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An Eli Lilly & Co.
experimental gene therapy has restored the hearing of a child who was born deaf, the pharmaceutical giant said Tuesday.

The child, who was 11 years old at the time the therapy was administered, experienced restored hearing within 30 days of treatment, Eli Lilly said in a release. The child participating in the clinical study was the first individual in the U.S. to receive the therapy for a genetic form of hearing loss, the company said.

The gene therapy, AK-OTOF, is being developed for the treatment of hearing loss due to mutations in the otoferlin gene. Eli Lilly added AK-OTOF to its portfolio through its 2022 acquisition of Akouos Inc., a genetic-medicine company focused on inner-ear conditions.

Doctors and scientists have been pursuing gene therapy for hearing loss for more than 20 years, and “these initial results show that it may restore hearing better than many thought possible,” Dr. John Germiller, director of clinical research in the otolaryngology department at the Children’s Hospital of Philadelphia and principal investigator in the clinical trial, said in a statement.

The child’s hearing was restored across all tested frequencies and was within a normal range at some frequencies at 30 days after the treatment, Lilly said.

Children with hearing problems due to otoferlin gene mutations are often born with profound hearing loss, but most of them have not had the genetic testing needed for a definitive diagnosis, Dr. Oliver Haag, head of otolaryngology at Sant Joan de Deu Hospital in Barcelona and an Akouos study investigator, said in a statement.

Roughly 200,000 people worldwide have hearing loss related to otoferlin gene mutations, Lilly said. For some of these people, the company said, hearing can be restored by delivering a healthy version of the gene to a target cell within the inner ear.

The study results, including initial data from a second patient to receive the therapy, will be presented at the Association for Research in Otolaryngology’s meeting on Feb. 3 in Anaheim, Calif., Lilly said.

AK-OTOF has been granted the U.S. Food and Drug Administration’s orphan-drug designation, which gives companies developing treatments for rare diseases tax credits for qualified clinical trials, along with other perks.

Lilly shares were down 0.2% Tuesday afternoon and have gained 84% over the past 12 months, while the S&P 500
is up 20.6% in that period.

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