Eligo Bioscience’s EB300 Gains Orphan Drug and Rare Pediatric Disease Designations
The FDA has granted Eligo Bioscience Orphan Drug and Rare Pediatric Disease designations for its investigational gene therapy EB003 for treating patients with Shiga-toxin producing bacterial infection to prevent hemolytic uremic syndrome (HUS), which affects the kidneys and blood vessels.
EB003 is a CRISPR-based therapy designed to target Shiga toxin-producing E. coli (STEC) bacteria in the gut of infected patients.
Preclinical data support EB003’s capacity to eliminate Shiga-toxin genes from patients’ gut, leading to a rapid decrease in toxin levels and associated symptoms and preventing their evolution towards hemolytic uremic syndrome, the company said.
There are currently no approved therapies for the potentially fatal syndrome.