The partnership with Rznomics strengthens Lilly’s multi-pronged approach to hearing restoration, combining both gene therapy and RNA-editing platforms.
USA— Eli Lilly has announced a significant collaboration with South Korean biotech company Rznomics to develop innovative RNA-based gene therapies targeting sensorineural hearing loss, a common form of hearing impairment.
The deal, disclosed on May 15, 2025, could be worth over US$1.3 billion in milestone payments, although the upfront payment details remain confidential.
Under this partnership, Rznomics will use its proprietary trans-splicing ribozyme RNA platform to lead early-stage research.
This platform enables precise RNA editing, a process that modifies RNA molecules to correct genetic errors without altering the underlying DNA.
Unlike DNA-editing technologies such as CRISPR, which make permanent changes to the genome, RNA editing is reversible and allows targeted corrections, potentially offering greater safety and specificity.
Following the initial research phase, Eli Lilly will take responsibility for preclinical development, clinical trials, and commercialization of the therapies worldwide.
This collaboration complements Lilly’s existing efforts in genetic hearing loss treatments, notably its AK-OTOF gene therapy developed by its subsidiary Akouos.
AK-OTOF uses a dual adeno-associated virus (AAV) vector to deliver functional copies of the otoferlin (OTOF) gene to the inner ear and is currently in Phase I/II clinical trials.
Early results from January 2024 showed promising outcomes, with an 11-year-old patient regaining hearing within 30 days after a single treatment, and no serious side effects reported.
The partnership with Rznomics strengthens Lilly’s multi-pronged approach to hearing restoration, combining both gene therapy and RNA-editing platforms.
This strategic move is part of Lilly’s broader commitment to genetic medicine, which also includes a US$1.4 billion deal signed in April 2025 with Sangamo Therapeutics to develop gene therapies for central nervous system diseases.
The focus on genetic therapies for hearing loss is gaining momentum across the industry.
For example, Regeneron is developing DB-OTO, a dual AAV gene therapy currently in Phase I/II trials, which has shown measurable hearing improvements in most pediatric patients treated.
Rznomics, which has raised approximately US$58 million in funding, including $16.5 million in pre-IPO capital earlier this year, views this collaboration as a major step in expanding its global biotech presence.
CEO Seong-Wook Lee emphasized the potential of their RNA-editing platform to treat diseases that were previously difficult to address, highlighting the partnership as a validation of their technology and a gateway to developing precision RNA therapeutics.
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