BOSTON, MASSACHUSSETTS — Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a Phase 1/2 clinical trial in pediatric patients of 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 preclinical 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 Pediatric 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. We look forward to initiating a Phase 1/2 clinical trial of DB-OTO in pediatric patients in the first half of 2023, pending regulatory clearance.”
–Laurence Reid, Ph.D., Chief Executive Officer at Decibel
About Decibel Therapeutics
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel’s pipeline, including its lead gene therapy product candidate, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of creating a world of connection for people with hearing and balance disorders.
Source: Decibel Therapeutics