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Akouos, a biotech startup that's trying to tackle a $750 billion global problem, just announced it's raised a new funding round

Aug 7, 2018, 16:30 IST

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  • Boston-based biotech startup Akouos is the first of its kind to use gene therapy to tackle genetic-based hearing disorders.
  • The company announced $50 million in Series A funding on Tuesday.
  • The funding will help Akouos advance its first program into human trials, support its pipeline for therapies, and build out both its team and its platform.

Akouos is a new biotech startup that's trying to address a $750 billion problem: hearing loss.

Manny Simons, co-founder and CEO of Akouos, spent his college and grad school years figuring out the biology of the ear and how to deliver drugs to the right parts of the ear. His interests drew him to his now co-founders at Massachusetts Eye and Ear about 15 years ago.

The Boston-based company announced on Tuesday that it raised $50 million in its Series A funding round. It was co-led by Seed investors 5AM Ventures and New Enterprise Associates (NEA), followed by Partners Innovation Fund, Sofinnova Ventures, RA Capital Management and Novartis Venture Fund.

The company is the first to use gene therapy to tackle hearing loss.

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"We identify specific forms of hearing loss that are genetically driven, well characterized, and we're delivering specific genes to particular target cells within the inner ear with a goal of restoring hearing," explained Manny Simons, CEO and co-founder of Akouos.

According to the World Health Organization, 466 million people worldwide have disabling hearing loss, and by 2050, that number is estimated to rise to over 900 million. Hearing loss could be caused by genetics, health complications, certain infectious diseases, chronic ear infections, the use of particular drugs, exposure to excessive noise, and aging.

"For many forms of hearing loss, the cause is complex. We're still understanding it," said Simons. "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."

The genetic forms of hearing loss are all a form of sensorineural hearing loss, which results from dysfunction or damage to the sensory cells themselves, or to the neurons they connect to. That type of hearing loss is not addressable by hearing aids, and there are currently no FDA approved drugs for them. Over 150 genes are implicated in these monogenic forms of deafness, and Akouos is focusing on several of these genes.

The gene therapy works by using a special type of virus as the delivery vessel for genetic material to target cells. They are directly delivered to the inner ear via a minimally invasive surgical procedure through the round window membrane of the ear. The process is currently preclinical, with its first program intended to undergo regulatory review from the FDA is next year. With added direction, the team will then work to better determine the design of the first in-human studies.

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"The five year plan is to have multiple gene therapies in clinical development with a lead demonstrating clinical efficacy by that time," said Simons.

The funding will support the advancement of the first program into human studies. It will also help advance the pipeline behind it throughout clinical development and help build out the team.

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