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, has announced the submission of Clinical Trial Applications (CTAs) to the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom and the Spanish Agency of Medicines and Medical Devices (AEMPS) for a Phase 1/2 clinical trial in pediatric patients of DB-OTO, a gene therapy product candidate designed to provide durable hearing in individuals with profound congenital hearing loss due to an otoferlin deficiency.
“This submission is an important milestone for our DB-OTO product candidate. Over the past several years, we have partnered with leading otolaryngologists and clinical sites in Europe who form a key part of our global clinical development strategy for DB-OTO. We are excited to work with clinicians and families in the deaf and hard of hearing community to advance our efforts to discover and develop transformative potential treatments for hearing and balance disorders.”
–Jonathon Whitton, Au.D., Ph.D., SVP of Clinical Research and Development at Decibel
DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals and is an adeno-associated virus (AAV)-based, dual-vector, gene therapy product candidate.
Otoferlin is a protein expressed in cochlear inner hair cells that enables communication between the sensory hair cells of the inner ear and the auditory nerve. Newborns born with mutations in the otoferlin gene have fully developed structures within the inner ear. However, these newborns have profound hearing loss because signaling between the ear and the brain is disrupted. DB-OTO 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.
DB-OTO received Orphan Drug and Rare Pediatric Disease designations from the U.S. FDA in 2021, and on October 17, 2022 Decibel received clearance from the U.S. FDA for its Investigational New Drug (IND) application to initiate a pediatric Phase 1/2 clinical trial in the United States in children and infants.
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
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