30 Best Small Companies To Watch 2018

Healthy hearing available to all: Akouos


Restoring and preserving high-acuity hearing is one of the greatest unmet challenges of medicine: 360 million people worldwide have disabling hearing loss, but there are no FDA-approved therapies to address it. Akouos is developing targeted adeno-associated viral vector (AAV)-based gene therapies for sensorineural hearing loss, which results from dysfunction or damage to sensory cells and/or nerve fibers of the inner ear. Sensorineural hearing loss is found in most cases of newborn deafness and affects nearly a quarter of all adults over the age of 65, making it the most common form of hearing loss and one of the most common of all sensory disorders.

Its gene therapy platform can be leveraged to restore and preserve hearing in individuals with many forms of sensorineural hearing loss, from a single genetic mutation to ototoxic drug exposure to the aging process. Initially, the company is focused on monogenic forms of sensorineural hearing loss, in which mutations in individual genes lead to profound deafness. Monogenic forms of deafness affect more than 300,000 people in the U.S. and millions of children throughout the world. The same genes responsible for monogenic deafness are known to contribute to broader hearing dysfunction, including hearing loss due to drug exposure, noise and advanced age.

Akouos is developing precision genetic medicines to restore and preserve hearing in genetically-defined patient populations.

Targeted Delivery

Ensuring delivery to the right cells, in the right amounts, and at the right time is central to Akouos’ ability to restore and preserve hearing.

  • Novel AAV Technology

Recombinant adeno-associated viruses (AAVs) can be harnessed as powerful vectors that are capable of safely and efficiently delivering therapeutic nucleic acid payloads to the nuclei of target cells. The company has exclusive rights to the Ancestral AAV (Anc-AAV) Technology platform developed in the laboratory of its scientific co-founder, Dr. Luk H. Vandenberghe, at Massachusetts Eye and Ear. Anc80, one of 40,000 Anc-AAV vectors, has been shown to transduce sensory cells of the inner ear more efficiently than other AAV capsids.

  • Minimally-Invasive Surgical Delivery

Target cells of the inner ear sit on a sensitive epithelial membrane, suspended between two fixed-volume fluid spaces and encased in dense bone. Its delivery team is focused on achieving consistent vector distribution along the sensory epithelium without disrupting normal hearing sensitivity. Its scientific co-founders Drs. Michael J. McKenna and William F. Sewell are leading experts in inner ear drug delivery and pharmacokinetics, and have developed novel technology to safely deliver therapies of any modality, from small molecules to AAV vectors, throughout the inner ear.

Precision Therapies

Akouos is focused on the restoration and preservation of hearing in genetically-defined patient populations with clear etiology. Its ability to match precision medicines with the right patients is rooted in its team’s understanding of hearing loss genetics and causes.

The founding team of Akouos has over 100 years of combined research experience characterizing inner ear structure-function relationships. The work of co-founder Dr. Richard J. Smith, world-renowned for his scientific and clinical expertise in hereditary hearing loss, allows using genomic signatures associated with specific hearing disorders to design precision therapies. The team is also developing novel biomarkers and diagnostic technologies to ensure its precision medicines are delivered to the right individuals at the right time.

Clinical Translation

The team’s commitment to families affected by hearing loss fuels their efforts to establish Akouos as an integrated platform capable of delivering life-changing treatments to individuals in need. To enable the translation of scientific breakthroughs into real-world impact, the company is building enduring partnerships with leading healthcare institutions. Its strategic partnership with Lonza demonstrates its commitment to clinical translation with world-class manufacturing capabilities.

Mass Eye and Ear, its founding strategic partner, is the world’s largest hearing research center and one of the highest-ranked hospitals for diagnosis and treatment of ear and hearing disorders.

Technology Platform

Akouos’ Technology Platform enables safe delivery to specific cellular targets in the hearing circuit. This allows it to restore the inner ear’s ability to produce functional proteins required for hearing, re-establish structures of the hearing circuit critical for high-fidelity signal transduction and inner ear homeostasis, and achieve local, enduring protein production to protect against drug, noise and age associated ototoxicity.

The leadership team

Emmanuel J. (Manny) Simons, Ph.D., M.B.A., Founder and CEO

Emmanuel J., is CEO, President, and Co-Founder of Akouos. He is a scientist and entrepreneur with over fifteen years of experience in hearing science research and new venture formation. Prior to founding Akouos, he held leadership roles in business and corporate development at Voyager Therapeutics (NASDAQ:VYGR) and WarpDriveBio, where he was instrumental in leading strategic partnerships with aggregate value exceeding $1 billion.

Earlier in his career, Dr. Simons was an Entrepreneurial Fellow at Flagship Ventures, where he was a member of the founding team of Seres Therapeutics (NASDAQ:MCRB).

Dr. Simons earned an AB magna cum laude in neuroscience and music from Harvard College; a PhD in biomedical engineering from MIT under Institute Professor Robert Langer; and an MBA from Harvard Business School.


Michael J. McKenna, M.D., Founder and Chief Medical Officer

Michael J. McKenna, a scientific co-founder of Akouos, is a world-renowned neurotologist with expertise in translational inner ear drug delivery research. He holds the Joseph B. Nadol, Jr. Chair and is the director of the Division of Otology and Neurotology at Massachusetts Eye and Ear, and is a professor of otolaryngology at Harvard Medical School. Dr. McKenna’s laboratory has shaped the field’s understanding of the molecular pathophysiology of otosclerosis through over three decades of pioneering research. He and his colleagues were the first to demonstrate an association between the COL1A1 gene and clinical otosclerosis. For the past 15 years, Dr. McKenna has led efforts to establish inner ear drug delivery systems to enable the development of hearing restoration therapies.

Dr. McKenna received his MD from the University of Southern California School of Medicine, completed his otolaryngology residency at Harvard Medical School, and his fellowship in neurotology and base skull surgery at the House Ear Clinic.

“We are a precision genetic medicine company focused on developing gene therapies that restore and preserve hearing.”