SB012

SB012 is a first-in-class DNAzyme-based GATA-3 antagonist developed for the treatment of moderate to severe ulcerative colitis. It is formulated as an enema containing the DNAzyme hgd40, which specifically binds and cleaves GATA-3 mRNA, thereby inhibiting the expression of the GATA-3 protein. GATA-3 is a master transcription factor that regulates Th2-driven inflammatory responses by controlling cytokines such as IL-4, IL-5, and IL-13. By targeting and degrading GATA-3 mRNA, SB012 downregulates these pro-inflammatory cytokines and reduces mucosal inflammation in ulcerative colitis. The drug has demonstrated safety and efficacy in Phase IIa clinical trials with significant improvements in disease activity scores compared to placebo[1][4][7]. DNAzymes like hgd40 are single-stranded synthetic DNA molecules with catalytic activity that enables them to cleave specific RNA targets.