Decibel Therapeutics, a biotechnology company that licensed patented innovations from the University of Missouri, has reached a new milestone in the development of a gene therapy to restore hearing in individuals born with profound congenital hearing loss due to an otoferlin (OTOF) deficiency.
The U.S. Food and Drug Administration (FDA) recently approved the company’s Investigational New Drug (IND) application, clearing the way for an early-stage clinical trial to evaluate the safety, tolerability and efficacy of a new therapy delivered using technology invented by Dongsheng Duan, Curators’ Distinguished Professor of Molecular Microbiology and Immunology; Research Specialist Lead Yongping Yue; and graduate student Arkasubhra Ghosh.
Duan specializes in designing the vehicles, or viral vector systems, that bring a therapeutic gene to cells or tissues. In this case, his innovations expand the capacity of the Adeno-Associated Virus (AAV) to deliver genes that, in the past, have been too big to fit on a single AAV vector.
“This technology enables gene therapy to be used across a wider variety of genetic diseases,” said Brian Buntaine, senior technology transfer manager who works with Duan and other faculty inventors to leverage the commercial potential of their research. “Decibel’s success in obtaining IND approval from the FDA is an exciting and important milestone in bringing this technology to market.”
Mutations of the OTOF gene are one of the most common causes of genetic, congenital hearing loss, according to Geoff Horwitz, vice president of corporate development at Decibel. OTOF is a protein expressed in the cochlear inner hair cells that enables communication between the sensory cells of the inner ear and the auditory nerve.
“Normal functioning OTOF enables inner hair cells to release neurotransmitters in response to stimulations by sound, which then activates the auditory nerve and carries the signal to the brain,” Horwitz said. “Babies born with OTOF mutations have fully developed structures within the inner ear; however, they have profound hearing loss because signaling between the ear and the brain is disrupted.”
Accelerating personalized treatments tailored for each individual, rather than a traditional one-size-fits-all approach, is the focus of the university’s NextGen Precision Health initiative. The goal is to provide crucial resources to faculty like Duan that will help streamline the journey from laboratory research to effective clinical treatments.
“Many diseases are caused by gene mutations,” Duan said. “If you can fix a particular mutation for each patient, then that’s going to be a major step in helping them get better.”
MU’s Technology Advancement team partners with companies like Decibel to bring faculty innovations to commercial settings for development into products and services that benefit society.
“Despite having an incredible team of scientists and industry veterans here at Decibel, we recognize how valuable the innovative, groundbreaking research being conducted in top academic research institutions is,” Horwitz said. “We share a passion for helping people and know that we have a better chance to accomplish this together than we do independently.”