Report: 10 out of 11 children born deaf gained some hearing through Regeneron gene therapy

I read a report from BioPharmaDive.com that said 10 out of 11 children who were born profoundly deaf from a very rare form of deafness caused by mutations in a gene known as OTOF experienced some degree of hearing improvement after receiving experimental gene therapy developed by Regeneron Pharmaceuticals, which is a New York company.

The report said a few of the children can now hear sound at near-normal levels, like conversational speech. One of them was 10 months old when treated and after a year could correctly identify spoken words like, “mommy,” “cookies,” and “airplane.” However, one child did not experience improvements in their hearing.

Some children received an “intracochlear injection” in one ear and others received bilateral injections. The process involves surgery and is similar to the approach in a cochlear implantation surgery.

Those with OTOF mutations have problems with passing along the vibrations of sound into a signal to the brain.

I’ve done several news reports in the past about gene therapies on deaf children involving OTOF. It’s a controversial topic in the Deaf community, which has long resisted the idea that we need to be “fixed” by science and medicine.

These types of gene therapy experiments are going on full-steam ahead, though, with other studies and trials conducted by U.S. pharmaceutical giant Eli Lilly, France’s Sensorion, and researchers at Fudan University in Shanghai.

The report said although the gene therapy trials have shown some measure of success, the field of gene therapy in general is struggling because funding has dried up and with OTOF therapy, it’s a complex therapy in a tiny market with only 20 to 45 new patients with OTOF-related hearing loss in the U.S. each year.

https://www.biopharmadive.com/news/regeneron-otoferlin-deafness-gene-therapy-db-oto-results/740810/

https://investor.regeneron.com/news-releases/news-release-details/laest-db-oto-results-demonstrate-clinically-meaningful-hearing