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Sensorion, a biotech company based in France, has filed applications to begin clinical trials for a gene therapy called SENS-601, which is designed to treat a form of hearing loss that people are born with. Regulators in Canada and France have received the filings, and submissions to the FDA and Australian regulators are planned by the end of 2026.
The therapy targets mutations in the GJB2 gene, the most common genetic cause of congenital deafness. GJB2 mutations are thought to account for about half of all cases of hereditary non-syndromic hearing loss. The gene normally produces a protein that helps the inner ear convert sound vibrations into signals the brain can interpret. When the gene is mutated, that process breaks down, typically resulting in severe to profound hearing loss from birth.
SENS-601 works by delivering a functional copy of the GJB2 gene directly into the inner ear using a modified virus as a carrier. If successful, the goal is for the inner ear to begin producing the missing protein. The planned clinical study, called Hearconnex, will assess safety and effectiveness in children with GJB2-related hearing loss. No human trial results exist yet for SENS-601.
In the same announcement, Sensorion said it is discontinuing a separate gene therapy program that targeted a different genetic cause of hearing loss, the OTOF gene, in order to concentrate resources on SENS-601 and its much larger patient population.
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