Chris Cunningham, PhD, Assistant Professor of Otolaryngology-Head & Neck Surgery, has made a lot of progress in preclinical work developing a gene therapy for congenital hearing loss associated with mutations in the TOMT gene, a rare form of hearing loss. The technology works by using local delivery of a therapeutic viral vector to replace mutant copies of the TOMT gene specifically in cochlear hair cells.
Dr. Cunningham, Thanos Tzounopoulos, PhD, Director of the Pittsburgh Hearing Research Center and Vice Chair of Basic Science Research in the Department, and José P. Zevallos, MD, MPH, FACS, Chair of the Department, have formed a startup company called Echogenesis Therapeutics to continue development of their gene therapies for hearing loss. They have received funding for this from the NIH STTR program, the EEF Wiegand Entrepreneurial Award, the Pitt Innovation Institute, and the RNID in the UK. The company has also IP filed in the US, EU, Japan, China, South Korea, Canada, Israel, and Australia for its technology.
These gene therapies have been successful in mouse models, and the FDA has been very supportive of the work and immediate plans, which include additional development and testing in large animal models. Echogenesis is currently raising more funds from angel investors, incubators, foundations, and the NIH, and has plans for clinical trials in the future.
“In addition, our technology has the potential to be applied to many other forms of hearing loss,” Dr. Cunningham said. “It specifically targets cochlear hair cells, which are the main cell type affected in many different forms of hearing loss. We are currently expanding our therapeutic development toward treating age and noise-related hearing loss with a gene therapy that would be specifically targeted to hair cells. This work is in earlier stages, but we are very excited about it because it has the potential to be used for many patients at different stages of life.”