MONTPELLIER, France - Sensorion, a biotech company specializing in treatments for hearing loss, has received regulatory approval to commence a Phase 1/2 clinical trial in France for its gene therapy candidate SENS-501. The trial, named Audiogene, is designed to assess the safety, tolerability, and efficacy of SENS-501 in children aged 6 to 31 months who suffer from severe to profound hearing loss due to OTOF gene mutations.
The approval follows extensive preclinical studies and successful manufacturing of the gene therapy drug product. The trial aims to take advantage of the optimal auditory system plasticity in early childhood to help affected children develop normal speech and language skills. The primary endpoint for the dose escalation cohort is safety, while the primary efficacy endpoint for the dose expansion cohort is the auditory brainstem response.
Nawal Ouzren, CEO of Sensorion, expressed that the approval represents a significant milestone, positioning Sensorion as a leader in gene therapies for hearing loss. With no approved curative treatments for congenital deafness caused by OTOF gene mutations, this development addresses a critical unmet medical need.
Professor Natalie Loundon, the study's Coordinating Investigator, highlighted the potential impact of SENS-501, noting the necessity of advanced hospital infrastructure and skilled healthcare teams for the administration of single-injection gene therapies.
SENS-501 has previously been granted Orphan Drug Designation by both the FDA and EMA and received Rare Pediatric Disease Designation from the FDA in Q4 2022. The gene therapy is a result of Sensorion's collaboration with the Institut Pasteur, which began in 2019, and is part of the RHU AUDINNOVE project, backed by the French National Research Agency.
Sensorion plans to announce the first patient inclusion in the trial in the second half of 2024. This article is based on a press release statement from Sensorion.
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