Shanghai startup’s gene therapy restores hearing in five deaf children, running ahead of Lilly and Regeneron trials

Five of six chil­dren who had been deaf since or soon af­ter birth can now hear af­ter re­ceiv­ing a gene ther­a­py de­vel­oped by Shang­hai Re­fresh­gene Ther­a­peu­tics, ac­cord­ing to a new study pub­lished in the Lancet Wednes­day.

The chil­dren in the tri­al had a mu­tat­ed ver­sion of a gene that en­codes otofer­lin, dis­rupt­ing sig­nal­ing to the brain from the ear’s sound-sens­ing hair cells. The ther­a­py, which is in­ject­ed in­to the in­ner ear, us­es a vi­ral vec­tor known as an AAV to de­liv­er a func­tion­al copy of the gene.

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