The Silicon Review
Every year, thousands of children are born deaf or hard-of-hearing in the United States and Europe. Approximately 80% of congenital hearing loss in the United States and Europe is sensorineural hearing loss due to genetic causes. To date, over 150 genes are known to be directly linked to deafness. Gene therapy or the treatment of human disease using genetic material, for inner ear dysfunction is coming of age. Recent progress in developing gene therapy treatments for genetic hearing loss has demonstrated tantalizing proof-of-principle in animal models. While successful translation of this progress into treatments for humans waits, there is growing interest from patients, scientists, clinicians, and industry. By comparing genetic data between deaf and hearing family members, scientists have been able to learn the identity and location of these deafness-related genes
Akouos is a precision genetic medicine company focused on developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. Akouos is bridging the scientific community’s growing understanding of hearing loss genetics with recent advances in gene therapy. First, the firm understands which genes are affected and how they impair hearing. Healthy copies of these genes can be encased in engineered viral vectors to enter relevant cells and enable them to produce functional proteins. They are currently conducting preclinical studies to support initial clinical trials that will assess the safety and efficacy of a gene therapy for individuals with sensorineural hearing loss due to mutations in the otoferlin (OTOF) gene.
The AK-OTOF Technology
AK-OTOF is a gene therapy intended for the treatment of, and potential restoration of hearing, in individuals with sensorineural hearing loss due to mutations in the otoferlin (OTOF) gene. Normal otoferlin function enables the sensory cells of the ear (hair cells) to release neurotransmitter in response to stimulation by sound to activate auditory neurons.
Without functional otoferlin protein, auditory signals received by the ear cannot be transmitted to the brain. AK-OTOF uses an adeno-associated viral (AAV) vector to deliver a healthy copy of the OTOF gene to cochlear hair cells, with the goal of restoring long-term physiologic hearing following a single administration to the inner ear. AK-OTOF is intended to treat individuals with sensorineural hearing loss due to mutations in the OTOF gene, who typically have severe to profound bilateral hearing loss from birth, by promoting the expression of normal, functional otoferlin protein in affected cells of the cochlea. Mutations in the OTOF gene are reported to be a major cause of genetic hearing loss, affecting an estimated 200,000 individuals worldwide.
What Akouos does
At Akouos, it combines a novel delivery approach with the latest advances in gene therapy and hearing research with the goal of restoring the natural function of the inner ear. Akouos is advancing multiple candidate therapies that target sensory cells (such as inner hair cells) and nonsensory cells (such as supporting cells), in an effort to provide new potential therapeutic options for as many people as possible. Its lead program, AK-OTOF, is an AAV gene therapy intended for the treatment of, and potential restoration of hearing in individuals with sensorineural hearing loss due to mutations in the otoferlin (OTOF) gene.
Hearing Loss Registry
Together with leading clinicians, researchers, and their medical institutions, Akouos is proud to sponsor the Sing (sensorineural genetic) hearing loss registry. The aim of the Sing Registry is to establish the global infrastructure required to assess hearing loss genotype-phenotype relationships and intervention outcomes over time.
The visionary behind the success of Akouos
Emmanuel J. Simons, Ph.D., M.B.A., is Co-Founder, President, and serves as the Chief Executive Officer of Akouos. Dr. Simons is a scientist and entrepreneur with over fifteen years of experience in hearing science research and new venture formation. Prior to founding Akouos, he held leadership roles in business and corporate development at Voyager Therapeutics and Warp Drive Bio, where he was instrumental in leading strategic partnerships with aggregate value exceeding $1 billion. Earlier in his career, Dr. Simons was an Entrepreneurial Fellow at Flagship Ventures, where he was a member of the founding team of Seres Therapeutics. He earned an A.B. magna cum laude in Neuroscience and Music from Harvard College; a Ph.D. in Biomedical Engineering from MIT under Professor Robert Langer; and an M.B.A. from Harvard Business School.