Akouos raises $50 million to develop gene therapies for hearing loss

The roster of well-funded start-ups attempting to treat hearing loss has expanded with the launch of Akouos. The Boston-based firm has raised $50 million in its first major funding round to develop gene therapies to correct genetic forms of hearing loss.

Akouos, like other companies in its field, has a long-term vision looking to breach the vast, untapped market for age-related hearing loss: Some 360 million people have serious hearing loss worldwide, and over 300,000 people have genetic-related hearing loss in the U.S. No drugs are approved to treat these conditions. “The number of people affected and the complete lack of therapies available for patients with hearing loss is unparalleled,” says Manny Simons, founder and CEO of Akouos.

The field has attracted a number of companies in recent years, including Decibel Therapeutics, which in June raised $55 million in its series C funding round to develop therapies to treat hearing loss and tinnitus. Last year, Frequency Therapeutics launched to develop small molecule regenerative medicines for hearing loss based on technology from the labs of MIT’s Robert Langer and Harvard Medical School’s Jeffrey Karp. And Novartis has resumed a previously halted trial testing the potential of an adenovirus gene therapy to restore hearing.

Simons grew up playing classical piano and developed an interest in hearing loss while studying both music and neuroscience at Harvard University as an undergraduate. He then earned his Ph.D. in Langer’s lab at MIT before heading to Harvard Business School for his MBA. Most recently, he led business development at Voyager Therapeutics, a biotech company developing gene therapies for Parkinson’s disease and other neurological conditions.

All that experience culminated in the creation of Akouos in August 2016. Simons says the new funding will get the company’s most advanced program into clinical testing, but he isn’t disclosing the specific diseases it is targeting yet. The start-up did previously announce it will use synthetic adeno-associated viruses (AAVs) that were computationally created in the lab of Luk H. Vandenberghe at Massachusetts Eye & Ear.

Last year, Vandenberghe and colleagues used one of his synthetic viral particles, called Anc80, to deliver a gene therapy into the sensory hair cells of mice with genetic mutations modeled after those found in humans with Usher syndrome, a severe condition of blindness, deafness, and loss of balance. The gene therapy, which encoded a scaffolding protein that is broken in Usher syndrome, dramatically restored hearing and balance in the mutant mice to near normal levels (Nat. Biotechnol. 2017, DOI: 10.1038/nbt.3801).

Akouos has licensed the rights to use Anc80 for hearing and balance disorders from Massachusetts Eye & Ear and the Swiss drug manufacturer Lonza, which previously struck a licensing agreement to manufacture Vandenberghe’s synthetic AAVs.