Gene therapy for hearing loss has moved from concept to clinic, with early trial results now available. The Fall Forum reviews outcomes from current pediatric trials, discusses what’s been learned about patient selection, timing, and delivery, and explores questions on durability, candidacy criteria, and how gene therapy fits alongside cochlear implantation.

Gene therapy for hearing loss has advanced rapidly from preclinical promise to early clinical reality. With the first FDA-approved gene therapy for hearing loss now available and multiple sponsored trials underway, the field has entered a new phase where early patient outcomes are beginning to reshape how clinicians think about candidacy, timing, and long-term management of genetic hearing loss.

The Fall Forum will open with a didactic overview covering early efficacy and safety data across current trials, surgical delivery approaches and how they compare to established procedures like cochlear implantation, and practical considerations in identifying and counseling candidate families, including the role of genetic testing in diagnosis and eligibility.

The format then shifts to case presentations with small group discussions where attendees can work through real clinical scenarios and hear directly from experts on a panel of experts. Discussion will address open questions the field is still working through: how durable are treatment effects over time, how should gene therapy be sequenced or combined with existing interventions like cochlear implants, which genetic etiologies are best suited to current approaches, and what infrastructure is needed to support broader access as more therapies move toward approval.

Attendees will leave with a grounded, case-based understanding of where gene therapy for pediatric hearing loss stands today and the clinical and research priorities shaping the next several years. This session is designed for clinicians, researchers, and family advocates seeking a realistic, evidence-based view of this rapidly evolving field.