Korea’s GC Biopharma expands mRNA development to include rare disease treatment

GC Biopharma Corp.’s Hwasun plant in South Jeolla Province. [Courtesy of GC Biopharma]

South Korean biopharmaceutical company GC Biopharma Corp. is set to develop a treatment for succinic semialdehyde dehydrogenase deficiency (SSADHD), a rare and incurable disease, in the form of messenger ribonucleic acid (mRNA).

According to multiple industry sources on Wednesday, GC Biopharma has recently decided to transform its SSADHD drug into an mRNA formulation instead of protein.

The change in formulation has resulted in a longer half-life, according to the company. GC Biopharma has taken the first step in its strategy to develop the next growth engine by expanding mRNA development to include innovative new drugs for rare diseases.

SSADHD is an inherited neurodegenerative disease caused by a lack of an enzyme from gene defects and affects one in one million people.

GC Biopharma has been jointly developing a treatment with U.S. Speragen Inc. since 2021 with the goal of producing the world’s first-in-class drug for the disease. The company plans to come up with the final candidate material in the first half of next year.

GC Biopharma has been working hard to develop mRNA as one of its new growth engines on the belief that its existing vaccine business faces limited growth.

In April last year, it confirmed the possibility of vaccine development in a development option agreement related to lipid nanoparticle (LNP) signed with Canada-based Acuitas Therapeutics Inc. and exercised the option in the license agreement early this year.

The company aims to accelerate mRNA projects centered on vaccines and rare disease therapeutics by introducing LNP technology, which is critical for mRNA development.

Currently, its mRNA flu vaccine is being developed with the objective of entering Phase I clinical trials next year.

The Korean drug maker is also making preemptive investments in production. The company plans to set up an mRNA pilot production facility at its Hwasun plant in South Jeolla Province, which produces conventional flu vaccines.

In March this year, it announced a plan to invest 15 billion won ($11.18 million) to expand its mRNA facilities in Hwasun. It will complete construction and begin trial operations this year with plans to directly produce mRNA-related vaccines and therapeutics.