FDA grant Orphan Drug and Rare Paediatric Disease Designations for AK-OTOF for the treatment of otoferlin gene-mediated hearing loss

There are currently no pharmacologic treatments for OTOF-mediated hearing loss or any other form of sensorineural hearing loss. Nonclinical data reported to date for AK-OTOF, a gene therapy, demonstrate durable recovery of auditory function and support future clinical development

Biospace Inc.