Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates are designed to be eukaryotic ribosomal selective glycosides (ERSGs) to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.
Read-through therapeutic development is focused on increasing functional protein synthesis by enabling the cytoplasmic ribosome to read-through premature stop codons to produce full-length proteins. Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical trials in cystic fibrosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx’s preclinical candidate pool consists of a library of novel ERSG drug candidates identified based on read-through potential. Eloxx is currently conducting IND-enabling studies in kidney diseases such as autosomal dominant polycystic kidney disease (ADPKD) and in rare ocular genetic disorders such as Usher Syndrome.
The company is led by a management team experienced in the development of rare disease therapeutics. All of us at Eloxx share the same goal: to bring safe and effective therapies to the children and adults suffering from genetic diseases who need them, as quickly as possible.