MONTPELLIER, FRANCE — Sensorion announced that the independent Data Monitoring Committee (DMC) has identified no safety concerns and supports continued enrollment in the Audiogene Phase 1/2 clinical trial evaluating SENS-501, the company’s investigational gene therapy designed to treat a rare form of congenital deafness caused by mutations in the OTOF (otoferlin) gene. The committee’s review included favorable procedural tolerance and no reports of serious adverse events to date.
The Audiogene trial is assessing the safety, tolerability, and efficacy of intra-cochlear administration of SENS-501 in pediatric patients aged 6 to 31 months at the time of treatment. The study intervenes during an early period of heightened brain plasticity and enrolls children who have not undergone cochlear implantation, allowing researchers to evaluate the potential of gene therapy as a monotherapy.
The trial includes two dose-escalation cohorts with unilateral administration. The DMC review included data from the second cohort, which completed enrollment in July 2025 and included three patients treated at the higher dose. Across both cohorts, the surgical procedure and intra-cochlear administration were reported as well tolerated, with no serious adverse events or serious side effects observed.
“The safety findings combined with the early efficacy signals observed so far in infants and toddlers support further clinical investigation of SENS-501. Treating otoferlin-mediated congenital deafness before cochlear implantation and during heightened neural plasticity is essential to fully evaluate the potential of gene therapy as a monotherapy. We will continue to generate rigorous and comprehensive data to guide development decisions for this important patient population.”
–Nawal Ouzren, Chief Executive Officer of Sensorion
In children with age and baseline hearing characteristics comparable to those reported in recent publications, Sensorion has observed in the second cohort early directional improvements in two of the three treated patients by Month 3 on pure-tone audiometry. At Month 3, Patient 4 demonstrated a behavioural threshold of approximately 60 dB HL at the best-performing frequency, while Patient 5 demonstrated approximately 70 dB HL at the best-performing frequency. Follow-up is ongoing to assess the durability of these effects and the potential for further clinically meaningful functional gains.
Sensorion plans to review six-month efficacy data from the study and provide an update in the first quarter of 2026 once the dataset reaches sufficient maturity.
About SENS-501
SENS-501 (OTOF-GT) is an investigational gene therapy intended to treat congenital deafness linked to mutations in the OTOF gene, which plays a critical role in transmitting auditory signals from inner ear hair cells to the auditory nerve. The therapy delivers a functional copy of the OTOF gene to restore sound signal transmission using viral vector technology. The program is being developed in collaboration with multiple French medical and research institutions and is partially funded by the French National Research Agency.
About the Audiogene Trial
The Audiogene study is evaluating the safety, tolerability, and efficacy of SENS-501 in infants and toddlers with pre-linguistic OTOF-mediated hearing loss. The trial includes dose-escalation cohorts followed by an expansion phase at the selected dose. While safety is the primary endpoint in early stages, auditory brainstem response (ABR) will serve as the primary efficacy endpoint in the expansion phase.
About Sensorion
Sensorion is a clinical-stage biotechnology company focused on developing therapies to treat, restore, and prevent hearing loss disorders. Its pipeline includes gene therapy programs targeting hereditary deafness as well as small-molecule drug candidates aimed at preventing ototoxic and sudden hearing loss. The company maintains research collaborations with the Institut Pasteur and other institutions.
Source: Sensorion
