SOTA: Cell and Gene Therapy: Opportunities in the Translation from Humans to Horses, and Back Again

Presentation Description / Summary: The use of cells for the treatment of injury and disease has attained credibility in both human and equine medicine, although the range and severity of diseases treated appears to be greater in the former than the latter. Hematopoietic stem cell transplantation is a universally recognized and accepted form of therapy for haematological malignancies and other haematological disorders in humans, and an increasing number of cell therapy products have received approval for marketing in several jurisdictions. Cell therapy at present appears to be limited to the musculoskeletal system in horses. With regard to gene therapy in humans, an increasing number of applications are being approved for routine use. In addition, gene modification of somatic cells such as lymphocytes has dramatically changed the face of approved therapies for haematological diseases (leukaemia, lymphoma, myeloma) – so called chimeric antigen receptor (CAR) T-cells. Gene therapy in horses appears to be limited at present to musculoskeletal conditions, although innovative opportunities lie in the cardiovascular, pulmonary and central nervous systems.

Learner Outcomes: 1. Similarities and differences in human and equine physiology and disease pathogenesis relevant to CGTs will be highlighted; 2. Advantages and limitations in the use of horses as a robust pre-clinical modal for novel CGTs in humans, and vice-versa will be discussed; and 3. In moving beyond the need to improve quality of life, practical considerations relevant to human and equine markets including funding of both research and routine practice will be addressed.