Lunsotogene parvec is a adeno-associated viral vector-based gene therapy for the treatment of severe-to-profound sensorineural hearing loss associated with biallelic OTOF gene variants. It acts by delivering functional copies of the human OTOF gene specifically to inner hair cells via an engineered hair-cell-specific Myo15 promoter, which restores essential synaptic transmission and physiological auditory signaling.
- Mechanism
- Curator reviewed · 2 references
Lunsotogene parvec is a dual adeno-associated virus serotype 1 (AAV1) vector-based gene therapy that utilizes an engineered hair cell-specific promoter derived from regulatory elements of myosin 15 (Myo15) to drive the complementary DNA (cDNA) expression of human OTOF transcript variant 5, which encodes isoform e of the otoferlin protein. Intracochlear infusion of the drug results in the expression of the OTOF gene primarily in inner hair, which facilitates the production of functional otoferlin protein and restores synaptic transmission to the auditory nerve.
- Primary indication
- Lunsotogene parvec-cwha is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved...Curator reviewed · 1 structured indication
- First approval
- United States, 2026
- Also known as
- DB-OTO · Lunsotogene parvec-cwha
- Brand names
Resolves to
What you can answer from here — as of August 10, 2026
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