(Boursier.com) — Sensorion a pioneering clinical-stage biotechnology company dedicated to developing innovative therapies to restore, treat and prevent hearing loss, today announces that it has submitted an initial Clinical Trial Authorization Application (CTA) for OTOF-GT with the Medicines Healthcare products Regulatory (MHRA) UK Medicines Regulatory Agency (MHRA).
Phase 1/2 clinical trial (Audiogene) aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of OTOF-GT for the treatment of otoferlin gene-related hearing loss in patients up to 31 months of age. The CTA’s submission follows extensive preclinical studies that assessed the safety and efficacy of OTOF-GT and the successful production of a batch of the gene therapy pharmaceutical product for the trial. clinical.
OTOF-GT, Sensorion’s dual-vector Adeno-Associated Gene Therapy (AAV) program is being developed to restore hearing to patients with otoferlin deficiency-related mutations with hearing loss severe to profound non-syndromic prelingual sensorineural neuroscience. Otoferlin is a protein that is expressed in the inner hair cells (IHC) found in the cochlea and is essential for signal transmission to the auditory nerve. Hearing loss related to otoferlin deficiency is responsible for nearly 8% of all cases of congenital hearing loss and approximately 20,000 people are affected in the United States and Europe.
OTOF-GT previously received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA)2 and European Medicines Agency (EMA)3 as well as Rare Pediatric Disease Designation (RPDD) from the FDA in the 4th quarter of 2022.
Nawal Ouzren, Chief Executive Officer of Sensorion, commented: “This first submission marks a major milestone for our OTOF-GT program as well as for Sensorion’s entire gene therapy franchise. trials in other countries, this being the first, and we hope to be able to start recruiting patients as soon as possible, subject to CTA approval. We look forward to continuing our collaboration with clinicians, regulators and patient groups to address unmet medical needs in the highly complex area of hearing.”
Géraldine Honnet, Medical Director of Sensorion, added: “There are currently no approved drugs for patients with mutations in the gene coding for otoferlin. Our ambition is to change the care of newborns suffering from otoferlin deficiency, reducing dependence on cochlear implants, which would significantly improve their quality of life.We believe that gene therapy has the potential to offer long-term therapeutic solutions to these patients and to those suffering from ‘other inner ear diseases. We are very excited to advance OTOF-GT to the clinical stage.”
Sensorion expects to submit an application for clinical trial authorization in Europe in the coming weeks.
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