Akouos Raises $50 Million Series A to Advance Gene Therapy for Hearing Loss

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Akouos, based in Boston, announced a Series A financing worth $50 million. The round was led by seed investors 5AM Ventures and New Enterprise Associates (NEA). They were joined by existing seed investor Partners Innovation Fund and new investors Sofinnova Ventures, RA Capital Management and Novartis Venture Fund.

Akouos is focused on using gene therapies to restore and preserve hearing. It is developing targeted adeno-associated viral vector (AAV)-based gene therapies for sensorineural hearing loss caused by problems or damage to sensory cells or the nerve fibers of the inner ear. This type of hearing loss is the cause of most cases of newborn deafness and affects about 25 percent of all adults over the age of 65. The initial focus is on monogenic types of sensorineural hearing loss where specific gene mutations lead to profound deafness.

The company expects to use the funds to advance its lead program to first-in-human clinical trials. It also will use it to bolster its various pipeline programs.

“Hearing loss is one of the greatest challenges in medicine today, and with no FDA-approved therapies available, an area of severe unmet need,” said Manny Simons, founder and chief executive officer of Akouos, in a statement. “Akouos has a unique opportunity to bring forward the world’s first precision medicines for individuals with genetically-driven forms of hearing loss. The expansion of our team and investor base, in addition to our founding partnerships with Massachusetts Eye and Ear and Lonza, puts us in a strong position to advance the clinical studies that will convert the promise of our approach into meaningful therapies.”

In addition to the financing, Arthur Tzianabos, president and chief executive officer of Homology Medicines and Christopher Smith, former chief executive officer of Cochlear Ltd., will join the company’s board of directors.

It is also expanding its executive team. It has appointed Michael McKenna as chief medical officer. McKenna is a scientific co-founder of Akouos and is the Joseph B. Nadol, Jr. Chair and most recent director of the Division of Otology and Neurotology at Massachusetts Eye and Ear, and professor of otolaryngology at Harvard Medical School. Jennifer Wellman will join the company as senior vice president, regulatory. Wellman is the co-founder of Spark Therapeutics, where she led regulatory strategy for Luxturna, the first in vivo gene therapy approved in the U.S.

Simons told Business Insider, “For many forms of hearing loss, the cause is complex. We’re still understanding it. We started with a focus on the forms of hearing loss that are best understood because those are the ones we feel we have the best opportunity to have a dramatic impact.”

In the company’s approach, the modified genetic material is delivered by the viral vector. They are placed into the inner ear by way of a minimally invasive surgical procedure. At this point, the procedure is entirely preclinical, and the company expects to submit for clinical trials with the U.S. Food and Drug Administration (FDA) in 2019.

“The five-year plan is to have multiple gene therapies in clinical development with a lead demonstrating clinical efficacy by that time,” Simons told Business Insider.

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