Akouos, a precision genetic medicine company developing gene therapies to restore and preserve hearing, announced its lead program, AK-OTOF, a gene therapy focused on restoring 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 neurotransmitters in response to stimulation by sound to activate auditory neurons, according to the company’s announcement. 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 hearing loss in both ears 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.
“The Akouos team has made great progress building the leading precision genetic medicine platform focused on inner ear disorders, and we are proud to announce our first gene therapy candidate from that platform, AK-OTOF,” said Manny Simons, PhD, Founder, President, and CEO of Akouos. “Together with leading scientists and clinicians around the world, we are working with urgency to advance AK-OTOF for individuals with sensorineural hearing loss due to mutations in the otoferlin gene. We have already begun interacting with the US Food and Drug Administration about our IND-enabling studies to support first-in-human clinical trials, and we will provide an update on commencement of our first clinical trial as soon as possible.”
Akouos Co-founder and Chief Medical Officer, Michael McKenna, MD, will present preclinical data on AK-OTOF, as well as other Akouos updates, during the Frank M. Rizer Memorial Lecture at the 54th Annual American Neurotology Society Fall Meeting. The presentation, “Emerging Role for Gene Therapy in the Treatment of Sensorineural Hearing Loss,” will take place on Saturday, September 14, 2019 at 2:25 pm CT in New Orleans.