Speakers

Christopher P. Austin, M.D., is a CEO-Partner at Flagship Pioneering in Cambridge, Massachusetts, where he serves as CEO of one of Flagship’s franchise companies and advises on the operation and creation of other Flagship entities. Before joining Flagship in 2021, he served for almost a decade as the founding director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH), where he formulated the strategic vision and scientific directions of the new center and led its efforts in developing, demonstrating, and disseminating scientific and operational advances across the spectrum of translational science to get more treatments to more patients more quickly, from target validation to preclinical therapeutic development to clinical trials to community health implementation. Before NCATS, Dr. Austin founded and directed a number of scientific and technology initiatives at the National Human Genome Research Institute (NHGRI) at the NIH to derive biological insights and therapeutic potential from the human genome. He came to the NIH in 2002 from Merck, where his work focused on genome-based discovery of novel targets and drugs, with a particular focus on common complex neuropsychiatric diseases. He received his A.B. in biology from Princeton University and his M.D. from Harvard Medical School and completed clinical training in internal medicine and neurology at Massachusetts General Hospital and a research fellowship in genetics at Harvard. 

Diana W. Bianchi, M.D., is the director of the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) and head of the Prenatal Genomics and Therapy Section for the Medical Genetics Branch at the National Human Genome Research Institute (NHGRI). She oversees the research on pediatric health and development, maternal health, reproductive health, intellectual and developmental disabilities, and rehabilitation medicine, among other areas. These efforts include managing a staff of approximately 1,400 people and an annual budget of approximately $1.3 billion. She also serves as an ambassador and spokesperson for NICHD. She is a practicing medical geneticist with special expertise in reproductive genetics. Dr. Bianchi's translational research focuses on two broad themes: prenatal genomics with the goal of advancing noninvasive prenatal DNA screening and diagnosing and investigating the fetal transcriptome to develop new therapies for genetic disorders that can be given prenatally. She is editor-in-chief of the international journal Prenatal Diagnosis and is a past president of the International Society for Prenatal Diagnosis and the Perinatal Research Society. Previous positions include founding executive director of the Mother Infant Research Institute at Tufts Medical Center; the Natalie V. Zucker Professor of Pediatrics, Obstetrics, and Gynecology at Tufts University School of Medicine; the vice chair for Pediatric Research at the Floating Hospital for Children, Boston; and a member of the National Advisory Council of NICHD. Dr. Bianchi has published more than 300 peer-reviewed articles and is 1 of 4 authors of Fetology: Diagnosis and Management of the Fetal Patient. She received her B.A. in biology magna cum laude from the University of Pennsylvania and her M.D. from Stanford University School of Medicine. She completed her residency training in pediatrics at the Children's Hospital, Boston, and her postdoctoral fellowship training in medical genetics and neonatal-perinatal medicine at Harvard.

R. Alta Charo, J.D., is the Knowles Professor Emerita of Law and Bioethics at the University of Wisconsin; the David A. Hamburg Inaugural Fellow at the Nuclear Threat Initiative, working in its global biosecurity program; and the lead co-chair of the new 4S (safety, security, sustainability, and social responsibility) Unit for BioMADE. She is an elected member of the American Academy of Arts and Sciences; the National Academy of Medicine (NAM), and the American Association for the Advancement of Science (AAAS). At present Ms. Charo is concluding service on the World Health Organization (WHO) expert advisory committee on human genome editing. She was a member of President Clinton’s National Bioethics Advisory Commission, served as a senior policy advisor at the Food and Drug Administration (FDA), and co-chaired the National Academy of Science (NAS)/NAM committees on embryonic stem cell research and governance of human genome editing. She received her B.A. in biology from Harvard University and her J.D. from Columbia University.

Fitz Kettler, not yet 2 years old, is already a science trailblazer. Born from in vitro fertilization (IVF) using a donor egg, his newborn screening test revealed he had severe combined immunodeficiency (SCID.) With 20 different kinds of SCID, doctors needed to know precisely which type of immune deficiency Fitz had. His blood sample was sent to Rady Children’s Institute of Genomic Medicine for rapid whole genome sequencing. Within 92 hours, the test scanned all 3 billion pairs of genes allowing genome analysts to find a mutation in his genetic code. Fitz was diagnosed with one of the rarest immune deficiencies, Artemis SCID. It meant he had no immune system and first-time parents Christina and Daniel Kettler were told he would not live to be a year old. Fitz was accepted into a gene therapy clinical trial at the University of California, San Francisco (UCSF). At 2 months old, he had his bone marrow harvested followed by chemo and then an experimental gene therapy to replace his defective cells. The lab-made cells began providing instructions so his body could develop a healthy immune system. At 5 months old, Fitz was discharged to live in home isolation as his T and B cells continued to develop and function. Now, ready to celebrate his second birthday, Fitz is living life as a normal toddler and getting ready to begin receiving vaccinations. 

Walter J. Koroshetz, M.D., is the director of the National Institute of Neurological Disorders and Stroke (NINDS). He joined NINDS in 2007 as the deputy director and also served as acting director. When he was the deputy director under Dr. Story Landis, they directed program planning and budgeting and oversaw the scientific and administrative functions of the Institute. He has held leadership roles in a number of National Institutes of Health (NIH) and NINDS programs including the NIH’s Brain Research Through Advancing Innovative Neurotechnologies® (BRAIN) Initiative, the Traumatic Brain Injury Center collaborative effort between the NIH intramural program and the Uniformed Health Services University, and the multi-year work to develop and establish the NIH Office of Emergency Care Research to coordinate NIH emergency care research and research training. Previously he was the vice chair of neurology and director of stroke and neurointensive care at the Massachusetts General Hospital. He received his M.D. from the University of Chicago Pritzker School of Medicine.

David R. Liu, Ph.D., is the Richard Merkin Professor and director of the Merkin Institute of Transformative  Technologies in Healthcare, vice chair of the faculty at the Broad Institute of Harvard and Massachusetts of Technology (MIT), the Thomas Dudley Cabot Professor of the Natural Sciences at Harvard University, and a Howard Hughes Medical Institute (HHMI) investigator. His research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. His major research interests include the engineering, evolution, and in vivo delivery of genome-editing proteins such as base editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries. Base editing—the first general method to perform precision gene editing without double-stranded breaks and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of laboratories around the world and have enabled the study and potential treatment of many genetic diseases. Dr. Liu has been elected to the U.S. National Academy of Sciences, the U.S. National Academy of Medicine, and the American Association for the Advancement of Science. He has earned several university-wide distinctions for teaching at Harvard, including the Joseph R. Levenson Memorial Teaching Prize, the Roslyn Abramson Award, and a Harvard College Professorship. He has published more than 200 papers and is the inventor on more than 75 issued U.S. patents. His research accomplishments have earned distinctions including the Ronald Breslow Award for Biomimetic Chemistry; the American Chemical Society (ACS) David Perlman Award; the ACS Chemical Biology Award; the ACS Pure Chemistry Award; the Arthur Cope Young Scholar Award; the National institutes of Health (NIH) Marshall Nirenberg Lecturer; and awards from the Sloan Foundation, Beckman Foundation, National Science Foundation (NSF) CAREER Program, and Searle Scholars Program. In 2016 and 2020 he was named one of the Top 20 Translational Researchers in the world by Nature Biotechnology and was named one of Nature’s 10 researchers in world and to the Foreign Policy Leading Global Thinkers in 2017. He is the founder or co-founder of several biotechnology and therapeutics companies, including Beam Therapeutics, Prime Medicine, Editas Medicine, Pairwise Plants, Exo Therapeutics, and Chroma Medicine. He graduated first in his class at Harvard College and received his Ph.D. from the University of California, Berkeley.

Joni L. Rutter, Ph.D., is the acting director of the National Institutes of Health’s (NIH’s) National Center for Advancing Translational Sciences (NCATS). She is responsible for planning and executing the Center’s complex and multifaceted preclinical and clinical programs. Before joining NCATS, she served as the director of Scientific Programs in the All of Us Research Program of the NIH’s Precision Medicine Initiative and, prior to that, as the director of the Division of Neuroscience and Behavior at the National Institute on Drug Abuse (NIDA). Dr. Rutter is recognized for her work in basic and clinical research in human genetics and in the study of genetic and environmental risk factors focusing on the fields of cancer and addiction. Her primary scientific objective is in applying translational science approaches to basic discoveries and getting more treatments to more people more efficiently. She received her Ph.D. in pharmacology and toxicology from Dartmouth Medical School and completed a fellowship at the National Cancer Institute.

Teresa Sparks, M.D., is an assistant professor of maternal-fetal medicine, a clinical geneticist, and a women’s reproductive health research scholar at the University of California, San Francisco (UCSF). Clinically, she provides care for women whose pregnancies are complicated by fetal anomalies, maternal and fetal genetic diseases, and other pregnancy complications. Dr. Sparks oversees the Hydrops: Diagnosing and Redefining Outcomes with Precision Studies (HyDROPS) research program at UCSF, where her research is focused on understanding the fetal genetic diseases underlying non-immune hydrops fetalis and the fetal phenotypes of genetic diseases as well as on applying novel approaches to treating and managing fetal disease during pregnancy. She received her M.D. from UCSF, completed her residency in obstetrics and gynecology at the Brigham and Women's Hospital/Massachusetts General Hospital combined program, and completed a combined fellowship in maternal-fetal medicine and clinical genetics at UCSF. 

Julia Vitarello is the founder and chief executive officer of Mila’s Miracle Foundation (MMF). She founded MMF in December 2016 upon learning that her seemingly healthy 6-year-old daughter, Mila, had Batten disease, a fatal genetic condition with no cure. In an unprecedented race against time to save her daughter, Julia’s collaboration with Dr. Timothy Yu from Boston Children’s Hospital led to the first ever drug tailored to just one person, affectionately named milasen. After Mila showed great promise in the first year of treatment, her disease slowly continued to progress. In March 2021 Mila’s spirit left her body. Driven by a sense of hope and responsibility, Julia is on a mission to turn the groundbreaking work that went into milasen into a promising platform solution to the drastic problem of rare disease in children. In her quest to open the field of individualized medicines for rare disease that Mila pioneered, Julia has created a strong global following of Mila’s story. She regularly engages with scientists, academic and government institutions, biotechs, and patient advocates and presents at scientific meetings and conferences across the country. Through MMF, Julia initiated the work toward a recently launched novel gene replacement therapy trial targeting Mila’s variant of Batten (CLN7), launched a Batten and Neurodegenerative Disease Clinic at Children’s Hospital Colorado, funded basic science, and hosted scientific meetings.

National Center for Advancing Translational Sciences