Advancing Precision Genetic Therapies With the Potential to Restore, Improve, and Preserve Hearing

Emmanuel J. (Manny) Simons, Ph.D., M.B.A., is CEO and Co-Founder of Akouos. Dr. Simons is a scientist and entrepreneur with over fifteen years of experience in hearing science research and new venture formation. Prior to founding Akouos, Dr. Simons held leadership roles in business and corporate development at Voyager Therapeutics and Warp Drive Bio, 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. Dr. Simons earned an A.B. magna cum laude in Neuroscience and Music from Harvard College; a Ph.D. in Biomedical Engineering from MIT under Professor Robert Langer; and an M.B.A. from Harvard Business School.

Richard 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 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 associate 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.