AAV – Medxy AI

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

By MedXY 05/12/2026

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.

news Otorhinolaryngology Pediatrics

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

By MedXY 11/04/2025

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 follow‑up is required.

Clinical Updates Hematology-Oncology news Specialties

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

By MedXY 08/01/2025

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.