Saswati Chatterjee, Ph.D., Co-Founder and Chair of the Scientific Advisory Board
Saswati Chatterjee, Ph.D., is a Professor of Virology at the Beckman Research Institute at the City of Hope in California, where she directs the adeno-associated virus (AAV) gene therapy group. Her research focuses on developing gene-based therapeutic strategies for genetic diseases, stem cells and AAV biology. Her group pioneered AAV-based gene transfer into human hematopoietic stem cells and subsequently identified and isolated a series of naturally-occurring AAVs from human CD34+ cells. Dr. Chatterjee is a member of the Recombinant DNA Advisory Committee (RAC) to the Office of the Director, National Institutes of Health and is a member the Biosafety Working Group of Office of Biotechnology Activity, NIH. Dr. Chatterjee served as a charter member of the Therapeutic Approaches to Genetic Diseases Study Section of the NIH. She has also served on the Gene Therapy & Inborn Errors, Gene and Drug Delivery and Medical Biochemistry study sections, as well as numerous special review panels and special emphasis committees of the NIH. She serves on various committees at the California Institute of Technology and is a member of the editorial boards of Human Gene Therapy and Molecular Cellular Therapies. Dr. Chatterjee's current research on genetic therapeutic strategies represents the culmination of her long-standing interests in gene therapies, molecular virology and transplantation biology. She has co-authored numerous publications, been awarded several R01 and program project grants from the NIH and holds multiple patents. Dr. Chatterjee received her Ph.D. from McGill University, Montreal, Canada. She pursued her postdoctoral training in the Transplantation Biology Section of the Immunology Branch of the NCI, NIH. This led to her work on molecular virology of AAV in the Laboratory of Biology of Viruses and in the Laboratory of Viral Diseases, NIAID, NIH.
Morton J. Cowan, M.D.
Morton J. Cowan, M.D. is a pediatric immunologist, professor emeritus of pediatrics and member of the Allergy, Immunology and Blood and Marrow Transplant Division in the Department of Pediatrics at UCSF Benioff Children's Hospital. His career has focused on children with primary immunodeficiency disease (PIDD) and Dr. Cowan has authored or co-authored over 200 peer-reviewed publications on this subject. He is principal investigator, with UCSF as the lead institution, for the NIH-funded Primary Immune Deficiency Treatment Consortium (PIDTC), a rare disease network that joins 41 pediatric centers in North America to study the natural history and define optimal treatments for children with PIDD. Dr. Cowan serves on the steering committee of the Pediatric Blood and Marrow Transplant Consortium (PBMTC), which represents over 70 pediatric transplant centers in North America and on the PBMTC Executive Committee. He founded and was the initial chair of the pediatric special interest group within the American Society of Blood and Marrow Transplantation (ASBMT). Dr. Cowan performed one of the first T cell depleted mismatched bone marrow stem cell transplants in North America in 1982, reported one of the first in utero transplants in the world, the first patient with multiple biotin-dependent carboxylase deficiencies and immune deficiency, one of the first children with ZAP70 deficient SCID, and the first children with HIV infection. He also identified the mutation that causes SCID in Athabascan-speaking native Americans and is currently developing gene therapy for this disorder. His other major research interest is in identifying non-chemotherapeutic approaches to opening marrow niches in children with non-malignant diseases prior to bone marrow transplantation and gene therapy. Dr. Cowan holds a B.S. from MIT and an M.D. from University of Pennsylvania.
Stephen J. Elledge, Ph.D.
Stephen J. Elledge, Ph.D., is a Professor of Genetics at Harvard Medical School. Dr. Elledge's research has uncovered important clues about key biological pathways driving the cell cycle and how cells sense and respond to DNA damage. His research has also contributed to advances in scientific disciplines by developing new cloning methods, novel approaches to build cDNA libraries that could be expressed in yeast. Dr. Elledge has made instrumental discoveries isolating and identifying genes that play key roles in the development of various cancers, including p21 and p57. He and his colleagues' research also led to important discoveries about how cells detect and repair DNA damage, uncovering a whole signal transduction mechanism that alerts cells to chromosome defects. He recently identified the Chk2 enzyme, which activates the tumor-suppressor p53 to prevent cells with damaged DNA from dividing. When this enzyme is missing or defective, the "brakes" on cell division are released, increasing the risk of cancer. Dr. Elledge holds a B.S. in Chemistry from the University of Illinois and a Ph.D. in Biology from the Massachusetts Institute of Technology (MIT).
Mason Freeman, M.D.
Mason Freeman, M.D. is a Professor of Medicine at Harvard Medical School and a Venture Partner at 5AM Ventures. He also serves as Chief of the Lipid Metabolism Unit and Director of the Massachusetts General Hospital's (MGH) Translational Research Center. Trained in internal medicine and endocrinology, Dr. Freeman has spent 25 years studying the trafficking of lipids in and out of cells via receptors and transmembrane transporters. Previously, he led the Novartis Translational Medicine Program for Cardiovascular & Metabolic Diseases and was Global Head of Biomarker Development. At Novartis and 5AM, he contributed to the development of ENTRESTO for heart failure and VELTASSA for hyperkalemia. Dr. Freeman currently is a Director of Crinetics Pharmaceuticals and previously served as Clinical Advisor to Relypsa and a Director for Envoy (acquired by Takeda). He holds a B.A. from Harvard College, an M.D. from the University of California, San Francisco and completed a postdoctoral research fellowship in the Biology department at MIT and the Endocrine Department at MGH.
Hans-Peter Kiem, M.D., Ph.D.
Hans-Peter Kiem, M.D., Ph.D. is currently the Fred Hutch Endowed Chair for Cell and Gene Therapy, Associate Head, Program in Transplantation Biology, Member, Clinical Research Division, Fred Hutchinson, Professor of Medicine / Adjunct Professor of Pathology University of Washington School of Medicine and Associate Head, Heme Malignancy Program, UW / Fred Hutchinson Cancer Consortium.
Dr. Kiem has an extensive background working in pre-clinical and clinical studies involving stem cell biology, including induced pluripotent stem cells, hematopoietic stem cell transplantation and gene therapy. His research focus has been the study of stem cell biology and cell engineering with applications involving genetic and infectious diseases and cancer. Dr. Kiem is the sponsor of four clinical stem cell gene therapy studies (HIV, glioblastoma, and Fanconi anemia) and is the PI or Co-PI of many R01 or P01 grants, including a Martin Delaney Consortium grant to study HIV cure strategies (defeatHIV). He also serves as the Chair of the NIH Recombinant DNA Advisory Committee (RAC), the American Society for Gene and Cell Therapy (ASGCT) Stem Cell Committee, and the ASH (Amercian Society of Hematology) Stem Cell and Regenerative Medicine Committee. Dr. Kiem holds an M.D. and Ph.D. from the University of Ulm in Germany. As a research fellow at Stanford University, he studied molecular abnormalities and minimal residual in patients with lymphoma and the use of a novel immunodeficient mouse model for lymphoma. Dr. Kiem completed his residency in Internal Medicine and his Physician/Scientist training at Vanderbilt University. He then joined the Fred Hutchinson Cancer Research Center, where he completed his clinical fellowship in Oncology.