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A 2022–2024 nonblinded cohort study found OTOF gene therapy produced rapid, durable gains in auditory thresholds, speech perception, mismatch-negativity responses, and music perception versus cochlear implantation in children with genetical
DB-OTO, a hair-cell-specific dual-AAV gene therapy encoding full-length human otoferlin, achieved dose-dependent and sustained auditory restoration in OTOF-deficient mice, supporting an ongoing Phase I/II pediatric trial.
This study demonstrates the long-term effectiveness and safety of autologous gene therapy for treating ADA deficiency, showing sustained immune function and minimal adverse effects over a median follow-up of 7.5 years.
Regeneron’s DB-OTO gene therapy shows promising results in restoring natural hearing in children with profound hearing loss caused by OTOF gene mutations, with 75% achieving significant hearing improvement after a single treatment.
Recent clinical trials report encouraging tumor shrinkage with KRAS G12D inhibitor VS-7375, significant progression-free survival benefit in soft tissue sarcoma with ozekibart, and a novel gene therapy acquisition targeting wet AMD.
A phase 3 trial demonstrates prademagene zamikeracel significantly accelerates healing and reduces pain in large, chronic RDEB wounds, offering a promising gene therapy with a favorable safety profile.
Gene therapy using autologous, gene-corrected stem cells without conditioning offers sustained engraftment and reverses bone marrow failure in Fanconi anaemia-A, with a favorable safety profile.
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.
