Startup Developing the First Gene Therapy for Hearing Loss

Akouos, a Boston startup founded in 2016, recently presented nonclinical data at the annual American Society of Gene and Cell Therapy meeting that supports its goal to develop the first gene therapy to treat hearing loss. If Akouos is successful, it can offer new hope to the estimated 300,000 Americans who suffer from gene-based hearing loss—a number that includes more than 4,000 newborns.
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Akouos is experimenting with adeno-associated viruses, using them as vectors to deliver DNA that encodes a functioning gene in certain target cells. Adeno-associated viruses are considered viable candidates because they don’t usually cause disease and can be customized to treat a variety of genetic conditions.

At the meeting, the company gave two presentations. One supports Akouos’ planned clinical development of AK-OTOF, a gene therapy intended to treat OTOF-mediated hearing loss, by sharing the promising results of administrating AK-OTOF to mice and nonhuman primates. According to Manny Simons, Akouos’ co-founder and chief executive, “the AK-OTOF nonclinical data demonstrate durable restoration of auditory function and show that the product candidate was systemically and locally well tolerated in two translationally relevant animal species.”¹

The other presentation addressed the future development of gene therapies to treat a wide range of inner ear conditions. It supports the use of microRNA target site (miR-TS) incorporations in adeno-associated viral (AAV) vectors for regulated gene expression in the inner ear. Simons said that Akouos is encouraged by these efforts “to advance preclinical development of other potential gene therapies for inner ear conditions, such as GJB2-mediated hearing loss, and to develop platform capabilities that can be applied to regenerative medicine approaches in the inner ear.”1
Simons holds a degree in neuroscience from Harvard College, a Ph.D. in biomedical engineering from the Massachusetts Institute of Technology and an MBA from Harvard Business School. His desire to help those with hearing loss stems from his passion for playing the piano.
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For more information about Akouos and its groundbreaking work, go to akouos.com.

¹ Hearing Health Matters. (2022). Akouos Presents Nonclinical Data Supporting the Planned Clinical Development of AK-OTOF at ASGCT. https://hearinghealthmatters.org/hearingnewswatch/2022/akouos-akotof-development-strategies-presentation/?fbclid=IwAR1686DyyU6kBooVU01tH07zgxkwWWw57iyqeoVPqfEkm5MgXi-4MERz87I

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