by Ellie Khadir, DVM, PhD, Scientist & Study Director, CBSET and Lise Byelyayeva, MSc, Associate Director, Sales, Funding, & New Partnerships, Cilcare

Hearing loss is a significant public health issue affecting over 466 million people globally. Hearing aids and cochlear implants fail to address the underlying causes. Gene therapy targets the fundamental genetic causes of hearing loss, providing true auditory regeneration and natural hearing restoration, offering hope for millions seeking effective solutions.

Gene therapy has advanced rapidly (2024-2025) with FDA approvals for Pfizer's Beqvez, PTC's Kebilidi, and Vertex's Casgevy, yet none of 44 approved therapies target hearing disorders. The current strategies gene replacement, editing, and silencing aim to address mutations across 173 genes linked to various inheritance patterns including dominant, recessive, X-linked, mitochondrial, and auditory neuropathy patterns. Monogenic disorders such as OTOF mutations are prime early targets, with approaches guided by mutation prevalence and timing before irreversible cochlear damage.

This paper outlines the translational roadmap from preclinical development to clinical implementation.