“Regeneron’s investigational gene therapy has been tied to notable hearing improvements in 10 of 11 children who were treated for a rare genetic condition that causes hearing loss.

‘You can see the efficacy very early on,’ Regeneron’s senior vice president and co-head of genetic medicines, Christos Kyratsous, Ph.D., told Fierce Biotech in an interview. ‘We are actively talking to FDA and other agencies around the world to understand what the clinical data set [is] but also everything else that you need to put together for your package to eventually get a drug approved.’

The findings come from a phase 1/2 trial, called CHORD, and build off earlier data demonstrating restored hearing for two patients who received the gene therapy.”

From Fierce Biotech.