Review highlights auditory recovery and safety of inner-ear gene therapies in genetic deafness, demonstrating the feasibility of biologic interventions in clinical otolaryngology.
Key Points
To review the evolving landscape of inner-ear gene therapy for hereditary hearing loss, addressing clinical trial design, translational pipelines, regulatory strategies, and ethical considerations.
Evaluated emerging preclinical therapeutic modalities including gene replacement, antisense oligonucleotides, RNA interference, and genome editing.
Early-phase clinical trials for DFNB9 deafness demonstrated favorable safety profiles and meaningful auditory restoration, leading to regulatory approval for Otarmeni.
Preclinical pipelines are actively expanding to address recessive, dominant, and syndromic hearing loss forms.
Primary translational challenges include outcome measure heterogeneity, long-term durability uncertainties, regulatory complexities, and global treatment access inequities.