Reaches an Inflection Point: From AAV Delivery to Precision Genome Editing
CardiologyAAVcardiomyopathy

Cardiomyopathy Gene Therapy Reaches an Inflection Point: From AAV Delivery to Precision Genome Editing

By MedXY|May 12, 2026

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.

AAV‑OTOF Gene Therapy Restores Substantial Hearing in DFNB9 Across Ages — Rapid, Age‑dependent Benefits in a First-in‑Human Trial
newsAAVgene therapy

AAV‑OTOF Gene Therapy Restores Substantial Hearing in DFNB9 Across Ages — Rapid, Age‑dependent Benefits in a First-in‑Human Trial

By MedXY|Nov 4, 2025

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

Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results
Clinical UpdatesAAVclinical trial

Durable Efficacy and Long-term Safety of AAV Gene Therapy in Severe Hemophilia B: 13-Year Follow-up Results

By MedXY|Sep 30, 2025

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.

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