13: Progress and Challenges for Gene Therapy for Hearing and Balance Disorders

Hearing loss is a prevalent condition that affects millions globally. While hearing aids and implants alleviate the burden, personalized hearing medicine and inner ear therapeutics offer promising solutions. Gene therapies (gene replacement and gene editing) have demonstrated effectiveness in animal models, and ongoing studies continue to tackle the remaining challenges for their clinical translation. Maintaining baso-apical cochlear spatial tonotopic organization, selecting optimal vectors according to target cell types, and routes of administration for gene delivery, discovering new efficient nuclease variants, and identifying precise clinical endpoints are key considerations. The safety and efficacy of these therapies must be rigorously evaluated, particularly regarding immune responses and the overall safety of surgical procedures and gene delivery vectors. While challenges remain, ongoing research and collaborations between scientists, clinicians, audiologists, engineers, academic, private companies, patient organizations, and regulatory bodies are necessary to overcome obstacles and pave the way for effective and safe personalized inner ear therapies.