Decibel Therapeutics submits IND application for hearing loss gene therapy candidate

Decibel Therapeutics has announced the submission of an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for a Phase I/II clinical trial in paediatric patients with DB-OTO, a gene therapy product candidate designed to provide durable restoration of hearing in individuals with profound congenital hearing loss due to an otoferlin deficiency.

DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals and is an adeno-associated virus (AAV)-based dual-vector gene therapy designed to provide durable hearing to individuals with profound congenital hearing loss caused by mutations of the otoferlin gene. The product candidate uses a proprietary, cell-selective promoter to express the otoferlin transgene in hair cells, with the goal of enabling the ear to transmit sound to the brain and provide hearing. In pre-clinical studies, Decibel observed that local delivery of DB-OTO to the ear resulted in production of otoferlin protein and instatement of auditory brainstem responses to sound in a congenitally deaf rodent disease model. DB-OTO received Orphan Drug and Rare Paediatric Disease Designations from the FDA in 2021.

“This submission is an important milestone for the Decibel team as we continue advancing our gene therapy pipeline to address areas of unmet medical need. DB-OTO may potentially provide a new treatment option for children born with otoferlin deficiency, a leading cause of infant hearing loss, for which there are no approved pharmaceutical remedies,” said Dr Laurence Reid, Chief Executive Officer at Decibel. “We look forward to initiating a Phase I/II clinical trial of DB-OTO in paediatric patients in the first half of 2023, pending regulatory clearance.”