The University of Colorado (CU) has signed exclusive, worldwide licensing and collaboration agreements with rare-disease research-and-development firm Orphan Technologies Ltd to develop an enzyme replacement therapy for Cystathionine Beta-Synthase (CBS) -deficient homocystinuria, a rare, inherited metabolic disease that is often fatal at a young age. The collaboration was initiated in 2010 and includes sponsored-research funding by OT to continue development of the treatment approach pioneered by Jan P. Kraus, a professor of pediatrics at the CU School of Medicine. Orphan Technologies plans to begin first-in-human trials in 2015.
Clinical trials to commence for ERT for Classical Homocystinuria
