About Opportunity:

Nephropathic cystinosis is a rare autosomal recessive disease. It is characterised by raised lysosomal levels of cystine in the cells of most organs. If untreated, the disease, results in death from renal failure by the second decade of life.

Cysteamine, the treatment for cystinosis, posesses an offensive taste and smell and irritates the gastrointestinal tract leading to nausea and vomiting following administration. In addition, cysteamine and its metabolites are excreted in breath and sweat. As a result of these problems patient compliance can be poor.

RGU has succeeded in the design and synthesis of a novel prodrug alleviating these patient compliance problems.