Atonal Therapy is a product concept to restore hearing or balance function through the regeneration of critical cells of the inner ear. Hearing and Balance require specialized cells of the inner ear called sensory hair cells. During embryonic development a gene termed atonal (ATOH) induces the generation of these cells. GenVec has shown preclinically that the production of the ATOH protein results in the formation of new inner ear sensory hair cells, and the restoration of hearing and balance function. TherAtoH is an adenovector product delivering the human atonal gene (Hath1) to trigger the production of therapeutic proteins by cells in the inner ear.

Addresses Unmet Medical Needs – There are no drug therapies available to treat hearing loss or balance disorders. This investigational drug is being developed to improve the treatment of inner ear disorders including hearing loss and balance function disorders. According to the National Institute on Deafness and Other Communication Disorders, approximately 28 million Americans have a hearing impairment. Even with the most conservative estimates, this represents an opportunity of at least $6 billion. TherAtoh therapy could be explored for patient segments including those with hearing loss caused by exposure to excessive noise or other trauma, aging, and diseases and infections or their treatment (also in pediatric patients). These segments total approximately 24 million patients.In the United States alone, it is estimated that 5-10% of the population suffers from balance loss and there is little available treatment.The cost of medical care for patients with balance disorders has been estimated to exceed $1 billion per year.

Scientific Rationale and Status Overview – The program is based on the work of Dr. Huda Zoghbi (Baylor College of Medicine) who reported in an issue of Science that mouse inner ear hair cell development was dependent upon the ATOH gene. GenVec has since preclinically demonstrated that atonal genes can function in the ears of adult mice and guinea pigs. We have achieved this with our collaborators in two independent preclinical models. The Raphael lab used a guinea pig model to demonstrate improvement of hearing function, and the the Staecker lab (University of Kansas) has shown that atonal gene delivery into the semicircular canal of the inner ear (mouse model) provides a clear improvement in balance function. Aspects of this work were published in Nature Medicine in 2005 and Otology & Neurotology in 2007.