Clinical trials to commence for ERT for Classical Homocystinuria

Clinical trials to commence for ERT for Classical Homocystinuria

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.

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