For pediatric patients with autosomal recessive deafness 9 (DFNB9), bilateral gene therapy with adeno-associated virus (AAV) serotype 1 carrying human OTOF transgene is safe and efficacious, according to a study published online June 5 in Nature Medicine.

Hui Wang, M.D., from the Eye & ENT Hospital of Fudan University in Shanghai, and colleagues report on the results of an interim analysis of a single-arm trial examining the safety and efficacy of binaural therapy among five pediatric patients with DFNB9.

The researchers observed no dose-limiting toxicity at six weeks, nor any serious adverse events. Thirty-six adverse events occurred, with increased lymphocyte counts (six of 36) and increased cholesterol levels (six of 36) being the most common events. Bilateral hearing restoration occurred in all patients. The average auditory brainstem response threshold in the right (left) ear was restored to 58 (58) dB, 75 (85) dB, and 55 (50) dB in patients 1, 2, and 3, respectively, at 26 weeks and to 75 (78) dB and 63 (63) dB in patients 4 and 5, respectively, at 13 weeks compared with >95 (>95) dB in all patients at baseline. In all five patients, speech perception and capability of sound source localization were restored.

“Binaural AAV1-hOTOF gene therapy resulted in bilateral hearing restoration, the improvement of auditory and speech function, and the restoration of the ability of sound source localization in all treated patients,” the authors write.

One author is employed by Shanghai Refresh Therapeutics Co., which funded the study; a second author is cofounder of Salubritas Therapeutics.

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