Zheng-Yi Chen, DPhil, on Unmet Needs With Congenital, Sensorineural Hearing Loss


The associate professor of otolaryngology from Harvard Medical School discussed investigating gene therapy for DFNB9 deafness.

“For all hearing loss, no matter what cause: aging, noise-induced, etc, there's no single FDA approved treatment, that's key. So, we have a major disease category with no single treatment, you really can't think of any other major disease without any treatment, but hearing loss is one. So, that's why this is such an incentive for all of us working in the field to try to develop a new way to treat hearing loss.”

Five of 6 children with autosomal recessive deafness 9 (DFNB9) caused by mutations of the OTOF gene had significant, clinically meaningful improvements in hearing after receiving AAV1-hOTOF gene therapy. The children were treated as part of a clinical trial conducted at the Eye & ENT Hospital of Fudan University in Shanghai, China, in collaboration with investigators from Mass Eye and Ear.

The participants had hearing recovery, significant reductions in the average auditory brainstem response, and improvements in speech recovery. One nonresponding participant had higher concentrations of neutralizing antibodies at baseline, which may explain their lack of response, or, alternatively, there may have been possible leakage of the AAV1-hOTOF solution from the round window membrane during or after surgery. Altogether, there were no serious adverse events or dose-limiting toxicities.

CGTLive spoke with study investigator Zheng-Yi Chen, DPhil, associate scientist, Eaton-Peabody Laboratories, Mass Eye and Ear, and associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, to learn more about the unmet needs in treating patients with congenital deafness and other forms of hearing loss in general.

Lu J, Wang H, Cheng X, et al. AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial. Lancet. Published online January 24, 2024. doi: 10.1016/S0140-6736(23)02874-X
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