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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