Eli Lilly Enters $1.3bn Gene Therapy Deal with Rznomics for Hearing Loss

Eli Lilly is bolstering its genetic medicine portfolio for hearing loss through a new partnership with South Korean biotech Rznomics, focusing on the development of innovative RNA-based gene therapies for sensorineural hearing loss. This agreement carries a potential value exceeding $1.3 billion in milestone payments.

Rznomics will utilize its proprietary trans-splicing ribozyme RNA platform to develop these RNA-editing therapies, which offer a reversible approach unlike permanent DNA editing methods. The company will handle early-stage research, with Eli Lilly taking over preclinical advancement, clinical development, and commercialization.

This RNA-based collaboration strategically complements Lilly’s existing efforts in genetic hearing loss, notably AK-OTOF, a dual adeno-associated virus (AAV) vector-based gene therapy currently in Phase I/II clinical trials. Developed by Lilly’s subsidiary Akouos, AK-OTOF targets hearing loss caused by mutations in the otoferlin (OTOF) gene, using an AAV vector for delivery.

Early data from the AAV-delivered AK-OTOF in January 2024 demonstrated restored hearing in a participant after a single intracochlear injection, with no serious adverse events. This AAV therapy delivers functional OTOF transgenes to the inner ear. Lilly’s broader commitment to gene therapy also includes a recent significant deal in the central nervous system space.

The collaboration with Rznomics underscores the growing industry focus on genetic solutions for hearing loss, with Regeneron also advancing a dual AAV gene therapy candidate, DB-OTO, showing promising early results. Rznomics views this partnership as a key step in its global expansion strategy.