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Eli Lilly gene therapy allows child born deaf to hear again after clinical trial

The first participant in the study, an 11-year-old with profound hearing loss from birth, had their hearing fully restored within 30 days of the therapy.

INDIANAPOLIS — A child's hearing loss was recently restored after a new 30-day clinical trial.

Eli Lilly and Company announced positive initial clinical results from a month-long trial of "AK-OTOF," its new gene therapy.

The first participant in the study, an 11-year-old with profound hearing loss from birth, had their hearing fully restored within 30 days of the therapy. 

"These initial results highlight the potential impact genetic medicines could have on individuals with OTOF-mediated hearing loss and reinforce our mission to make healthy hearing available to all," said Emmanuel Simons, who works on gene therapy for Eli Lilly.

Hearing loss is the most common sensory condition, and there is still no approved pharmacologic treatments for it. With this new therapy, millions of people across the world have the potential to hear again.

"Gene therapy for hearing loss is something physicians and scientists around the world have been working toward for over 20 years," said John Germiller, who administered AK-OTOF to the first child. "These initial results show that it may restore hearing better than many thought possible."

The results from this trial will be shared at the 2024 Association for Research in Otolaryngology MidWinter Meeting in hopes of growing the trial even more.

The treatment has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the Federal Drug Administration.

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