UBC has a proud history of leadership in rare disease gene discovery and the translation of genetic insights into tangible benefits for patients. The first ever gene therapy approved for clinical use in the Western world was developed at UBC. UBC ran one of the largest trial sites for the MPS I enzyme replacement therapy trial that resulted in the licensing and approval of Laronidase for the treatment of MPS I. In 2015, the first human clinical trial of a new gene therapy for Huntington’s started at UBC; this is expected to be the first successful genetic therapy for Huntington’s. These important advancements and initiatives were only possible because talented, dedicated scientists had the skills and resources they needed to pursue new knowledge disease research.
Your gift will support pilot studies of rare diseases and help ensure that promising early-stage research with the potential to inform diagnosis or treatment of rare disease proceeds without delay.