SC1: Gene Edited Cell-Based Therapies: Opportunities and Challenges

MONDAY, SEPTEMBER 23 | 12:30 – 3:00 PM

Instructors:

Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital

H. Trent Spencer, PhD, Associate Professor of Pediatrics and Director, Gene and Cell Therapy Program in the Aflac Cancer and Blood Disorders Center, Emory University School of Medicine; President and Co-founder, Expression Therapeutics LLC

Engineered cell-based therapies are yielding promising clinical benefits for treating many diseases, especially cancer. This course will cover some of the important topics such as sources of therapeutic cells (stem cells, T cells, NK cells and cancer cells), novel engineered viral vectors and their engineering capabilities, the role of CRISPR gene editing and clinical translation and safety aspects of engineered cell therapies. The instructors will give short talks followed by open discussion with attendees, where they can elaborate on their experiences and expertise.

What you will learn:

Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital

• Sources of therapeutic cells (Stem cells, T cells, NK cells and Cancer cells)
• Novel engineered viral vectors and their engineering capabilities
• Role of CRISPR gene editing in creating therapeutic cells
• Applications of gene edited cells in pre-clinical models
• Clinical translation and safety aspects of engineered cell therapies

H. Trent Spencer, PhD, Associate Professor of Pediatrics and Director, Gene and Cell Therapy Program in the Aflac Cancer and Blood Disorders Center, Emory University School of Medicine; President and Co-founder, Expression Therapeutics LLC

• Methods to expand γδ T cells in serum free media
• Transduction strategies using both AAV and lentiviral vectors
• Optimization of transduction timing and growth
• Decorating γδ T cells with chimeric antigen receptors and non-activating CARs
• Strategies to combine chemotherapy with non-engineered and genetically engineered γδ T cells to treat leukemias and solid tumors
• Avenues of study for enhancing the use genetically engineered γδ T cells
• Challenges in production

Additional Instructors to be Announced

Meet the Instructors:

Khalid Shah, MS, PhD, Director, Center for Stem Cell Therapies and Imaging, Harvard Medical School; Vice Chair of Research, Brigham and Women’s Hospital

Khalid Shah, MS, PhD is the Director of the Center for Stem Cell Therapeutics and Imaging and the Center of Excellence in Biomedicine at Brigham and Women’s Hospital (BWH). He is also the Vice Chair of Research for the Department of Neurosurgery at BWH and a Principal Faculty at Harvard Stem Cell Institute in Boston. Dr. Shah and his team have pioneered major developments in the cell therapy field, successfully developing experimental models to understand basic cancer biology and therapeutic cells for cancer. Recently, Dr. Shah’s laboratory has reverse engineered cancer cells using CRISPR/Cas9 technology and utilized them as therapeutics to treat cancer. He has founded 2 biotech companies, AMASA Therapeutics and ALIM Therapeutics, whose main objective is the clinical translation of therapeutic stem cells in cancer patients.

H. Trent Spencer, PhD, Associate Professor of Pediatrics and Director, Gene and Cell Therapy Program in the Aflac Cancer and Blood Disorders Center, Emory University School of Medicine; President and Co-founder, Expression Therapeutics LLC

Dr. Spencer is a Professor in the Department of Pediatrics, he holds the Jean Amos Cell and Gene Therapy Chair, he is the Director of the Cell and Gene Therapy Program, Emory University School of Medicine, and Director of the Center for Pediatric Cellular Therapeutics, Children’s Hospital of Atlanta. He is also the President and Co-founder of Expression Therapeutics, LLC, which is developing AAV and lentiviral-based gene and cell therapies for inherited diseases. He has headed a well-funded gene therapy laboratory for over 20 years. His research interests continue to be the development of gene therapy applications for the treatment of childhood cancer and inherited diseases.