Sensorion, a clinical-stage biotechnology company specializing in hearing loss disorders, has announced the submission of its first Clinical Trial Application (CTA) to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).
The CTA is for the phase 1/2 clinical trial of OTOF-GT, a gene therapy aimed at treating otoferlin gene-mediated hearing loss in pediatric patients up to 31 months old. The trial will assess the safety, tolerability, and efficacy of intra-cochlear injection of OTOF-GT, following successful preclinical studies and manufacturing of the gene therapy Drug Product.
OTOF-GT Gene Therapy
OTOF-GT is part of Sensorion’s dual AAV vector gene therapy development program, with the goal of restoring hearing in patients with severe to profound sensorineural prelingual non-syndromic hearing loss caused by OTOF gene mutations.
Otoferlin, a protein crucial for auditory signal transmission in the cochlea’s inner hair cells, is affected in otoferlin-related hearing loss, which accounts for up to 8% of congenital hearing loss cases, impacting around 20,000 individuals in the US and Europe. OTOF-GT has previously received Orphan Drug Designation from the US FDA and the EMA, as well as Rare Pediatric Disease Designation from the FDA.
“There are currently no approved drug for patients with mutations of the gene encoding for otoferlin. 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.”
—Géraldine Honnet, Chief Medical Officer at Sensorion
Nawal Ouzren, CEO of Sensorion, expressed the significance of the CTA filing as a milestone for the OTOF-GT program and the company’s gene therapy franchise. She highlighted the aim to address unmet medical needs in the hearing space and the importance of collaboration with clinicians, regulatory authorities, and patient groups.
Sensorion plans to submit the CTA in Europe in the coming weeks.
Source: Sensorion
