Decibel Therapeutics, a clinical-stage biotechnology company developing novel restorative gene therapeutics to treat hearing loss and balance disorders, has announced exclusive license agreements with the University of Florida (UF) and the University of California, San Francisco (UCSF) for an adeno-associated virus (AAV) gene therapy technology designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene.
Dr. William Hauswirth, a professor in the department of ophthalmology and the Maida and Morris Rybaczki Eminent Scholar Chair in Ophthalmic Sciences at UF, is one of the principal inventors of the technology.
Otoferlin is a protein present in the inner hair cells of the cochlea that is critical for the communication between sensory cells of the inner ear and the auditory nerve by regulating the release of neurotransmitters. People born with biallelic mutations in the otoferlin gene have profound hearing loss because this signal between the ear and the brain is lost. Decibel aims to restore functional otoferlin using gene therapy. A principal challenge is the size of the otoferlin gene, which is too large for the packaging capacity of AAV vectors. To overcome this challenge, Dr. Hauswirth, Dr. Omar Akil (UCSF), and collaborators employed a dual-vector approach to delivering the gene in two separate AAV vectors. This approach resulted in the expression of the complete otoferlin gene, restored the signaling connection between the ear and the brain, and rescued normal hearing in a deaf, otoferlin-deficient mouse model.Learn more about Therapeutics Company Announces Exclusive Licensing Agreements for Hearing Loss Gene Therapy Technology Developed by UF Researcher.