Hearing loss therapy biotech company Sensorion is seeking to kickstart the Phase I/II trial for its new gene therapy candidate after submitting a clinical trial application (CTA) to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

Paris-based Sensorion has developed an intra-cochlear injection to restore hearing in patients with mutations in the OTOF gene who have sensorineural prelingual non-syndromic hearing loss. OTOF codes for the protein otoferlin, which is expressed in the inner hair cells and is important for neuronal transmission of signals that encode sound.

The clinical trial will evaluate the safety, efficacy and tolerability of the OTOF-GT treatment in paediatric patients aged up to 31 months. The candidate is a dual adeno-associated viral (AAV) vector gene therapy. Currently, no approved drugs for patients with mutations in the OTOF gene have been reported. OTOF-GT received orphan drug designation in 2022 from the US Food and Drug Administration (FDA).

Sensorion, which also has a candidate called SENS-401 (Arazasetron) that recently met secondary endpoints in a trial for patients scheduled for cochlear implants, also intends to submit a CTA for OTOF-GT in Europe in the coming weeks.

Nawal Ouzren, Sensorion’s CEO, commented: “This first gene therapy CTA filing is a major milestone for our OTOF-GT program and Sensorion’s broader gene therapy franchise. This is the first of several planned CTA filings and we hope to commence patient recruitment soon, subject to the CTA approval.”

Géraldine Honnet, Chief Medical Officer of Sensorion added: “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. We believe that gene therapy has the potential to offer permanent solutions to patients with diseases caused by the OTOF mutation and other inner ear diseases and we are really excited to move OTOF-GT forward towards clinical development.”