Orphan Technologies currently has ongoing collaborations with multiple centers of excellence including:
University of Colorado
on multiple pre clinical programs in the Kraus lab at the University of Colorado School of Medicine. Dr. Kraus and his team are globally renowned leaders in the molecular basis of metabolic disorders including cystathionine Beta-synthase and Proprionyl CoA Carboxylase.
Saint Georges University of London
on an innovative and highly specialized cell-based and personalized ERT for Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE). This technology was invented by Bridget Bax, Ph.D one of the world’s leading authorities on MNGIE. It addresses the fundamental lesion in MNGIE by replacement of the deficient enzyme thereby preventing or reversing the severe and life-threatening complications in patients with MNGIE. Several patients with urgent medical needs have obtained access to the therapy through compassionate use treatment. OT-15 has been granted orphan designation by both FDA and EMA.
Partner of Choice
We understand that developing first-in-class therapies requires collaborating with the medical community, academic researchers, and patient groups. Fostering long term partnerships to advance patient care is part of our DNA and we are always looking to expand our pipeline with new innovative products targeting rare and ultra-rare diseases.