The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.

Design Brief:

• A profoundly deaf baby who received Regeneron’s experimental gene therapy was able to hear normally within six months, the company announced Wednesday.

• The data, presented at a medical conference, build on initially promising research released in October. The baby was dosed at 11 months of age, one of the youngest children in the world to receive gene therapy for genetic deafness, Regeneron said. A second patient, treated at age 4, also showed hearing improvements at a six-week assessment, the company said.

• Both children suffer from a type of deafness caused by mutations in a gene known as otoferlin. The initial research on Regeneron’s DB-OTO therapy is part of an ongoing Phase 1/2 trial known as CHORD enrolling infants and children in the U.S., U.K. and Spain.

Dive Insight:

Regeneron’s announcement follows news that Eli Lilly’s competing experimental gene therapy gave an 11-year-old boy who was born deaf the ability to hear. The treatment, which Lilly acquired in a buyout of biotechnology company Akouos, also targets otoferlin.

The results suggest gene therapy may become a viable therapeutic option for people with congenital deafness. The field has attracted a great deal of attention, as evidenced in abstracts of data presented at the American Society of Gene and Cell Therapy’s yearly meeting. One, from a group of researchers based in China and the U.S., found hearing recovery in 10 out of 11 patients.

Regeneron acquired its entrant in the field with its $109 million purchase of Decibel Therapeutics in August. The deal includes potential payments that could add up to $104 million more for Decibel shareholders for reaching certain milestones.

The success with such a young child in the Regeneron trial of DB-OTO may offer hope for other developmental milestones, such as speech, said Lawrence Lustig, a study investigator and chair of the department of otolaryngology – head and neck surgery at Columbia University. 

“The opportunity of providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime,” Lustig said in Regeneron’s statement. “We are excited to see how this translates into an individual’s development.”

DB-OTO is designed to deliver a working copy of the otoferlin gene with an injection into the cochlea after patients are sedated with general anesthesia. Like Lilly’s experimental treatment, Regeneron’s medicine uses a specially engineered virus to deliver the gene.

Regeneron’s therapy has received orphan drug, rare paediatric disease and fast track designations from the Food and Drug Administration.