The promise of gene therapy is a single treatment for people with hemophilia (PWH) will be covered by the WFH Virtual Summit session, “Medical Plenary: Gene Therapy”, taking place at 9am EDT on Monday, June 15. The session will be moderated by Glenn Pierce, MD, PhD, WFH Vice-President, Medical, with talks by Luigi Naldini and Steven Pipe.
Delivering on the promise of gene therapy will require broad-based multi-stakeholder preparation with a focused emphasis on education, approval of safe and effective therapies, removal of barriers to access and excellence in clinical delivery. New strategies to overcome the major hurdles to safe and effective gene transfer—and translate them into new therapeutic strategies for genetic disease and cancer—are currently being investigated. This has generated new insights into hematopoietic stem cell function, and the induction of immunological tolerance and tumour angiogenesis. Information regarding the further development of lentiviral vectors as a systemic gene delivery vehicle to complement the reach of AAV vectors will also be presented at the session.
To find out more about this session, have a look at the WFH Virtual Summit program.