Source – Sensorion
On July 10, Sensorion filed its first regulatory application to start human testing of OTOF-GT, a gene therapy for one of the most common forms of congenital deafness, otoferlin deficiency.
OTOF-GT is designed to correct mutations in the otoferlin gene, which codes for a protein that is essential for hearing. The gene therapy is delivered by a dual adeno-associated virus (AAV) vector and has been shown to restore hearing in a mouse model of OTOF-related deafness for at least a year after a single injection.
Sensorion has applied to the UK’s Medicines & Healthcare products Regulatory Agency (MHRA) to start a Phase I/II clinical trial of OTOF-GT in children with otoferlin deficiency aged up to 31 months. An application in the EU is expected to follow within the next few weeks.
The company is one of a small number of biotechs trying to develop gene therapies for OTOF deafness, along with the likes of Decibel Therapeutics/Regeneron and Akouos/Eli Lilly.
“There are currently no approved drugs 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, Sensorion’s chief medical officer
The French biotech is also developing another drug, called SENS-401 (arazasetron), for residual hearing loss after cochlear implantation in adults and recently reported proof-of-concept data in a Phase IIa trial.
The drug previously failed a Phase II study in sudden sensorineural hearing loss (SNHL), but is also in mid-stage testing for deafness associated with cisplatin chemotherapy.
