Lunsotogene Parvec

Lunsotogene Parvec is indicated for the treatment of hearing loss caused by mutations in the OTOF gene, resulting in auditory neuropathy spectrum disorder (ANSD). It is intended for patients who have documented OTOF mutations and demonstrate inner hair cell dysfunction despite preserved outer hair cell function.

This gene therapy is administered via intracochlear infusion directly into the inner ear. It delivers a functional OTOF gene that enables inner hair cells to produce otoferlin, a protein essential for synaptic vesicle fusion and neurotransmitter release at the ribbon synapse. By restoring otoferlin production, the therapy re-establishes normal communication between inner hair cells and the auditory nerve, effectively treating the synaptic component of hearing loss.

Lunsotogene Parvec is an investigational therapy currently undergoing clinical evaluation. It requires surgical administration via intracochlear infusion by specialists experienced in inner ear procedures. Patients must undergo genetic testing to confirm OTOF mutations prior to treatment. Safety and efficacy data in human subjects are limited as this therapy is in early-stage development.