Summary:
Sensorion will present early Audiogene clinical trial results of its gene therapy SENS-501 for treating severe pediatric hearing loss at the 2025 ASPO meeting, highlighting promising developments in genetic treatments for DFNB9-related deafness.
Key Takeaways:
- Audiogene Trial Focus: The Phase 1/2 Audiogene trial evaluates the safety and efficacy of SENS-501 gene therapy for young children with hearing loss caused by OTOF gene mutations.
- Clinical Presentation: Dr. Natalie Loundon, principal investigator, will present the trial’s progress and rationale at ASPO on May 1, 2025.
- Early Intervention Goal: The trial targets infants and toddlers to leverage early brain plasticity, aiming to restore hearing and support normal speech development.
Sensorion, a clinical-stage biotechnology company specializing in the development of novel therapies to restore, treat, and prevent hearing loss disorders, announced that Natalie Loundon, pediatric ENT surgeon, director of the Center for Research in Pediatric Audiology at Necker Enfants Malades Hospital, AP-HP, in Paris, France, will deliver an oral presentation on the company’s Audiogene clinical trial at the annual meeting of the American Society of Pediatric Otolaryngology (ASPO).
The Conference is being held in Montreal, Canada, April 30 – May 3, 2025, and Dr Loundon will give her presentation, “Principle and Practice of a Gene Therapy for Hearing loss: A Phase 1/2 Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss,” on May 1st at 4.20 pm CET (10.20 am ET).
Loundon is the principal investigator of Audiogene, Sensorion’s Phase 1/2 clinical trial evaluating SENS-501, a gene therapy treatment for DFNB9, a genetic disorder causing severe to profound hearing loss due to mutations in the OTOF gene. Her presentation will include an overview of the rationale behind gene therapy approaches for inner ear hearing loss disorders and the company’s Audiogene clinical trial.
About the Audiogene Trial
Audiogene aims to evaluate the safety, tolerability, and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in infants and toddlers aged 6 months to 31 months at the time of gene therapy treatment. By targeting the first years of life, when brain plasticity is optimal, the chances of these young children with pre-linguistic hearing loss acquiring normal speech and language are maximized. The study comprises two cohorts of two doses followed by an expansion cohort at the selected dose. While safety will be the primary endpoint of the first part of the dose escalation study, auditory brainstem response (ABR) will be the primary efficacy endpoint of the second part of the expansion. Audiogene will also evaluate the clinical safety, performance, and ease of use of the delivery system developed by Sensorion.
About Sensorion
Sensorion is a clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur.
SENS-501
SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses.
SENS-401
Sensorion’s portfolio also comprises programs of a clinical-stage small molecule, SENS-401 (Arazasetron), for the treatment and prevention of hearing loss disorders. Sensorion’s small molecule progresses in a Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) for the preservation of residual hearing. Sensorion, with partner Cochlear Limited, completed in 2024 a Phase 2a study of SENS-401 for residual hearing preservation in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022.
Featured image: Dreamstime
Incredible progress. I remember meeting a family a few years ago whose baby was diagnosed with genetic hearing loss — they were told early intervention could change everything, but options were limited. Seeing trials like Audiogene and SENS-501 now gives real hope that future families won’t have to face the same uncertainty. Early treatment during brain plasticity could truly change lives.