Rare Genetic Diseases
6 undisclosed programs RD/Clin0 epidermolysis bullosa, cystic fibrosis, familial adenomatous polyposis, colorectal cancer, oncology, undisclosed
Eloxx Pharmaceuticals is developing ribosome targeted genetic therapies. Not all diseases are caused by mutations in the amino acid sequence of a protein. Around 10-12% of mutations are nonsense mutations that cause the synthesis of truncated proteins affecting more than 100,000 patients. Eloxx aims to correct these mutations with their TURBO-ZM technology and Eukaryotic Ribosomal Selective Glycosides Library that allows rapid synthesis of novel oral Ribosome Modulating Agents (RMAs). RMAs are optimized compared to traditional macrolides to modulate protein synthesis, minimize antibiotic activity and be orally and chronically delivered. Eloxx has three initial programs focused on developing therapies for cystic fibrosis, junctional epidermolysis bullosa and APC mutant colorectal cancer.