Blair K. Gage (PhD) is a Scientist at the Ottawa Hospital Research Institute in the Regenerative Medicine Program and an Assistant Professor in the Department of Cellular and Molecular Medicine at the University of Ottawa. After a BSc in Biotechnology from UBC and BCIT, he completed a PhD in Cell and Developmental Biology exploring how transcription factors regulated pancreatic endocrine subtype specification from human pluripotent stem cells. His postdoctoral work shifted to generate organ-specific endothelial cells of the liver from human pluripotent stem cells to create potential cell-based and cell-informed therapeutics for liver disease and Hemophilia A. Dr. Gage started his lab in December 2023 and focuses on understanding how endothelial cells gain and maintain organ specific functions to build new therapies for human liver diseases where these functions are lost. The Gage lab’s primary research model is human pluripotent stem cells which are differentiated to become endothelial cells that can be transplanted in mice where they durably engraft and become functional. This research model and approach has potential therapeutic value as delivery of stem cell-derived endothelial cells to mouse models of Hemophilia A resulted in correction of the severe bleeding disorder by sustained production of bioactive coagulation factors. Building on this cell-based therapy leverages many computational, genetic, and molecular approaches to identify and apply new therapies aimed at controlling endothelial function to fight disease.

Professor Edward (Ted) Tuddenham's journey into haemophilia began in 1969 at the Royal Victoria Infirmary in Liverpool, where he treated haemophilic patients. His interest deepened in Cardiff under Arthur Bloom, where he investigated the relationship between Von Willebrand Disease and Haemophilia A. Tuddenham dedicated seven years to purifying factor VIII, first at the University of Connecticut and then at the Royal Free Hospital Haemophilia Centre. His work led to the complete purification of factor VIII, enabling its amino acid sequencing and the cloning of its gene. These breakthroughs facilitated accurate carrier testing, antenatal diagnosis, and the discovery of mutations causing haemophilia A.

In 1987, Tuddenham established the Haemostasis Research Group for the Medical Research Council, focusing on rare bleeding disorders. In 2006, he returned to the Royal Free Hospital to pursue gene therapy for haemophilia. His team demonstrated the safety and efficacy of gene therapy for haemophilia B, using a vector that allowed high levels of factor IX expression. This resulted in most patients discontinuing replacement therapy and remaining free of spontaneous bleeding for up to 13 years. This vector was later modified by Uniqure, leading to the marketing approval of Hemgenix in 2023.

Collaborating with Amit Nathwani's laboratory, Tuddenham also helped develop a vector for transferring the factor VIII gene to treat Haemophilia A, culminating in market approval in the EU in 2022. He continues his work on gene therapy trials for haemophilia and other rare bleeding disorders, with 350 publications, an H-index of 74, and over 21,000 citations.