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Eli Lilly (NYSE:LLY) announced that its gene therapy candidate AK-OTOF restored hearing in the first participant in its Phase 1/2 AK-OTOF-101 study for a congenital form of hearing loss.
The patient, an 11-year-old boy who had had significant hearing loss for more than ten years, was the first to undergo gene therapy for a genetically-driven hearing loss in the U.S., the Indiana-based drugmaker said.
His hearing came back within 30 days from a single administration of the therapy, LLY noted, adding that initial data from a second participant to receive AK-OTOF will be presented at an ENT medical event in early February.
The surgical procedure used for drug administration and the treatment were well tolerated, with no serious adverse events.
The trial enrolled patients with a genetic form of hearing loss linked to mutations in the otoferlin gene (OTOF).
“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,” LLY’s senior vice president of gene therapy, Emmanuel Simons, remarked.
