GC Pharma has signed a joint research and license agreement to develop orally administered chaperone therapy of gangliosidosis (GM1) with Japans’ Tottori University.

A neurodegenerative disease, GM1 is a recessively inherited genetic defect that leads to a lack of enzymes in the body. It happens to one in 100,000 newborns and usually before the age of six. In addition to neurodegeneration, it accompanies symptoms like seizures and muscle weakness. Currently, there is no treatment launched in the market.

To develop orally administered chaperone therapy of GM1, GC Pharma and Tottori University will look for new candidate materials.

In the joint research, GC Pharma will synthesize candidate materials and conduct a non-clinical toxicity test. From the stage of a clinical trial, GC Pharma will proceed independently. Through the contract, the company secured monopoly rights of the patent for new candidate materials and exclusive license.

Afterward, the company will challenge its global commercialization.

Tottori University will screen candidate materials and conduct a non-clinical efficacy test. The screening technology of the university was secured in cooperation with the National Institutes of Biomedical Innovation, Health and Nutrition (NIBIOHN), supported by the ID3 Booster Project of Japan Agency for Medical Research and Development (AMED). Professor Higaki Katsumi, the project’s leader, is an expert who has studied chaperone therapy of rare diseases for 20 years.

Tottori University will receive down payments, milestone payments, and the right to impose loyalty after the commercialization of the product.

“We will continue to cooperate in suggesting new ways of therapy that help patients suffering from rare diseases,” GC Pharma CEO Huh Eun-chul said.