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
Retrieved on:
Tuesday, October 11, 2022
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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.
Key Points:
- 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.
- EB003 is IND-enabled with a robust manufacturing process at the 100L-scale, full preclinical package and a clear regulatory path-to-clinic.
- The FDA grants Orphan Drug Designation to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S.
- The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases primarily affecting individuals ages 18 or younger and fewer than 200,000 individuals in the United States.