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Sensorion Seeking Regulatory Permission to Start Hearing Loss Gene Therapy Trials in UK

NEW YORK – Sensorion has submitted the first clinical trial application for its hearing loss gene therapy OTOF-GT to the UK Medicines and Healthcare Products Regulatory Agency, the French biotechnology company said Monday.

The company is seeking permission to conduct a Phase I/II clinical trial to assess the safety, tolerability, and efficacy of OTOF-GT in restoring hearing in pediatric patients up to 31 months old and suffering from severe, sensorineural, prelingual non-syndromic hearing loss caused by mutations in the OTOF gene.

"There are currently no approved drugs for patients with mutations of the gene encoding for otoferlin," Sensorion Chief Medical Officer Géraldine Honnet said in a statement. "Our goal is to transform the standard of care for OTOF newborns, by reducing dependence on cochlear implants, which would transform their quality of life."

OTOF-GT is a dual adeno-associated virus vector-based gene therapy, in which two parts of the OTOF gene are delivered through an intra-cochlear injection in an effort to assemble otoferlin, a protein expressed in the inner hair cells in the cochlea that is critical for transmitting signals to the auditory nerve.

Around 8 percent of congenital hearing loss is due to otoferlin deficiencies, and around 20,000 people in the US and Europe have the condition. The US Food and Drug Administration and the European Medicines Agency have granted orphan drug designation to OTOF-GT. 

Sensorion said it plans to submit a clinical trial application for the gene therapy in the EU in the coming weeks.