NEW YORK – Sensorion on Friday said it has received permission to begin a Phase I/II trial of its lead gene therapy candidate in some European countries, beginning in France.
In the Audiogene trial, Montpellier, France-based Sensorion will assess the safety, tolerability, and efficacy of SENS-501 (OTOF-GT), a gene therapy that aims to restore hearing in patients with deafness caused by mutations in the OTOF gene, specifically testing it in children who are 6 to 31 months old.
Patients participating in the trial will receive an intra-cochlear injection of SENS-501, which delivers two parts of the OTOF gene that codes for otoferlin using a dual adeno-associated virus vector. Otoferlin is a protein expressed in the inner hair cells in the cochlea that is critical for transmitting signals to the auditory nerve.
The first part of the trial will involve two cohorts of patients that will receive different doses, and during this phase, investigators will primarily assess safety. Then in an expansion cohort that will receive a single selected dose, investigators will monitor auditory brainstem response. Investigators will also evaluate the safety, performance, and usability of the device used for the intra-cochlear injection, which is being developed through a partnership with medical device maker Eveon.
Sensorion is developing SENS-501 as part of its R&D partnership with the nonprofit French biomedical research foundation Institut Pasteur, which the organizations kicked off in 2019 and recently extended through 2028.