Regeneron's AAV1-hOTOF Gene Therapy Shows Promise in Treating Hereditary Deafness

May 28, 2024, 08:34 AM

Recent advancements in gene therapy have shown promising results in treating hereditary deafness. Biotech giant Regeneron has successfully enabled two children to hear for the first time through a single shot in their ear. These early-stage trials, focusing on autosomal recessive deafness 9, have demonstrated the safety and efficacy of the treatment. The Lancet has published findings from the first-in-human clinical trial, confirming the potential of gene therapy to transform the lives of approximately 30 million children born deaf. The AAV1-hOTOF gene therapy was tested in a single-arm trial. However, this breakthrough has sparked a debate within the deaf community, with some expressing concerns about the cultural and social implications of a 'cure' for deafness, fearing it could deplete their communities and make them feel endangered.