Sensorion announced that it has submitted a Clinical Trial Application (CTA) to initiate a Phase 1/2 clinical trial of OTOF-GT in Europe (France, Italy and Germany). On July 10th, 2023, Sensorion submitted a first CTA for OTOF-GT to the UK's Medicines and Healthcare products Regulatory Agency (MHRA). The phase 1/2 clinical trial (Audiogene) aims to evaluate the safety, tolerability and efficacy of intra-cochlear administration of OTOF-GT, for the treatment of otoferlin gene-mediated hearing loss in pediatric patients aged from 6 to 31 months.

The CTAs submission follows extensive preclinical studies assessing the safety and efficacy of OTOF-GT and successful manufacturing of the gene therapy Drug Product for the clinical trial. Sensorion's OTOF-GT dual AAV vector gene therapy development program aims to restore hearing in patients with mutations in OTOF who suffer from severe to profound sensorineural prelingual non syndromic hearing loss. Otoferlin is a protein expressed in the inner hair cells (IHC) present in the cochlea and is critical for the transmission of the signal to the auditory nerve.

Otoferlin related hearing loss is responsible for up to 8% of all cases of congenital hearing loss, with around 20,000 people affected in the US and Europe. OTOF-GT previously received Orphan Drug Designation from the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and Rare Pediatric Disease Designation from the FDA in Fourth Quarter 2022.