GC-1130A
- Generic Name
- GC-1130A
GC Biopharma and Novel Pharma Initiate Phase I Trial of GC1130A for Sanfilippo Syndrome Type A
• GC Biopharma and Novel Pharma have dosed the first patient in a multinational Phase I clinical trial of GC1130A for Sanfilippo syndrome type A (MPS IIIA).
• The Phase I trial will assess the safety, tolerability, and efficacy of GC1130A in children aged 2-6 years with MPS IIIA over a two-year period.
• GC1130A is a first-in-class treatment using GC Biopharma’s platform to deliver concentrated recombinant protein via intracerebroventricular (ICV) injection.
• The trial will be conducted across multiple sites in the US, Korea, and Japan, addressing a critical unmet need for this devastating genetic disorder.
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