CDCM LEADERSHIP
Matthew Porteus, MD, PhD
Director, Center for Definitive and Curative Medicine
A pediatric hematologist/oncologist by training, Dr Porteus brings international leadership in basic and translational applications of genome editing. He has led the development of the field of genome editing and established the key approaches to using the CRISPR/Cas9 system in clinically relevant human somatic stem cells. The goal of his research is to develop safe and effective therapy for patients with monogenic diseases such as β-thalassemia, sickle cell disease, hemophilia, and severe combined immunodeficiency and for infectious diseases such as HIV using homologous recombination mediated genome editing. This platform can then be developed to treat genetic diseases of other organ systems and to enhance the potency and safety of cell based regenerative medicines.
Maria Grazia Roncarolo, MD
Co-Director, Center for Definitive and Curative Medicine
Maria Grazia Roncarolo, MD is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, and co-director of the Institute for Stem Cell Biology and Regenerative Medicine.
Dr. Roncarolo leads efforts to translate scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including treatments based on stem cells and gene therapy.
A pediatric immunologist by training, she earned her medical degree at the University of Turin, Italy. She spent her early career in Lyon, France, where she focused on severe inherited metabolic and immune diseases, including severe combined immunodeficiency (SCID), better known as the "bubble boy disease." Dr. Roncarolo was a key member of the team that carried out the first stem cell transplants given before birth to treat these genetic diseases.
Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine to cure patients with currently incurable diseases through the development of innovative stem cell-and gene-based therapies.
Anthony E. Oro, MD, PhD
Co-Director, Center for Definitive and Curative Medicine
A developmental biologist and practicing dermatologist, Dr. Oro brings expertise in basic and translational mechanisms of epithelial regeneration and carcinogenesis. He helped make the connection between the hedgehog developmental pathways and human cancer, was a principal investigator for the first hedgehog pathway inhibitor for skin cancer, and has identified and developed lead compounds for novel drug resistance pathways. A discoverer of nuclear receptors like retinoic acid receptor, Dr. Oro studies chromatin dynamic changes in the developing genome that have led to the use of iPSCs for clinical manufacturing of corrected skin grafts for Epidermolysis bullosa.
J. Fraser Wright, PhD
Director of Technology Innovation for CDCM and Research Professor of Pediatrics
Dr. Wright is an internationally recognized leader in the field of gene therapy, he has contributed to the development of several gene therapy products, including Kymriah (CAR-T immunotherapy) and Luxturna, the first therapy for a genetic blindness (RPE65 deficiency), recently approved in the United States.
Dr. Wright joins Stanford University as Professor of Pediatrics at the CDCM. His research program aims to address key immunological barriers to gene therapy through innovative approaches to viral vector design and generation, and to develop vectorized antibodies for serious human diseases. Dr. Wright will contribute expertise in vectorology, translational research, and clinical manufacturing and quality systems towards successful development of transformative stem cell and gene therapies discovered at the CDCM and the Institute for Stem Cell Biology and Regenerative Medicine.