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GC1119

Development stage: Phase 1
Lead developer: GC Biopharma
Modality: Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
Administration: Intravenous

Overview

GC1119 is a recombinant human alpha-galactosidase A developed as an enzyme replacement therapy for Fabry disease. Fabry disease is an X-linked lysosomal storage disorder caused by deficiency of the enzyme alpha-galactosidase A, leading to accumulation of globotriaosylceramide (Gb3) in various tissues. GC1119 aims to restore enzymatic activity and reduce substrate accumulation in affected patients. The drug has been evaluated in multicenter, dose-escalation Phase 1 clinical studies for safety and exploratory efficacy in Fabry disease patients[1][2][3].

Other names: recombinant human alpha-galactosidase A

Targets

Gb3 (Globotriaosylceramide)

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