Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials


The associate professor of otolaryngology from Harvard Medical School also shared his excitement for the positive data on AAV1-hOTOF.

“This clearly shows how this kind of collaborative approach can really advance medical discovery much more rapidly, and in the end will help everybody… So that I think is one of the very important messages. We need to have this kind of collaboration, we can do more… to find out where the patient population comes from, to treat more patients, to do more trials, and then we can have a new treatment out quicker.”

AAV1-hOTOF gene therapy yielded significant, clinically meaningful improvements in hearing in 5 of 6 children with autosomal recessive deafness 9 (DFNB9) caused by mutations of the OTOF gene. The clinical trial assessing AAV1-hOTOF is being conducted in collaboration with investigators from Mass Eye and Ear at the Eye & ENT Hospital of Fudan University in Shanghai, China.

Investigators observed no serious adverse events or dose-limiting toxicities. The improvements included hearing recovery, improvements in speech recovery, and significant reductions in the average auditory brainstem response. One participant did not have a response to AAV1-hOTOF, and the investigators theorized that the nonresponse may have been due to their higher concentration of neutralizing antibodies at baseline, or, alternatively, a possible leakage of the AAV1-hOTOF solution from the round window membrane during or after surgery.

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 international trial and the collaboration that allowed it and enriched it. He shared his excitement for the positive data and adding to the body of supporting literature for gene therapy 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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