Laboratory Implications for Hemophilia Gene Therapy and Other Novel Hemophilia Treatments

Course overview

After several decades of basic science and pre-clinical investigation hemophilia gene therapy has entered the clinic. Several Phase 3 studies have now been completed, and three hemophilia gene therapy products have received regulatory approvals. As these products begin to appear in regular clinical practice, it is important to appreciate the range of laboratory studies that have contributed to the advancement of this treatment paradigm. In this presentation, Dr. David Lillicrap, MD, FRCPC, Professor, Department of Pathology and Molecular Medicine, Queen's University, will discuss details of laboratory studies employed in the development of gene therapy programs and the requirements of laboratory monitoring in patients who have undergone gene therapy treatment. In addition to 'routine' substitution gene therapy, Dr. Lillicrap will also highlight challenges for the laboratory in the evaluation of other novel hemophilia treatments that are in early phases of development.