CDCM LEADERSHIP

Maria Grazia Roncarolo, MD

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, director of the Center for Definitive and Curative 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.

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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.

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Matthew Porteus, MD, PhD

Co-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.

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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.


Neehar Bhatia, PhD

Director of Translational Research for CDCM

Neehar Bhatia, PhD, will lead cell and gene therapy projects for discovery, development and clinical manufacturing in the CDCM pipeline. In addition, with her extended experience, she will also direct development of induced pluripotent stem cells (iPSC) platform technology and regenerative medicine projects for CDCM. Neehar has extensive experience in early innovation and technology transfer, development of cGMP compliant manufacturing processes and analytical assays, and clinical manufacturing of cell and gene therapy products.

She joined the LCGM in May 2017 as Associate Director, Process Development and Manufacturing and was promoted to Director a year later. After receiving her PhD in Biochemistry from Lucknow University, Neehar spent several years building her expertise as a scientist and specialist in manufacturing process development and cGMP manufacturing, within both academia and the biotechnology sector. She holds exemplary skills in clinical manufacturing team leadership, cell and gene therapy program management, as well as cGMP facilities and compliance.