Eloxx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, focuses on developing ribosome modulation for the treatment of rare and ultra-rare premature stop codon diseases. Its lead investigational drug product candidate is ELX-02, which is in Phase 2 clinical trial for the treatment of cystic fibrosis and nephropathic cystinosis patients with diagnosed nonsense mutations. The company also develops various preclinical programs for the treatment of alport syndrome, recessive dystrophic epidermolysis bullosa, junctional epidermolysis bullosa, and familial adenomatous polyposis, as well as various earlier discovery stage programs in oncology. Eloxx Pharmaceuticals, Inc. was founded in 2013 and is headquartered in Watertown, Massachusetts.
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Kidney morphology improved in all three patients with protein re-expression consistent with disease regression in its Phase 2 open-label clinical trial of ELX-02 for the treatment of Alport Syndrome ...
ELX-02 treatment improved podocyte foot process effacement in all three patients by an average of 60% based on a blinded kidney biopsy analysis by NIPOKA GmbH Biopsy results support clinical benefit ...
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