"Today the therapy for homocystinuria concentrates on lowering the concentration of a single metabolite, homocysteine, while metabolites further downstream are unaffected by the available treatment," said Professor Kraus. "However, these downstream metabolites may be of considerable clinical significance, and our approach has the potential to restore all the affected metabolites to their normal level, and as such reduce or prevent symptoms and enable a return to a normal diet."
CBS-deficient homocystinuria impacts at least 1 in 300,000 people worldwide (though prevalence may be higher due to under-diagnosis). It is designated by the U.S. Office of Rare Diseases Research (part of the National Institutes of Health) as a rare disease. Because of the smaller market, drugs to treat these 'orphan diseases' are eligible for special financial and clinical trial incentives (including fast-tracked FDA approval) and extended exclusivity periods in the U.S, Europe and other countries.
"We are excited to partner with CU and Professor Jan Kraus, a pioneer in the field of homocystinuria, in order to advance the development of a potential therapy for this tragic disease," said Josef Rosenberg, Director of Orphan Technologies. "Our collaboration is intended to accelerate the translation of fundamental advances in enzyme biochemistry at CU into the introduction of a new clinical therapy. Our alliance with CU constitutes another important step towards the reinforcement of Orphan's presence in the field of rare diseases worldwide."
"We are encouraged by the preclinical data for this technology and believe that it could bring hope for a patient population desperately in need of clinical alternatives," said David Poticha, a senior licensing manager at CU's
|Contact: Lindsey Lennox|
University of Colorado Denver