NEW YORK – Sensorion on Monday said that by year-end it expects to complete dosing the first cohort of pediatric patients with congenital hearing loss with its gene therapy candidate SENS-501 in the Phase I/II Audiogene trial.
So far, one patient in Australia with hearing loss caused by mutations in the OTOF gene has received SENS-501 in the third quarter and tolerated it well, experiencing no serious adverse events. The firm has screened two other patients in the trial.
Montpellier, France-based Sensorion is enrolling three patients between 6 and 31 months of age in each of the two cohorts in the Audiogene trial. Patients will receive one of two doses of an intracochlear injection of SENS-501, which delivers two parts of the OTOF gene using a dual adeno-associated virus vector. Sensorion is hoping SENS-501 will restore hearing for these pediatric patients early in life, giving them the best chance of being able to acquire normal speech and language skills.
Sensorion plans to complete recruiting the second cohort of patients by the end of the first half of 2025. After completing this dose-escalation portion of the trial, the company will test the gene therapy in an expansion cohort at the selected dose.
Multiple biotechs are developing gene therapies for OTOF-linked hearing loss, including Eli Lilly and Regeneron. This summer, researchers at the Eye & ENT Hospital of Fudan University in Shanghai and Mass General Brigham's Massachusetts Eye and Ear in Boston said five children exhibited signs of restored hearing after receiving a gene therapy they developed.