Funded by Génome Québec and Genome Canada under the Genome Canada Genomic Applications Partnership Program (GAPP), this trial will be led by Dr. Paul Goodyer, a professor of pediatrics at McGill University and recognized leader in hereditary renal disease. 

ELX-02 has the potential to improve the lives of cystinosis* patients. In fact, researchs led to this day by Dr. Goodyer’s team showed that this molecule decreases the cystine content in cellular and animal models.

Dr. Goodyer works in partnership with Eloxx Pharmaceuticals, an american biopharmaceutical company dedicated to the discovery and development of therapeutics to treat cystic fibrosis, cystinosis, inherited retinal disorders, and other diseases caused by nonsense mutations. 

Read Eloxx’s press release 

Learn more about Dr. Paul Goodyer’s project


* Cystinosis is a metabolic disease characterized by an accumulation of cystine inside the lysosomes, causing damage in different organs and tissues, particularly in the kidneys and eyes. Source