Lunsotogene parvec (lunsotogene parvec-cwha; OTARMENI ™ ) is a dual adeno-associated virus (AAV) gene transfer therapy developed by Regeneron Pharmaceuticals for the treatment of profound, congenital hearing loss caused by OTOF variants. Delivery of the OTOF transgene to the inner hair cells facilitates production of functional otoferlin protein to restore synaptic transmission to the auditory nerve....
✦ The floor
Discussion
Signed responses from readers of the wire.
This is the first approved dual-AAV gene therapy for congenital profound hearing loss; audiologists and CI teams should familiarise themselves with eligibility criteria and referral pathways as it enters clinical use.
The first regulatory approval of a gene therapy for congenital profound hearing loss marks a paradigm shift in the treatment landscape, potentially offering restoration of natural hearing rather than sensory substitution via cochlear implants.
- 01Lunsotogene parvec (OTARMENI) is the first dual AAV gene transfer therapy approved for congenital profound hearing loss.
- 02Developed by Regeneron Pharmaceuticals and targets a specific genetic cause of hereditary deafness.
- 03Uses a dual adeno-associated virus (AAV) vector to deliver a corrected gene copy to inner ear cells.
- 04Approval summary covers mechanism of action, clinical trial evidence, and safety profile.
- 05Represents a significant shift from implant-based to biology-based hearing restoration.
Lunsotogene parvec is the first approved dual AAV gene transfer therapy for profound congenital hearing loss.
press releasesupportedLunsotogene parvec restores hearing in patients with profound congenital hearing loss caused by a specific genetic mutation.
studypartially supportedDual AAV vector delivery achieves sufficient gene transfer to inner ear hair cells to produce a clinical benefit.
studypartially supported- PMID
- 42547733
- DOI
- 10.1007/s40265-026-02361-7.
- Journal
- Drugs
- Publication type
- review
- Evidence level
- 4
- Population
- Patients with profound congenital hearing loss due to specific genetic mutations
- Intervention
- Lunsotogene parvec (OTARMENI) — dual AAV gene transfer therapy
Primary outcomes
Regulatory approval basis and clinical trial evidence summary; Hearing restoration outcomes; Safety and tolerability profile
