Genome Engineering Technologies & the Development of Cell Therapies
Genome engineering technologies represent powerful tools in the generation of safe and
efficacious cell therapy drug products. The development of CRISPR-Cas systems for cell
engineering has provided flexible approaches to expand this toolbox. We will discuss the
application of CRISPR-Cas genome engineering to cell therapy drug development with a particular focus on the development of allogeneic T cell therapies and perspectives beyond primary immune cell engineering.
Attend this workshop to:
- Hear about how CRISPR-Cas systems have been harnessed and applied to cell therapy drug development
- Discuss the importance of specificity for in-cell editing when applying genome engineering technologies to cell therapy drug development
- Learn about strategies to increase the persistence of allogeneic cell therapies using genome engineering technologies
- Consider the challenges in the manufacture of an engineered cell therapy drug product
Understanding the Therapeutic Potential of Mesenchymal Stem Cells
Mesenchymal stem cells (MSCs) are multipotent adult stem cells that are capable of self-renewal and differentiation into cells of mesodermal lineage (e.g. bone, fat, and cartilage). MSCs have been isolated from numerous tissue sources, including bone marrow, adipose tissue, umbilical cord, placenta and dental pulp. MSCs produce factors that are proangiogenic, anti apoptotic, inhibit inflammation, promote wound remodeling. A primary therapeutic mechanism of MSCs is immunomodulation via paracrine-mediated signaling. MSCs appear to have an inherent low immunogenicity, due to the lack of or reduced expression of MHC Class II antigens. They also express low levels of major histocompatibility complex (MHC) class I, lack MHC class II and co-stimulatory molecules. These properties make MSCs viable candidates for allogeneic transplantation, which is often referred to as the “universal donor” scheme. This workshop will focus on the current state-of-the-art regarding allogeneic transplantation of MSCs.
Attend this workshop to Learn:
- The biology of and therapeutic potential of mesenchymal stem cells
- The potential clinical application of allogeneic mesenchymal stem cells
- Clinical scale production of allogenic mesenchymal stem cells
Professor & Director
Tulane University of Louisian
Improving Cytotoxicity of Allogeneic Peripheral Blood NK Cells Using Genetic Engineering
Peripheral Blood NK cells have therapeutic potential for a wide variety of human malignancies and are a very attractive candidate as an allogeneic cell therapy. We have been able to genetically modify PB-NK cells using DNA-free Cas9/RNP technology. We have had success combining this method with AAV6 vectors for site-directed gene insertion into PB-NK cells. Furthermore, to better study the effect of allogeneic NK cell transplantation in a spontaneous cancer model homologous to humans, we have been able to expand Canine NK cells and have initiated a clinical trial using ex-vivo expanded TGFB-imprinted NK cells to treat dogs with Osteosarcoma. This workshop will discuss novel genetic modification techniques and outline the benefits of canine models for NK cell therapies.
Attend this workshop to:
- Discuss novel genetic modification techniques for peripheral blood NK cells including Cas9/RNP and AAV to generate CARs with novel NK cell signaling domains
- Outline improved cytotoxic effects of NK cells by overcoming their resistance to tumor microenvironment and NK cell fratricide in monoclonal therapies for Multiple Myeloma.
Nationwide Children's Hospital
Developing Allogeneic Cell Therapies: Approaches, Tools, & Technologies
Cellular therapies have taken prominent position as a viable approach for effectively treating conditions that have long challenged conventional therapies. Whether in regenerative medicine or immuno oncology applications, cells have become “living drugs”. In the past few years, we have witnessed unprecedented results ranging from correction of genetically inherited disorders in patients’ own stem cells, to transplantation of in vitro generated organs, to cures of metastatic, terminal cancers by engineered immune cells. Autologous therapies were the first to lead us into groundbreaking results, but their limitations have prompted innovative developments using allogeneic sources. Today, the concept of a “universal cell therapy” for a given indication is no longer seen as unattainable, but instead as a reality we may soon experience. Technology has, likewise, advanced to meet the demands of this rapidly growing field, and a variety of new tools
have entered the cell therapy market.
Attend this workshop to:
- Discuss potential cell sources for allogeneic therapies, focusing on their applicability, advantages, and potential shortcomings;
- Review and discuss the current technologies often used in the field, and novel tools that may support process development, product manufacturing and characterization;
- Engage in roundtable discussions aimed at encouraging networking through the exchange of knowledge and experience.
Senior Research Scientist, Immunotherapy
MD Anderson Center