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GC Pharma and Tottori University Enter Research Collaboration and License Agreement for Development of GM1 Gangliosidosis Chaperone Therapy

By Alex

Posted on August 2, 2021

- Collaboration aims to accelerate global development of the chaperone therapy, leveraging expertise and resources of both parties

YONGIN, South Korea--(BUSINESS WIRE)--GC Pharma (006280.KS) and Tottori University today 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 1 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 is entitled to receive an upfront payment, as well as payments for the achievement of commercial milestones, with royalties based on sales. As part of the contract, 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.

"This collaboration is another significant milestone in delivering our vision for rare disorders,� said EC Huh, Ph.D., president of GC Pharma. �Our ultimate goal is to make a meaningful difference in the lives of those with rare diseases. We expect this partnership to make substantial improvement in the lives of those patients.�

Tottori University is dedicated to contribute the international research collaboration with GC Pharma to develop and deliver an innovative chaperone therapy for patients with this devastating neurodegenerative disease.

The screening technology to identify potential chaperone compounds was co-developed by Tottori University and NIBIOHN (National Institutes of Biomedical Innovation, Health and Nutrition) with the support of AMED�s (Japan Agency for Medical Research and Development) the iD3 Booster (the Project for accelerating the translation of promising basic researches into innovative new medicines). Katsumi Higaki, Ph.D., head of GM1 research at Tottori University, has been working on the chaperone therapy related to orphan disorders for more than 20 years.

The chaperone therapy is a latest molecular therapeutic approach to lysosomal diseases using small molecules that stabilize the mutant enzyme proteins and boost the enzyme activity. GC Pharma and Tottori University will jointly explore potential targets with the goal to develop chaperone compounds, and orally administrable drug option for patients.

About GM1 gangliosidosis

GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-galactosidase (�gal) enzyme. The resulting enzymatic deficiency leads to accumulation of GM1-ganglioside in cells. Clinical presentation is mainly neurological with rapidly progressive impairment (motor, cognitive and behavioral) leading to premature death, mostly in early childhood. It is a devastating disease for patients and families. There is currently no disease modifying treatment available.

About GC Pharma

GC Pharma (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Pharma is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century. Green Cross Corporation updated its corporate brand to GC Pharma in early 2018. Green Cross Corporation remains the company's legal name.

About Tottori University

Tottori University (National University in Japan), established in 1949, as a university rooted in the local community and playing an active role globally, contributes to the development of the region and the world through its energetic activities in different fields. In the research aspect, with its distinctive research facilities, such as the world-leading Arid Land Research Center, Fungus/Mushroom Resource and Research Center whose genetic resources are the world�s largest, and Chromosome Engineering Research Center, which is engaged in the fields of drug discovery and regenerative medicine, Tottori University disseminates new knowledge to the world. In addition, Faculty of Medicine Tottori University have strengths in a wide range of medical research including rare and intractable diseases in collaboration with public agency and other academia.

This press release may contain forward-looking statements, which express the current beliefs and expectations of GC Pharma's management. Such statements do not represent any guarantee by GC Pharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Pharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.