Our Mission

Healthy hearing available to all.

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

More

Our Focus

Precision Medicine for Hearing Disorders

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

Hearing connects us with one another and with the world around us.

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.

We are 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.

Our 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, we are 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.

Targeted Delivery

Ensuring delivery to the right cells, in the right amounts, and at the right time is central to our 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. We have exclusive rights to the Ancestral AAV (Anc-AAV) Technology platform developed in the laboratory of our 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. Our delivery team is focused on achieving consistent vector distribution along the sensory epithelium without disrupting normal hearing sensitivity. Our 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

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

Target Selection Guided by Clinical Genetics

Our founding team 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 us to use genomic signatures associated with specific hearing disorders to design precision therapies.

Next-Generation Diagnostics

We are developing novel biomarkers and diagnostic technologies to ensure our precision medicines are delivered to the right individuals at the right time.

Clinical Translation

Our commitment to families affected by hearing loss fuels our 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, we are building enduring partnerships with leading healthcare institutions.

GMP Manufacturing

Our strategic partnership with Lonza demonstrates our commitment to clinical translation with world-class manufacturing capabilities.


Clinical Research Infrastructure

Mass Eye and Ear, our 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

Our Technology Platform enables safe delivery to specific cellular targets in the hearing circuit. This allows us to:

  • Restore

    Restore the inner ear’s ability to produce functional proteins required for hearing.

  • Rejuvenate

    Re-establish structures of the hearing circuit critical for high-fidelity signal transduction and inner ear homeostasis.

  • Reinforce

    Achieve local, enduring protein production to protect against drug-, noise-, and age-associated ototoxicity.

Founding Team

Akouos was founded and is led by leaders in the fields of neurotology, inner ear drug delivery, clinical genetics, and AAV gene therapy.

  • Michael J. McKenna, M.D.

    Michael J. McKenna, M.D., 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.

  • William F. Sewell, Ph.D.

    William F. Sewell, Ph.D., is a leading expert in inner ear drug delivery and pharmacokinetics. He is professor of otolaryngology at Massachusetts Eye and Ear and Harvard Medical School, where for over four decades he has advanced the hearing research field through the identification and characterization of neurotransmitters and their receptors in the cochlea, and elucidating their functional roles in cochlear physiology. For the past 15 years, Dr. Sewell has collaborated with Dr. McKenna to quantitatively characterize inner ear pharmacokinetics and the unique translational challenges associated with local drug delivery to the human cochlea.

    Dr. Sewell earned his Ph.D. in pharmacology from Tulane University, and completed his postdoctoral training as a Fellow in Auditory Physiology in the Research of Laboratory Electronics at MIT.

  • Emmanuel J. Simons, Ph.D., M.B.A.

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

  • Richard J. Smith, M.D.

    Richard J. Smith, M.D., is recognized globally for his expertise in the human genetics of hearing loss. He is the founding director of the Iowa Institute of Human Genetics (IIHG) and the Molecular Otolaryngology and Renal Research Laboratories (MORL) at the University Iowa, where he is also professor of otolaryngology, pediatrics, medicine and molecular physiology & biophysics. In 1999, the MORL was the first laboratory in the world to offer CLIA-certified genetic testing for hearing loss, and in 2010, it was the first to develop and apply massively parallel sequencing as a clinical test for any disease or disorder.  Scientists at the MORL have collaborated in the mapping and/or cloning of about 20% of all genes implicated in non-syndromic hearing loss and remain focused on advancing our understanding of deafness and translating this knowledge to improved care for persons with hearing loss.

    Dr. Smith completed his M.D. and otolaryngology residence at Baylor College of Medicine, a fellowship in pediatric otolaryngology at the Hospital for Sick Children, and a postdoctoral fellowship at the Medical Research Council Institute for Hearing Research.

  • Luk H. Vandenberghe, Ph.D.

    Luk H. Vandenberghe, Ph.D., is distinguished for his breakthroughs in the AAV field, including the discovery and characterization of new AAV serotypes, and improved understanding of immune responses to AAV vectors. He is director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear and assistant professor of ophthalmology at Harvard Medical School. Dr. Vandenberghe has discovered, developed and characterized some of the most widely used gene therapy carriers for in vivo and ex vivo applications, including AAV, adeno- and lentiviral-based vectors. In 2015, his laboratory published the first work describing Ancestral AAV (Anc-AAV) Technology, using computational and evolutionary methods to predict novel conformations of the AAV particle. This unique synthetic biology and computational design approach has yielded Anc80, a putative ancestor of AAV1, 2, 3, 6, 7, 8, rh.10, and AAV9, which has been shown in mice and non-human primates to be a safe and potent therapeutic gene delivery vector in preclinical studies. Anc80 was further shown to target auditory hair cells with high efficiency in a manner that can lead to functional correction of hearing loss in mice.

    Dr. Vandenberghe received his training in the fields of engineering, virology, and gene therapy at the University of Leuven, Belgium, and University of Pennsylvania in Philadelphia.

Michael J. McKenna, M.D., 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.

William F. Sewell, Ph.D., is a leading expert in inner ear drug delivery and pharmacokinetics. He is professor of otolaryngology at Massachusetts Eye and Ear and Harvard Medical School, where for over four decades he has advanced the hearing research field through the identification and characterization of neurotransmitters and their receptors in the cochlea, and elucidating their functional roles in cochlear physiology. For the past 15 years, Dr. Sewell has collaborated with Dr. McKenna to quantitatively characterize inner ear pharmacokinetics and the unique translational challenges associated with local drug delivery to the human cochlea.

Dr. Sewell earned his Ph.D. in pharmacology from Tulane University, and completed his postdoctoral training as a Fellow in Auditory Physiology in the Research of Laboratory Electronics at MIT.

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

Richard J. Smith, M.D., is recognized globally for his expertise in the human genetics of hearing loss. He is the founding director of the Iowa Institute of Human Genetics (IIHG) and the Molecular Otolaryngology and Renal Research Laboratories (MORL) at the University Iowa, where he is also professor of otolaryngology, pediatrics, medicine and molecular physiology & biophysics. In 1999, the MORL was the first laboratory in the world to offer CLIA-certified genetic testing for hearing loss, and in 2010, it was the first to develop and apply massively parallel sequencing as a clinical test for any disease or disorder.  Scientists at the MORL have collaborated in the mapping and/or cloning of about 20% of all genes implicated in non-syndromic hearing loss and remain focused on advancing our understanding of deafness and translating this knowledge to improved care for persons with hearing loss.

Dr. Smith completed his M.D. and otolaryngology residence at Baylor College of Medicine, a fellowship in pediatric otolaryngology at the Hospital for Sick Children, and a postdoctoral fellowship at the Medical Research Council Institute for Hearing Research.

Luk H. Vandenberghe, Ph.D., is distinguished for his breakthroughs in the AAV field, including the discovery and characterization of new AAV serotypes, and improved understanding of immune responses to AAV vectors. He is director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear and assistant professor of ophthalmology at Harvard Medical School. Dr. Vandenberghe has discovered, developed and characterized some of the most widely used gene therapy carriers for in vivo and ex vivo applications, including AAV, adeno- and lentiviral-based vectors. In 2015, his laboratory published the first work describing Ancestral AAV (Anc-AAV) Technology, using computational and evolutionary methods to predict novel conformations of the AAV particle. This unique synthetic biology and computational design approach has yielded Anc80, a putative ancestor of AAV1, 2, 3, 6, 7, 8, rh.10, and AAV9, which has been shown in mice and non-human primates to be a safe and potent therapeutic gene delivery vector in preclinical studies. Anc80 was further shown to target auditory hair cells with high efficiency in a manner that can lead to functional correction of hearing loss in mice.

Dr. Vandenberghe received his training in the fields of engineering, virology, and gene therapy at the University of Leuven, Belgium, and University of Pennsylvania in Philadelphia.

Leadership Team

EMMANUEL J. SIMONS, PH.D., M.B.A.
FOUNDER AND CHIEF EXECUTIVE OFFICER
MICHAEL MCKENNA, M.D.
FOUNDER AND CHIEF MEDICAL OFFICER
JENNIFER WELLMAN
SENIOR VICE PRESIDENT, REGULATORY


Scientific
Advisory Board

LUK VANDENBERGHE, PH.D., CHAIR AND FOUNDER 
DIRECTOR OF THE GROUSBECK GENE THERAPY CENTER, MASSACHUSETTS EYE AND EAR; ASSISTANT PROFESSOR OF OPHTHALMOLOGY, HARVARD MEDICAL SCHOOL
JEAN BENNETT, M.D., PH.D
F.M. KIRBY PROFESSOR OF OPHTHALMOLOGY, UNIVERSITY OF PENNSYLVANIA
MICHAEL MCKENNA, M.D., FOUNDER
CHIEF MEDICAL OFFICER, AKOUOS, AND THE JOSEPH B. NADOL, JR.
CHAIR AT MASSACHUSETTS EYE AND EAR
WILLIAM SEWELL, PH.D., FOUNDER
PROFESSOR OF OTOLARYNGOLOGY, MASSACHUSETTS EYE AND EAR
AND HARVARD MEDICAL SCHOOL
RICHARD SMITH, M.D., FOUNDER
FOUNDING DIRECTOR OF THE IOWA INSTITUTE OF HUMAN GENETICS AND THE MOLECULAR OTOLARYNGOLOGY AND RENAL RESEARCH LABORATORIES;
PROFESSOR OF OTOLARYNGOLOGY, PEDIATRICS, MEDICINE AND MOLECULAR PHYSIOLOGY & BIOPHYSICS, UNIVERSITY OF IOWA
AARON TWARD, M.D., PH.D.
ASSISTANT PROFESSOR IN RESIDENCE, DEPARTMENT OF OTOLARYNGOLOGY –
HEAD AND NECK SURGERY, UNIVERSITY OF CALIFORNIA AT SAN FRANCISCO


Board
 of Directors

ARTHUR TZIANABOS, PH.D.
PRESIDENT AND CEO OF HOMOLOGY MEDICINES
CHRISTOPHER SMITH
FORMER CEO OF COCHLEAR LTD
ED MATHERS
PARTNER AT NEA
KUSH PARMAR, M.D., PH.D.
MANAGING PARTNER AT 5AM VENTURES
EMMANUEL J. SIMONS PH.D., MBA
AKOUOS FOUNDER AND CEO

    Our Partners