Regeneron gives first glimpse at hearing loss gene therapy

A child un­der two years old who re­ceived an ex­per­i­men­tal gene ther­a­py from Re­gen­eron in one ear saw im­proved au­di­to­ry re­spons­es through six weeks, ac­cord­ing to the com­pa­ny.

In Sep­tem­ber, it ac­quired the gene ther­a­py as part of its $109 mil­lion pur­chase of long­time col­lab­o­ra­tor Deci­bel Ther­a­peu­tics. Re­gen­eron said there were no safe­ty red flags through six weeks.

The gene ther­a­py is in­ject­ed in­to the in­ner ear and de­liv­ers a func­tion­al copy of a gene called otofer­lin. Peo­ple with mu­ta­tions in the otofer­lin gene ex­pe­ri­ence hear­ing loss as a re­sult of de­fec­tive sig­nal­ing to the brain from the ear’s sound-sens­ing hair cells.

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