mutations including: Cystic fibrosis, Duchene Muscular Dystrophy, Usher syndrome, Ataxia-telangiectasia, Beta thalassemia, Tay-Sachs Hurler syndrome and many others.

Eloxx Pharmaceuticals approach and knowhow allows moving rapidly from target drugs discovery program to clinical drug candidates. Building a robust pipeline of molecules designed for an array of nonsense genetic diseases. Translation of mRNA to a protein is the event where the cellular machinery, i.e. ribosome, ultimately dictates the quantity and timing for each protein to be produced. Thus, molecules that induce ribosomal readthrough overcome the nonsense mutations and allow production of a full-length functional protein. These molecules hold great therapeutic potential for the treatment of many genetic diseases.