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Eligo Bioscience's EB003 is a first-in-class CRISPR-based microbiome gene therapy designed to treat Shiga-toxin producing E. coli (STEC) infections and prevent hemolytic uremic syndrome (HUS). It utilizes a non-replicative DNA payload encoding a CRISPR-Cas nuclease guided towards stx virulence genes in bacteria. This mechanism leads to the inactivation of stx genes, preventing Shiga toxin production and causing bacterial death, thereby offering a precise approach to modulate the microbiome. Developed by Eligo Bioscience, it has received Orphan Drug Designation and Rare Pediatric Disease designation from the FDA and EMA.
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