USA – Eli Lilly has deepened its investment in genetic therapies by partnering with South Korean biotech firm Rznomics in a deal worth over US $1.3 billion in potential milestone payments.
Announced on May 15, the collaboration focuses on developing RNA-based treatments for sensorineural hearing loss, a common form of hearing damage caused by inner ear or nerve problems.
At the heart of this agreement is Rznomics’ proprietary RNA-editing technology based on trans-splicing ribozymes.
Unlike traditional gene editing tools such as CRISPR, which make permanent changes to DNA, this platform works at the RNA level.
RNA editing allows for precise corrections without altering a person’s genetic code, offering a potentially safer and more flexible approach to gene therapy.
Under the partnership, Rznomics will handle early-stage research and development. Eli Lilly will then step in to oversee preclinical work, clinical trials, and eventual commercialization.
While the size of the upfront payment remains undisclosed, the overall deal underscores Lilly’s growing commitment to innovative genetic treatments.
This move complements Eli Lilly’s existing work in the field. The company is already advancing a gene therapy candidate called AK-OTOF through its subsidiary Akouos.
AK-OTOF uses a dual AAV vector to treat hearing loss caused by mutations in the otoferlin (OTOF) gene.
In early 2024, promising trial results showed that a single dose of AK-OTOF restored hearing in an 11-year-old child within 30 days, with no serious side effects.
The AK-OTOF therapy is delivered through an injection into the cochlea and is designed to restore function to the inner ear’s hair cells.
Alongside this, Eli Lilly also signed a US $1.4 billion agreement in April 2025 with Sangamo Therapeutics to develop another gene therapy targeting the central nervous system.
The deal with Rznomics reflects a broader industry trend as drugmakers race to address hearing loss through gene and RNA therapies.
Competitor Regeneron is testing its DB-OTO gene therapy in a similar clinical trial, where early results showed significant hearing improvements in most pediatric patients.
Rznomics, which raised US $16.5 million in pre-IPO funding earlier this year, sees the Lilly partnership as a major step toward global recognition.
“This collaboration validates our RNA platform and brings hope for treating diseases that were once untreatable,” said CEO Seong-Wook Lee.