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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.