Eligo Bioscience, a leading in vivo gene-editing company, today 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.
About ODD and RPD
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. ODD provides Eligo Bioscience with development incentives including tax credits for clinical testing, prescription drug user fee exemptions, and seven-year marketing exclusivity in the event of regulatory approval.
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. If EB003 is approved for a Biologics License Application (BLA) by the FDA, Eligo Bioscience may be eligible to receive a priority review voucher (PRV), redemption of which will result in priority review for any subsequent marketing application.
EB003 is a first-in-class microbiome gene therapy designed, built, and optimized to target STEC bacteria in the gut of infected patients, leveraging Eligo’s unique expertise in synthetic biology, phage biology, genetic engineering, and bioinformatics. As opposed to antibiotics which unfortunately lead to Shiga toxin overproduction in the process of killing the STEC bacteria, Eligo’s proprietary sequence-specific CRISPR technology mechanism of action leads to the disruption of the genes coding for the Shiga toxin, and therefore offers a unique approach to this unmet need.
EB003’s mechanism of action relies on the in-situ delivery of a non-replicative DNA payload encoding a CRISPR-Cas nuclease guided towards stx virulence genes. In bacteria where such genes are present, the nuclease mediates a DNA double-strand break, which leads to the inactivation of the stx genes, preventing any production of Shiga toxin and also causing death of the bacteria as their DNA repair mechanisms are highly inefficient.
Eligo Bioscience is the world leader in microbiome in vivo gene editing therapy and is advancing a highly differentiated pipeline of precision medicines to address unmet medical needs in immuno-inflammation and oncology caused by the expression of specific deleterious bacterial genes by our microbiome.
Eligo was founded by scientists from The Rockefeller University and from MIT. Eligo was named a Technology Pioneer by the World Economic Forum and has received venture capital funding from Khosla Ventures and Seventure Partners, and non-dilutive funding from GlaxoSmithKline, the European Commission, CARB-X, and Bpifrance.