Sensorion, a leading clinical-stage biotechnology company, has achieved a major milestone in the field of audiology and genetic research. The company has received approval for its Clinical Trial Application (CTA) in France to commence a Phase 1/2 trial of its groundbreaking gene therapy, SENS-501. This clinical trial, named Audiogene, is poised to explore the potential of SENS-501 in treating OTOF gene-mediated hearing impairment in young children.

Clinical Trial Aims and Structure:

– Target Patient Group: The trial focuses on pediatric patients aged 6 to 31 months suffering from pre-lingual hearing loss due to OTOF gene mutations.

– Treatment Approach: SENS-501 involves an intra-cochlear injection intended to restore normal hearing and speech development in affected children.

– Study Design: The trial comprises two initial cohorts with varying doses, followed by an expansion cohort at the selected dose. Safety is the primary focus in the initial stages, while the auditory brainstem response (ABR) will be the key efficacy metric in later phases.

This clinical endeavor has been greenlit following rigorous preclinical studies that demonstrated the safety and efficacy of SENS-501. Significantly, the development of the gene therapy product is a result of Sensorion’s collaboration with the renowned Institut Pasteur.

Expert Opinions and Contributions:

– Nawal Ouzren, CEO of Sensorion: Emphasized the trial’s importance in addressing a crucial unmet medical need for children with congenital deafness.

– Professor Natalie Loundon, M.D.: Highlighted the clinical trial’s potential to offer a curative treatment for children with otoferlin gene-related deafness.

– Professor Christine Petit, MD, PhD: Commended the regulatory approval as a crucial milestone in gene therapy for hearing disorders.

About SENS-501 and Its Impact:

SENS-501 aims to correct hearing loss caused by otoferlin protein deficiency, which accounts for a significant portion of congenital hearing loss cases. The therapy has garnered attention with Orphan Drug Designation from the US FDA and the European Medicines Agency, reflecting its potential to address a rare but impactful health issue.

Collaboration and Support:

Sensorion’s efforts are bolstered by partnerships with EVEON for device development and support from the French non-profit Fondation Pour l’Audition. The Audiogene trial is part of the RHU AUDINNOVE project, showcasing a collaborative effort involving Sensorion, Necker Enfants Malades Hospital, Institut Pasteur, and the Fondation pour l’Audition.

Future Outlook:

The success of this trial could revolutionize the treatment of genetic hearing loss, not only benefiting the direct recipients but also paving the way for further therapeutic innovations in audiology.

In summary, Sensorion’s initiation of the Audiogene clinical trial marks a significant advancement in the field of gene therapy for hearing disorders. It represents a beacon of hope for thousands of children suffering from genetic deafness, potentially transforming their lives and the landscape of pediatric healthcare.

  Christine Petit, France, Hearing Loss, Institut Pasteur, Natalie Loundon, Nawal Ouzren, Otoferlin Gene, SENS-501, Sensorion