Otarmeni (lunsotogene parvec-cwha) / Regeneron - LARVOL DELTA

DB-OTO Gene Therapy in Children with Profound Hearing Loss: CHORD Trial Early Speech Results (AAO-HNSF 2026) - P1/2 | "These data suggest that DB-OTO safely enables hearing that translates into favorable speech outcomes in children with OTOF-related deafness."

Clinical • Gene therapy • Gene Therapies • Otorhinolaryngology

A translational framework for early-phase inner-ear gene therapy: clinical trial design, regulatory strategy, and ethical considerations. (PubMed, Curr Opin Otolaryngol Head Neck Surg) - "The genetics of hearing loss is transitioning from a diagnostic modality to an interventional one. Widespread clinical impact will require advances in vector engineering, equitable implementation, multidisciplinary counseling, and integration with established rehabilitation pathways. For otolaryngologists, genetic literacy is becoming essential to contemporary hearing care."

First-in-human • Journal • Gene Therapies • Otorhinolaryngology

Lunsotogene Parvec: First Approval. (PubMed, Drugs) - "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...Lunsotogene parvec received its first approval on 23 April 2026 in the USA for the treatment of paediatric 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 outer hair cell function and no prior cochlear implant in the same ear. This article summarises the milestones in the development of lunsotogene parvec leading to this first approval for OTOF-associated sensorineural hearing loss."

Journal • Gene Therapies • Otorhinolaryngology • Pediatrics

Restoration, Not Bypass: Otoferlin Gene Therapy and a New Era in Hearing Loss Treatment. (PubMed, Otolaryngol Head Neck Surg) - "The FDA approval of Otarmeni, an AAV-based gene therapy for biallelic OTOF-associated sensorineural hearing loss, represents a conceptual shift in otology: from bypassing defective auditory physiology to restoring it...This commentary argues that otoferlin gene therapy initiates a new treatment hierarchy rather than rendering cochlear implantation obsolete: restore when biology permits, bypass when it does not. Realizing this potential will require advances in newborn genetic screening, workforce training, and equitable access to gene delivery."

Journal • Gene Therapies • Otorhinolaryngology

CHORD: A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants, Adolescents and Adults With Hearing Loss Due to Otoferlin Mutations (clinicaltrials.gov) - P1/2 | N=36 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial primary completion date: Feb 2032 ➔ Feb 2029

Trial primary completion date • Gene Therapies • Otorhinolaryngology

Lunsotogene Parvec-cwha. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

OTARMENI (lunsotogene parvec-cwha). (PubMed, Clin Ther) - No abstract available

Journal

The CHORD trial: an update on the efficacy and safety of DB-OTO gene therapy for profound deafness in children (ASGCT 2026) - P1/2 | "No correlation between observed immunogenicity and treatment outcomes was found. Conclusion Results from 12 participants through 48 weeks of observation demonstrate that DB-OTO provides rapid and significant hearing improvements that can translate to speech and language benefits in children with OTOF-related deafness, and is generally well tolerated."

Clinical • Gene therapy • Gene Therapies • Ophthalmology • Otorhinolaryngology

Otarmeni (lunsotogene parvec) Receives EMA Filing Acceptance for Genetic Hearing Loss (The Manila Times) - "The MAA is supported by data from the pivotal CHORD clinical trial, in which 24 participants (aged between 10 months to 16 years) received a single dose of Otarmeni via intracochlear infusion, either unilaterally (in one ear; n=10) or bilaterally (in both ears; n=14)."

EMA filing • Otorhinolaryngology

FDA Approval of the Congenital OTOF Gene Therapy Otarmeni Reshapes the Future of Hearing Care. (PubMed, J Assoc Res Otolaryngol) - No abstract available

FDA event • Journal • Gene Therapies

CHORD: A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations (clinicaltrials.gov) - P1/2 | N=30 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: Apr 2031 ➔ Feb 2032 | Trial primary completion date: Apr 2031 ➔ Feb 2032

Trial completion date • Trial primary completion date • Gene Therapies • Otorhinolaryngology

Novel OTOF slice site mutations found to be pathogenic (COSM 2026) - "The individuals were treated with DB-OTO intracochlear gene therapy under the CHORD trial with Regeneron (Tarrytown, NY)... A 3-year-old male was found to have profound hearing loss with present OAEs and cochlear microphonics on auditory brainstem response testing consistent with auditory neuropathy spectrum disorder (ANSD). Genetic testing was performed and he was found to be homozygous for a variant of uncertain significance, OTOF g.26684551C>A [c.5533+13G>T] NM_194248.2, that was predicted to alter a splice site and result in a truncated protein. The patient had one sibling, a 1-year-old female, who was also found to have ANSD and the same homozygous mutation."

Gene Therapies • Otorhinolaryngology

Safety and Efficacy of DB-OTO Gene Therapy in Children With Profound Deafness Due to Otoferlin Variants: Data From the Chord First-In-Human Registrational Trial (ARO 2026) - P1/2 | "In this first-in-human registrational gene therapy trial, DB-OTO resulted in significant hearing improvements in participants with OTOF-related deafness. DB-OTO administration enabled natural acoustic hearing for the first time, and normalized hearing sensitivity in some participants."

Clinical • First-in-human • Gene therapy • P1 data • Gene Therapies • Otorhinolaryngology