Eligo Bioscience Receives FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) Designation for EB003 for the Prevention of Hemolytic Uremic Syndrome with First-in-class CRISPR-based medicine

Eligo Bioscience announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) designation for its oral drug candidate EB003, for the treatment of Shiga-toxin producing bacterial infection as it relates to the prevention of hemolytic uremic syndrome (HUS).

“Granting of Orphan Drug Designation and Rare Pediatric Disease designation for EB003 highlights the FDA’s recognition of the potential of how our unique CRISPR-based modalities can be used to address devastating diseases driven by the expression of bacterial genes, such as hemolytic uremic syndrome,” said Xavier Duportet, Ph.D., Chief Executive Officer of Eligo Bioscience. “We are grateful that the FDA is providing additional support for the development of therapies geared towards rare pediatric diseases, and encourages us in our mission to propose highly innovative solutions to patients in need.”

Children under 5 years of age are particularly sensitive to the expression of Shiga toxins from E. coli (STEC) bacteria after their ingestion from contaminated foods. Toxin production in the gut triggers bloody diarrhea, and its translocation and accumulation in the systemic compartment can lead to HUS, a life-threatening clinical syndrome involving destruction of blood platelets, anemia and acute kidney injury. There are currently no approved therapies for this rare disease.

Strong preclinical data in multiple animal models support EB003’s capacity to efficiently and precisely eliminate Shiga-toxin genes from patients’ gut, leading to a rapid decrease in toxin levels and associated symptoms, and preventing their evolution towards HUS. EB003 is IND-enabled with a robust manufacturing process at the 100L-scale, full preclinical package and a clear regulatory path-to-clinic.

Incentives attached to the ODD and RPD designation support the development of EB003 and are further validations of the proprietary gene editing platform that Eligo is leveraging to build a pipeline of high-value drug candidates in immuno-inflammation and oncology.