Eli Lilly is strengthening its position in genetic medicine for hearing loss through a new collaboration with South Korea-based biotech Rznomics.
The agreement, centred on the development of RNA-based gene therapies for sensorineural hearing loss, carries a potential value exceeding $1.3bn in milestone payments. Rznomics disclosed the deal on 15 May, though financial details of the upfront payment were not shared.
Under the terms of the partnership, Rznomics will apply its proprietary trans-splicing ribozyme RNA platform to develop RNA-editing therapies. The company will lead early-stage research, while Eli Lilly will take over preclinical advancement, clinical development, and commercialisation efforts.
This approach is different to DNA editing approaches like CRISPR—clustered regularly interspaced short palindromic repeats (CRISPR)—which make permanent changes to the genome. RNA editing is a reversible process that allows for targeted corrections without altering the underlying genetic code.
This collaboration complements Lilly’s existing pipeline in genetic hearing loss therapies, which includes AK-OTOF, a dual adeno-associated virus (AAV) vector-based gene therapy currently in a Phase I/II clinical trial (NCT05821959). Developed by Lilly’s subsidiary Akouos, AK-OTOF targets hearing loss caused by mutations in the otoferlin (OTOF) gene.
In January 2024, early data from the trial showed that a single administration of AK-OTOF restored hearing in an 11-year-old participant within 30 days. The patient experienced improved hearing across multiple frequencies, with no serious adverse events reported.
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By GlobalDataAK-OTOF is administered through a unilateral intracochlear injection and is designed to deliver functional OTOF transgenes to the inner hair cells of the cochlea. The therapy is part of Lilly’s broader push into gene therapy. In April 2025, Lilly inked a deal worth $1.4bn with Sangamo Therapeutics to develop a gene therapy for a central nervous system disease.
The collaboration with Rznomics comes amid growing industry focus on genetic therapies for hearing loss. Regeneron is also pursuing a similar approach with DB-OTO, a dual AAV gene therapy candidate currently in a Phase I/II clinical trial (NCT05788536). According to data shared in February, 10 of 11 paediatric patients treated with DB-OTO showed measurable hearing improvements, with some reaching near-normal thresholds.
Rznomics raised KRW 20.3 bn ($16.5m) in pre-IPO funding in January, bringing its total raised capital to around $58m. The company said the partnership with Lilly is part of its strategy to “expand its presence in the global biotech arena,” in the 15 May announcement.
“This partnership validates our trans-splicing ribozyme platform and opens the door to treating previously intractable diseases with precision RNA therapeutics,” added Seong-Wook Lee, CEO of Rznomics.
