Sensorion Reports Positive Initial Results from Audiogene Phase 1/2 Gene Therapy Trial

MONTPELLIER, France | July 01, 2025

Sensorion, a leading clinical-stage biotechnology firm, has released promising preliminary data from the first cohort of the Phase 1/2 Audiogene clinical trial. This trial evaluates SENS-501, a gene therapy aimed at addressing congenital deafness caused by mutations in the OTOF gene. The trial's initial cohort used a lower dose of the therapeutic candidate and has shown positive outcomes, while a second cohort at a higher dose is underway and nearing completion in terms of recruitment.

The trial's early results indicate that the administration of SENS-501 is well-tolerated among all participating infants and toddlers. These children, who are between 6 to 31 months old and have not undergone cochlear implantation, experienced no serious adverse effects following the intracochlear administration of the therapy. Specifically, three children were administered a low dose of 1.5E11 vg/vector/ear, designed to be the minimal effective level as indicated by preclinical studies. The primary aim at this stage was to evaluate the safety and practicality of this mode of delivery.

Within Cohort 1, early improvements in hearing were noted, particularly in an 11-month-old patient, referred to as Patient 3. Investigators assessed these improvements using standard hearing tests such as Auditory Brainstem Response (ABR), Pure Tone Audiometry (PTA), and parent-reported outcomes. Three-month data from Patient 3 display encouraging signs of auditory progress.

As the trial progresses, Sensorion is close to completing recruitment for Cohort 2, which involves administering a higher dose of the gene therapy. The company plans to provide further updates once data from Cohort 2 reaches sufficient maturity, which will help in determining future steps for this groundbreaking program.

Professor Catherine Birman, an ENT specialist from the Children’s Hospital at Westmead in Australia, expressed optimism about the early findings from Cohort 1. She noted the favorable safety profile of SENS-501 and the promising early auditory responses in Patient 3. Though the current dose was primarily intended to evaluate safety, its effectiveness in initiating auditory improvements is a positive sign. Professor Birman looks forward to continuing the study with a higher dose in the second cohort. She emphasized the importance of treating young children who have not received cochlear implants, as restoring hearing within the first three years could significantly enhance language acquisition.

The Audiogene trial, developed through a strategic partnership with the Institut Pasteur and led by Professor Natalie Loundon from Necker Enfants Malades in Paris, aims to test the safety and potential benefits of SENS-501 for children suffering from hearing loss due to OTOF gene mutations. The trial involves two cohorts for dose escalation, with Cohort 1 focusing on a lower dose and Cohort 2 on a higher dose. A dose-expansion phase will follow, targeting optimal therapeutic dosage.

SENS-501 (OTOF-GT) represents an innovative gene therapy endeavor, aiming to restore auditory function by introducing a healthy copy of the OTOF gene into inner ear hair cells through viral vectors. This approach seeks to normalize sound signal conversion into electrical signals, potentially enabling patients to regain hearing. As part of the RHU AUDINNOVE consortium, the therapy is developed by Sensorion in collaboration with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l’Audition, partly funded by the French National Research Agency.

Sensorion continues to lead in the biotechnology sector, focusing on developing therapies to combat hearing loss, a significant unmet medical need globally. The company’s research and development platform is instrumental in identifying optimal targets and methods for innovative drug candidates, including gene therapies for hereditary deafness. Sensorion's efforts extend to identifying biomarkers for better diagnosis and developing small molecules like SENS-401, which is in clinical trials for various hearing loss conditions.