#South Korea #GC Biopharma #Yongin #Rare Diseases #lysosomal storage disorders

GC Biopharma's Breakthrough Updates on Rare Disorder Drugs at WORLD Symposium 2025

GC Biopharma, a prominent South Korean biopharmaceutical firm, shared noteworthy developments regarding its drug pipelines targeting lysosomal storage disorders at the WORLD Symposium 2025, held from February 3 to 7 in San Diego, USA. This international conference served as a platform for experts in lysosomal storage disorders to congregate and exchange valuable insights aimed at advancing treatment methodologies for these debilitating conditions.

Among the significant highlights from this year's presentations were GC Biopharma's detailed posters depicting successful non-clinical study outcomes for two key drug candidates: GC2025A, an innovative oral chaperone therapy tailored for GM1 gangliosidosis, and GC1130A, which targets Sanfilippo syndrome type A (MPS IIIA).

Understanding the Disorders

GM1 gangliosidosis is a neurodegenerative disorder stemming from a hereditary deficiency of the β-Galactosidase 1 (GLB1) enzyme, impacting approximately 1 in 100,000 newborns. Most affected children begin to display symptoms before the age of six, and unfortunately, there is currently no effective cure to halt or reverse the disease, which is marked by severe seizures and muscle weakening.

During GC Biopharma's presentation, the company shared compelling non-clinical data demonstrating that when GC2025A was administered orally to animal models over a seven-day period, the results indicated a more than 70% reduction in GM1 levels within the brain. This promising evidence showcases the potential GC2025A may hold for transforming the treatment landscape for GM1 gangliosidosis.

A New Hope for Sanfilippo Syndrome

Sanfilippo syndrome, on the other hand, is characterized by an accumulation of heparan sulfate (HS) due to inherited genetic defects, leading to severe neurological decline. Patients often experience tragic outcomes, with many succumbing around the age of 15. Despite the urgency of unmet medical needs in this area, there remains a significant lack of approved therapies.

In GC Biopharma's findings for GC1130A, the data highlighted robust drug delivery via intraventricular (ICV) injection when compared to traditional intrathecal methods. The ICV administration successfully reduced levels of cerebrospinal fluid (CSF) HS and neuro-inflammatory biomarkers in mouse models of the disease, demonstrating a critical advancement in helping to address the neurological deficits associated with Sanfilippo syndrome type A.

Collaborative Efforts and Future Directions

GC1130A stands as a First-in-Class treatment developed in collaboration with Nobel Pharma and is currently progressing through Phase 1 multinational clinical trials across the U.S., South Korea, and Japan, following IND approvals in each jurisdiction.

Sookyung Shin, the Head of the Medical Division at GC Biopharma, remarked, "We will continue to build on our experience, knowledge, and know-how in developing treatments for lysosomal storage diseases and expand our research into other rare conditions to provide novel solutions for patients in need."

About GC Biopharma

Established as a leading force in biopharmaceuticals, GC Biopharma (formerly Green Cross Corporation) has dedicated over fifty years to producing life-saving therapeutics and vaccines. Headquartered in Yongin, South Korea, the company has earned its reputation as a key player in the global plasma protein and vaccine industry, continuously striving to deliver high-quality healthcare solutions.

In conclusion, as GC Biopharma forges ahead in addressing the pressing challenges posed by rare lysosomal storage disorders, the presentations at the WORLD Symposium 2025 reflect a commitment to innovation and a brighter future for affected patients worldwide.