Although gene editing technologies such as CRISPR/Cas9 and TALENs are still new, they are widely being used to develop novel cellular therapies. To create safe allogeneic CAR T cells for the treatment of blood cancers and solid tumors, gene editing is used to modify the expression of key proteins within the donor T cells, creating highly specific genetic modifications for the prevention of graft T cells attacking host antigens, the prevention of host T cells attacking the graft, and the prevention of the graft T cells becoming functionally exhausted during treatment. Current and future cellular therapies utilizing gene editing technologies will touch the personal and professional lives of so many in the AABB community, therefore it is important we have a better understanding of what these technologies are, how they are used to create safe cellular therapies, and their specific therapeutic applications.
There are 3 main objectives of this session, first to educate attendees on basic scientific background principles of editing technologies to modify the genome. Dr. Dana Foss, Postdoctoral Fellow at the University of California, Berkley has agreed to talk on the scientific background of gene editing. Dr. Foss is a trainee of Dr. Ross Wilson, who trained under Dr. Jennifer Doudna, a leading expert in gene editing. Dr. Foss’ work focuses on tissue-specific delivery of gene editing enzymes and she was the primary contributor to a very through and informative review article recently published in Transfusion on clinical applications of CRISPR-based genome editing. Second, attendees will learn about the research-in-progress of specific technical applications of gene editing. Invited to talk about these applications is Dr. Toni Cathomen, Professor and Director, Institute for Transfusion Medicine and Gene Therapy at the University Medical Center in Freiburg, Germany. Dr. Cathomen has recently published a review article in Nature Medicine on the state of research toward employing gene editing technologies in hematopoietic stem cells and CAR T cells. Finally, attendees of this session will be introduced to specific clinical applications of gene editing technologies for cancer treatments. Cellectis, a clinical-stage biopharmaceutical company in Paris, France has pioneered the use of TALEN-edited T cells for creation of allogenic CAR T cell treatments for B cell malignancies. Dr. David Sourdive, Executive Vice President at Cellectis has agreed to speak about the company's work creating "off-the-shelf" therapies using gene editing technologies. The overall goal of this session is to provide basic education on genome editing and show the practical applications of cutting-edge editing technologies to the development of cellular therapies.
This session will be jointly chaired with Dr. Richard Schaefer of the AABB Board of Directors and Johann Wolfgang Goethe University in Frankfurt, Germany.
Explain to others how gene editing technologies work.
Recognize how gene editing technologies can be applied to the development of cellular therapies.
Describe specific examples of how gene editing technologies were used to create cell therapies being used to treat patients.
Please note: Dana Foss, PhD and David Sourdive, PhD did not consent to be recorded; therefore, the on-demand presentation and handout have been modified accordingly.
Science Officer for Cellular Therapies,
Oklahoma Blood Institute