Drug intelligence / Profile preview

EB003

Development stage
Unknown
Lead developer
Eligo Bioscience
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration
Oral
01

Overview

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.

Other names
Phage-derived, non-replicative delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli (STEC)
02

Targets

Stx2 (Shiga toxin 2)

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