Eloxx Pharmaceuticals is a clinical-stage biopharmaceutical company developing small molecule drugs that target the ribosome to treat rare genetic diseases caused by nonsense mutations (NMs) — single-point DNA mutations that cause the ribosome to stop prematurely, producing truncated, non-functional proteins. Eloxx's drugs are designed to promote "readthrough" of these premature stop signals, enabling the ribosome to produce full-length, functional proteins. Eloxx's lead candidate, exaluren, targets Alport syndrome with NMs (NMAS), a genetic kidney disorder that leads to kidney failure and has no FDA-approved disease-modifying therapies. Eloxx also plans to develop exaluren for ADPKD with NMs, a common genetic kidney disease. A second candidate, ZKN-013, is licensed to Almirall for rare skin blistering diseases. Eloxx received $6M upfront and is eligible for up to ~$470M in additional milestones plus royalties on ZKN-013 sales. Eloxx is pre-revenue from product sales and funds operations through licensing income and external capital, most recently a $17M equity investment in August 2025. The company has only 10 employees and relies on CROs and third-party manufacturers. Eloxx was delisted from Nasdaq in 2023 and is currently working to regain compliance and pursue an uplisting.