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      How Akouos is working to develop gene therapies for hearing loss

      Ακούω is a Greek word that means to listen. Akouos (pronounced ah KOO ohs) is a biotechnology company based in Boston, USA. It was formed in 2016 with the goal of applying precision genetic medicine to conditions that cause hearing loss. A key step in the development of such potential treatments underlies the choice of the company’s name.

      By: Jim McLaughlin, Akouos Therapeutics | 14 November 2019
      The team at Akouos believes the first step in any successful venture involves listening. For them, this means listening to people who are deaf or have hearing loss, to hearing researchers and healthcare providers, and to each other. The partnerships and collaborations Akouos has formed are based upon this approach. They are focused on the shared goals of developing and delivering effective and durable treatment options for hearing disorders.

      The Sing Registry


      Their philosophy is exemplified by the upcoming launch of The Sing Registry, a long-term project led by Akouos. The aim of the registry is to collect information on patient histories and treatment regimes, as well as relevant genetic testing information from people affected by genetic sensorineural hearing loss. The registry will enroll people who have been diagnosed through genetic testing, as well as those who first developed symptoms of suspected genetic hearing loss before the age of 55.

      One goal of a registry is to provide patients with a single place where their medical and family history can be stored and updated. It can also provide a source of information for clinicians and researchers who are studying genetic hearing loss. Registries like this also offer a unique way for doctors and researchers to understand the real-world impact of conditions such as hearing loss. They provide a view of patients’ experiences beyond the clinic that can help to speed up the development of future treatments. In addition, the registry is a way for participants to keep updated on the latest information about clinical trials, based upon their specific diagnosis and medical history. The Sing Registry is expected to be up and running by early 2020. For more information, including how to participate, please visit www.SingRegistry.com.

      Gene therapies for genetic hearing loss


      Akouos has also recently announced their lead therapeutic development program, called AK-OTOF. AK-OTOF is an investigational gene therapy for people with sensorineural hearing loss caused by mutations in the otoferlin gene (or OTOF). Otoferlin acts as a linker between the sound-sensing cells of the inner ear (hair cells) and the auditory nerve cells that send sound information to the brain. People with OTOF mutations typically have severe-to-profound hearing loss at birth, and as a result, their brain is unable to process sounds correctly. Mutations in the OTOF gene are one of the most common causes of genetic hearing loss, affecting an estimated 200,000 people worldwide. The AK-OTOF investigational therapy is designed to supply affected individuals with a replacement otoferlin gene in order to re-establish the link between hair cells and the sound-processing regions of the brain. The goal of the program is to begin the first clinical trial in people by 2021. If you are interested in this program, you can find more information here.

      Gene therapies represent a powerful new approach to potentially treat hearing disorders that traditional approaches have been unable to treat effectively. For many of these conditions, particularly those with an underlying genetic basis, gene therapies could ultimately provide a potential treatment that can restore lost hearing ability. The AK-OTOF program is the first of many envisioned Akouos programs that will use precision genetic techniques to research, refine, and potentially deliver new therapies. Using an approach that starts with listening to all stakeholders, the scientists and clinicians at Akouos will apply the techniques of genetic therapy to develop potential treatments for a diverse range of hearing disorders.

      Find out more


      For more information about Akouos, visit their website.

      You can find out more about the research we’re funding in our biomedical research section.

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