2024 Early Hearing Detection & Intervention Conference

March 17-19, 2024 • Denver, CO

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3/19/2024  |   12:15 PM - 1:30 PM   |  Panel Discussion -The Importance of Genetics and Gene Therapy for Early Hearing Detection and Intervention.   |  Centennial E-F

Panel Discussion -The Importance of Genetics and Gene Therapy for Early Hearing Detection and Intervention.

Most congenital sensorineural hearing loss is attributable to known genetic causes. Advances in our understanding of the genetics of hearing loss and in gene therapy have permitted the development of the first investigational gene therapies for a form of genetic hearing loss. Multiple clinical trials are currently underway to evaluate the effects of these investigational gene therapies in children with hearing loss caused by genetic variants of the otoferlin (OTOF) gene. Panelists will make short presentations describing the general principles of gene therapy, design considerations of these investigational gene therapies, as well as a presentation of preliminary results from these clinical trials. Opportunities, challenges, and barriers to the use of gene therapy for children with permanent hearing loss will be discussed. The panel will then conclude with discussion of the potential implications of genetic testing and gene therapy in the identification and care of children with permanent hearing loss.

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Presenters/Authors

Karl White (Primary Presenter), Utah State University, karl.white@usu.edu;
Dr. White is a Professor of Psychology, the Emma Eccles Jones Endowed Chair in Early Childhood Education, and the founding Director of the National Center for Hearing Assessment and Management. He has been the PI or Co-PI for over $50 million of competitively awarded research. His work has been recognized with awards from such diverse organizations as the Deafness Research Foundation, the American Association for Speech Language and Hearing, The Swedish Society of Medicine, and the Alexander Graham Bell Association for the Deaf. He has hundreds of publications and presentations at scholarly meetings, and has been an invited speaker to more than 35 countries. He also serves on many national and international advisory groups for organizations such as the United States Department of Health and Human Services, the World Health Organization, the Institute of Medicine, and the American Academy of Pediatrics.


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Vassili Valayannopoulos (Co-Presenter), Decibel Therapeutics, vvalayannopoulos@decibeltx.com;
Vassili Valayannopoulos, MD, PhD, MBA, is the Clinical Program Lead for Auditory Sciences and Rare Disease at Regeneron and is responsible for all clinical activities of gene therapy programs for congenital hearing loss. He has over 20 years of experience in the rare disease and gene therapy space, both in academia and in the pharmaceutical industry, and is passionate about developing innovative therapies for patients with unmet medical needs. Dr. Valayannopoulos has a strong background in pediatric metabolic medicine, clinical genetics, and pediatric neurology, and is board certified in these specialties. He has conducted clinical research in collaboration with Institute IMAGINE at Necker, focusing on neurodegenerative metabolic diseases, gene therapy, and lysosomal storage disorders. He has also served as a principal investigator and coordinator in several clinical trials, both academic and industry sponsored, and has published numerous peer-reviewed journal articles. Dr. Valayannopoulos also has an MBA from MIT Sloan School of Management, which has enhanced his strategic and operational skills in clinical development and medical affairs.


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Aaron Tward (Co-Presenter), Aaron@example.com;
Aaron Tward, MD, PhD, is a board-certified, fellowship-trained surgeon who specializes in the diagnosis and treatment of disorders of the ear and skull base. He is an Associate Professor of Otolaryngology Head and Neck Surgery at the University of California, San Francisco and the Chief Scientific Officer at Akouos Inc. (a wholly owned subsidiary of Eli Lilly). Dr. Tward has particular clinical interests in cochlear implantation, management of benign and malignant tumors of the skull base including acoustic neuroma (vestibular schwannoma), surgery for chronic otitis media, stapedectomy, superior semi-circular canal dehiscence repair, and endoscopic surgery of the ear and skull base. Dr. Tward’s research interests include tumor biology, the genetics and genomics of disorders of the head and neck, the development and physiology of the organs of hearing, and clinical outcomes of surgery of the ear and skull base.


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Teresa Blankmeyer Burke (Co-Presenter), teresa@example.com;
Teresa Blankmeyer Burke, PhD, is a Professor of Philosophy at Gallaudet University. Her work primarily focuses on deaf philosophy (the intersection of philosophy and Deaf studies) and bioethics. Her primary interests lay in areas such as virtue ethics, moral justification, and the ethics of sign-language interpreting. Burke received a bachelor’s degree in biology, Ethics, and Society from Mills College in 1993. She went on to obtain master's and doctoral degrees in philosophy from the University of New Mexico in 2003 and 2011, respectively. Burke served as an instructor at Gallaudet from 2005 to 2011, before receiving an appointment as Assistant Professor of Philosophy. Besides her academic appointments, Burke has served in a variety of other roles including chairing the U.S. National Association of the Deaf Subcommittee on Bioethics and is also being a member of the American Philosophical Association's Inclusiveness Committee, the American Society for Bioethics and Humanities Task Force on Disability, and the World Federation of the Deaf Bioethics Committee


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