GC Cell

GC Biopharma, a South Korean biopharmaceutical company, announced on Feb. 10th that it presented development updates on its LSD (lysosomal storage disorder) pipelines at the WORLD Symposium 2025, held from Feb. 3rd to 7th, 2025, in San Diego, USA.

WORLD Symposium 2025 is an international conference for lysosomal storage disorders experts to share and exchange insights for researching better treatment of the disorders. 

At this year's symposium, the latest advances in research of the rare diseases were shared by many presenters, including GC Biopharma's poster presentations on the non-clinical study results of "GC2025A", its oral chaperone therapy for GM1 gangliosidosis (GM1), and "GC1130A", for Sanfilippo syndrome type A (MPS IIIA).

GM1 gangliosidosis is a neurodegenerative disorder caused by an autosomal recessive deficiency of the enzyme β-Galactosidase 1 (GLB1). This disorder affects approximately 1 in 100,000 newborns, with the majority of those exhibiting symptoms before the age of 6. Currently, no effective treatment is available to cure or reverse the progression of this devastating disease, which is characterized by seizures and muscle weakness.

At its poster presentation, GC Biopharma unveiled promising non-clinical trial data for its GM1 candidate drug. The results demonstrated that, when administered orally to GM1 animal models over a 7-day period, the drug led to a remarkable reduction of more than 70% in GM1 levels within the brain.

The presentation continued to share the non-clinical study result of GC1130A, a Sanfilippo syndrome type A (MPS IIIA) drug. Sanfilippo syndrome is a rare disease inherited in an autosomal recessive trait, in which heparan sulfate (HS) accumulates in the body due to a genetic defect, causing gradual and severe brain deficit, mainly in the brain. This rare disease causes most patients to die around the age of 15. With no approved treatment out there so far, there are huge unmet medical needs among patients.

In the presentation, GC Biopharma shared that the non-clinical study results demonstrated robust drug delivery through ICV injection, as compared to intrathecal (IT). ICV administration effectively delivers GC1130A to the brain resulting in a reduction of cerebrospinal fluid (CSF) HS and neuro-inflammatory biomarkers in MPS IIIA disease mice model.

GC1130A is a First-in-Class drug that GC Biopharma is developing in collaboration with Novel Pharma. GC1130A is currently under Phase 1 multinational clinical trial in the U.S., Korea, and Japan based on IND approvals from each country.

Sookyung Shin, Head of Medical Division at GC Biopharma stated, "We will continue to build on our experience, knowledge, and know-how in developing treatments for lysosomal storage diseases and expand our reach to other rare diseases to provide new treatment options to the patients."