BRIEF—GC Pharma and Tottori link up in inherited lysosomal storage disease

Korean company GC Pharma and Japan’s Tottori University have announced a worldwide licensing and collaboration agreement for the orally administrable chaperone drug of GM1 gangliosidosis (GM1).

GM1 is one of the inherited lysosomal storage diseases caused by mutations in the GLB1 gene, that progressively destroys nerve cells in brain and spinal cord. It occurs in approximately one out of every 100,000 newborns. There is currently no cure.

Under the terms of the agreement, GC Pharma has obtained the exclusive right to develop and commercialize worldwide. In return, Tottori University will receive an undisclosed upfront payment, as well as payments for the achievement of commercial milestones, with royalties based on sales. As part of the contract, the two organizations will jointly conduct and complete derivation of candidates and non-clinical trials.

In addition, GC Pharma will be responsible for clinical trials as well as global commercialization.