Regeneron Pharmaceuticals’ gene therapy for otoferlin-related hearing loss, a genetic cause of deafness, has restored hearing to normal levels in one deaf child within just 24 weeks.
Sensorion, a biotech specializing in therapies for hearing loss disorders, has been granted Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for its lead gene therapy candidate, OTOF-GT.
Eli Lilly and Company will acquire Akouos, a precision genetic medicine company developing a portfolio of adeno-associated viral gene therapies for the treatment of inner ear conditions, including sensorineural hearing loss.