Sensorion, a clinical-stage biotechnology company focused on hearing loss therapies, has announced promising safety data from its Phase 1/2 clinical trial, Audiogene, for SENS-501. This gene therapy targets congenital deafness caused by mutations in the OTOF (otoferlin) gene, which is responsible for critical auditory signal transmission in the inner ear. The study, involving infants and toddlers aged 6 to 31 months, aims to evaluate the safety and efficacy of the therapy, particularly focusing on the first years of life, when brain plasticity is at its peak, to maximize the chances of speech and language development.
The first two patients in the Audiogene trial received intracochlear injections of SENS-501. The procedure was well-tolerated with no serious adverse events reported, and both children showed early signs of behavioral improvements. This marks a significant step in the development of gene therapy for hearing disorders, as the early intervention aims to restore hearing and facilitate speech acquisition, potentially transforming the lives of children with genetic hearing loss.
Dr. Géraldine Honnet, Sensorion’s Chief Medical Officer, expressed satisfaction with the progress, highlighting the safety and early positive outcomes observed in the first cohort. She emphasized the potential of SENS-501 to revolutionize hearing disorder treatments. The company plans to treat the third patient in the first cohort soon and will present further data from the trial in early 2025, including an in-depth webinar with key opinion leaders.
Professor Natalie Loundon, coordinating investigator for Audiogene, also praised the results, noting the hope gene therapy offers for improving hearing, speech acquisition, and quality of life for children with otoferlin gene mutations. The trial continues with recruitment expected to conclude for the first cohort by year-end and the second cohort by mid-2025.
SENS-501 is designed to restore hearing by introducing a functional copy of the OTOF gene into the inner ear using AAV (adeno-associated virus) technology. This therapy aims to address congenital deafness by correcting the gene defect that prevents the proper conversion of sound into electrical signals. Sensorion is developing this therapy in collaboration with the Pasteur Institute, Necker Enfants Malades Hospital, and the Hearing Foundation, with partial funding from the French National Research Agency.
Audiogene’s goal is not only to restore hearing but also to foster language development, which is a critical milestone in early childhood. With ongoing trials and the potential for groundbreaking results, SENS-501 holds promise for families affected by genetic hearing loss, offering a new avenue for treatment where none previously existed.
In addition to its work on gene therapy, Sensorion is advancing other clinical programs for hearing disorders, including small molecule therapies and its second gene therapy program for GJB2-related hearing loss. The company is committed to improving the diagnosis and treatment of hearing loss, a significant unmet medical need globally.
