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Eloxx Pharmaceuticals

  • Robert Ward, Eloxx Pharmaceuticals


Rare and Ultra-Rare Genetic Diseases

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are designed 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 extending mRNA half-life and increasing 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.

  • Date:Monday, February 11
  • Time:3:15 PM - 3:30 PM
  • Room:Hudson/Empire
  • Location:7th Floor
  • Session Type:Company Presentation
  • Company Presentation - Presentation Type:Publicly Traded Company
  • Submission ID:23539
  • Goal for Presentation:Reach Investors and Collaborators
  • Company
  • Company HQ City:Waltham
  • Company HQ State:Massachusetts
  • Company HQ Country:United States
  • Market Cap:$435 million
  • Ticker:ELOX
  • Exchange:Nasdaq
  • CEO/Top Company Official:Robert E. Ward
  • Year Founded:2013
  • Main Therapeutic Focus:Gene/Cell Therapy
  • Lead Product in Development:ELX-02 in Cystic Fibrosis, Library of Molecules for rare diseases
  • Development Phase of Primary Product:Phase II
  • Number Of Unlicensed Products (For Which You Are Seeking Partners):3
  • Additional Information/Comments:ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis and cystinosis. 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 drug candidates designed to be eukaryotic ribosomal selective glycosides identified based on read-through potential. Eloxx recently announced a new program focused on rare ocular genetic disorders. 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.
Robert Ward
Eloxx Pharmaceuticals