Harnessing Science

Targeting Rare and Ultra-rare Diseases

We intend to be the global leader in the application of the science of translational read-through and the associated pathway of nonsense mediated decay (NMD). We believe that expanding our expertise across these basic science areas of mRNA regulation, ribosomal function, and protein translation forms a solid foundation to support our discovery and development activities.

Our eukaryotic ribosomal selective glycoside (ERSG) compounds modulate the activity of the ribosome, a complex of RNAs and proteins, and therefore, a ribonucleoprotein, responsible for protein production, a process also known as translation. These novel small molecule ERSG compounds are designed to allow the ribosome to read-through a nonsense mutation in mRNA (which is transcribed from the DNA sequence), to restore the translation process to produce full-length, functional proteins and increase the amount of mRNA that would otherwise be degraded as part of a phenomenon called nonsense mediated mRNA decay. As our ERSG compounds target the general mechanism for protein production in the cell, we believe they have the potential to treat hundreds of genetic diseases where nonsense mutations have impaired gene function. Since nonsense mutations may occur at different positions within a given gene, a potential advantage of the small molecule ERSG approach is being able to use one molecule to address a range of mutations within a given disease state. Our subcutaneously injected ERSG molecules have the potential to be self-administered for systemic disease and to be active at most tissue locations across the body.

We hold worldwide development and commercialization rights to ELX-02 and other novel compounds in our ERSG read-through library, for all indications, in all territories, under a license from the Technion Research and Development Foundation Ltd. Professor Timor Baasov, the inventor of our compounds, who has served as our senior consultant since our inception.

Target Profile
for an ERSG

Eloxx’s read-through program is pursuing product candidates with the following characteristics:

  • Activity independent of gene size or complexity of genetic disorder
  • Molecular scaffold with defined ribosomal effect
  • Active at all three premature stop codons
  • Reduces rate of nonsense mediated decay
  • Restores functional protein production to a clinically significant level
  • Acceptable tolerability profile
  • Suitable for chronic administration


Our research and development strategy targets rare or ultra-rare diseases where: a high unmet medical need exists, identified nonsense mutation-bearing patient population is established, preclinical read-through can be established in predictive personalized medicine models, and a defined path through Orphan Drug development, regulatory approval, patient access and commercialization is identifiable. We believe patient advocacy to be an important element of patient focused drug development and seek opportunities to collaborate with patient advocacy groups throughout the discovery and development process. Our lead investigational drug product candidate, ELX-02, is in Phase 2 proof of concept clinical trials in cystic fibrosis.