Medicosnext
In a groundbreaking development, a Chinese company, Refreshgene Therapeutics, has demonstrated promising results in an experimental gene therapy aimed at restoring hearing in children with congenital deafness. Published in The Lancet, the clinical trial reported significant success, with five out of six children experiencing hearing recovery and enhanced speech recognition six months after undergoing the gene therapy. The participants, all afflicted with profound deafness due to mutations in the OTOF (otoferlin) gene, saw notable improvements in their hearing abilities and speech perception.
The OTOF gene mutations are identified as the cause in 2% to 8% of congenital deafness cases. The therapy involved using a harmless virus to introduce a modified version of the human OTOF gene into the inner ears of the children during surgical procedures at the Eye & ENT Hospital of Fudan University. The researchers observed exceptional progress in the children’s hearing capabilities while most side effects were minor and temporary. However, one child did not respond as expected, prompting speculation that potential leakage of the gene therapy solution during or after surgery might be a contributing factor.
Refreshgene is actively collaborating with OBiO Technology Shanghai on a gene therapy addressing neovascular age-related macular degeneration. This marks a significant milestone in the pursuit of treatments for various forms of hearing loss. Researchers remain optimistic about the potential impact of these therapies, highlighting the absence of effective treatments for deafness since the invention of cochlear implants six decades ago.
