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Participant Bios

Contributors

Robert J. Beall, PhD

Former President and CEO of the Cystic Fibrosis Foundation. 

Dr. Beall began his tenure at the CF Foundation in 1980 as Executive Vice President for Medical Affairs and served as President and Chief Executive Officer from 1994 to 2015. Prior to joining the CF Foundation, Dr. Beall was on the medical school faculty of Case Western Reserve University in Cleveland, and worked at the National Institutes of Health, where he managed a large portion of NIH’s cystic fibrosis program. During Dr. Beall’s tenure, the CF Foundation became one of the most respected voluntary health organizations in the country. The CF Foundation’s venture philanthropy model, pioneered by Dr. Beall, has raised and invested hundreds of millions of dollars to help develop nearly every CF drug available today including Kalydeco and Orkambi, the first two drugs to treat the underlying cause of the disease. This strategy has led to tremendous advances in life expectancy, which has doubled in the last 30 years. 

Jean Bennett, MD, PhD

Jean Bennett, MD, PhD, the F.M. Kirby Professor of Ophthalmology, Professor of Cell and Developmental Biology, and Director of the Center for Advanced Retinal and Ocular Therapeutics (CAROT) at the University of Pennsylvania Perelman School of Medicine, is a pioneer in retinal gene therapy. She has developed gene transfer approaches to test treatment strategies for retinal degenerative and ocular neovascular diseases, to elucidate retinal differentiation pathways and to identify pathogenetic mechanisms that lead to blindness. Her research, conducted at Penn over the past 24 years, has established the scientific underpinnings which made it possible to test the first potential retinal gene therapy treatment for patients with blinding retinal degenerations. Dr. Bennett is a Scientific Advisor of gene therapy clinical trials for congenital blindness. These Phase 3 studies, at Children's Hospital of Philadelphia and funded by Spark Therapeutics, could lead to approval of the first ocular gene therapy drug. Dr. Bennett's team initiated their second gene therapy clinical trial for a blinding disease called Choroideremia in January 2015 at the University of Pennsylvania.

Jennifer Doudna

The Li Ka Shing Chancellor's Chair in Biomedical and Health Sciences, Jennifer Doudna is Professor of Molecular and Cell Biology and Professor of Chemistry at UC Berkeley and an Investigator of the Howard Hughes Medical Institute. Doudna's research seeks to understand how RNA molecules control the expression of genetic information. Her research led to insights about CRISPR-Cas9-mediated bacterial immunity that enabled her lab and that of collaborator Emmanuelle Charpentier to re-design this system for efficient genome engineering in animals and plants, creating a transformative technology that is revolutionizing the fields of genetics, molecular biology and medicine. She is a member of the National Academy of Sciences, the American Academy of Arts and Sciences, the National Academy of Medicine and the National Academy of Inventors. She is a recipient of awards including the NSF Waterman Award, the FNIH Lurie Prize, the Paul Janssen Award for Biomedical Research, the Breakthrough Prize in Life Sciences, the Princess of Asturias Award (Spain), the Gruber Prize in Genetics, the Massry Prize, the Gairdner Award, the Nakasone Prize and the L'Oreal-UNESCO International Prize for Women in Science.

Matthew Heeney, MD

Matthew Heeney, MD, is the clinical director of the Blood Disorders Center at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and director of its Sickle Cell Program. He is also associate chief of hematology in the Division of Hematology/Oncology at Boston Children's Hospital and assistant professor of pediatrics at Harvard Medical School. Dr. Heeney has been Dana-Farber/Boston Children's principal investigator for several NIH funded clinical trial consortia and site investigator for several multicenter clinical trials in sickle cell disease. He also conducts translational research in inherited disorders of iron homeostasis with particular interest in the genetic basis of disorders of iron metabolism and sideroblastic anemia. Dr. Heeney earned his BSc from the University of Toronto and his MD from the University of Calgary, Alberta, Canada. He completed his residency at Montreal Children's Hospital, McGill University, and his fellowship in pediatric hematology/oncology at Duke University.

Edward Kaye

Senior Vice President, Chief Medical Officer, Interim CEO of Sarepta Therapeutics

Edward M. Kaye, M.D., has served as the Interim Chief Executive Officer of Sarepta since March 31, 2015 and as the Senior Vice President and Chief Medical Officer since June 2011. Dr. Kaye previously held roles at Genzyme Corporation since 2001, including Group Vice President of Clinical Development and Personalized Genetic Health, where he supervised clinical research in the lysosomal storage disease programs and in genetic neurological disorders. Dr. Kaye earned his B.S. in Biology from Loyola University and earned his M.D. at Loyola University Stritch School of Medicine. In 1996 he moved to Philadelphia to become Chief of Pediatric Neurology at St. Christopher's Hospital for Children. In 1998, Dr. Kaye accepted the appointment as Chief of Biochemical Genetics at Children's Hospital of Philadelphia, until moving to Genzyme Corporation at the end of 2001. Dr. Kaye is on the editorial boards of a number of journals including Journal of Child Neurology. Dr. Kaye is also on the Medical/Scientific Advisory Boards of the Spinal Muscular Atrophy Foundation, CureCMD and Prize4Life.

Freda Lewis-Hall, MD, DFAPA

Executive Vice President and Chief Medical Officer, Pfizer

As Chief Medical Officer, Freda Lewis-Hall leads efforts at Pfizer to ensure the safe, effective and appropriate use of the company's medicines and vaccines and to collaborate with members of the medical research community to accelerate the development of new treatments and improve outcomes for patients. She chairs the Cures Acceleration Network at the National Institutes of Health and serves on the boards of the Patient-Centered Outcomes Research Institute, Harvard Medical School, Tenet Healthcare Corporation and Save the Children. A passionate advocate for empowering patients through access to medical information, she appears frequently on TV shows including The Doctors and Dr. Phil and blogs on GetHealthyStayHealthy.com. Dr. Lewis-Hall earned her undergraduate degree at The Johns Hopkins University and her medical degree from Howard University College of Medicine. She is the author of Make Your Mark: Why Legacy Still Matters.

Albert Maguire, MD

Albert Maguire, MD, is a Professor of Ophthalmology, Co-Director of the Center for Advanced Retinal and Ocular Therapeutics, and vitreo-retinal surgeon at the Scheie Eye Institute at University of Pennsylvania Perelman School of Medicine. He is also the retina specialist at the Children's Hospital of Philadelphia. Dr. Maguire developed surgical approaches to demonstrate proof-of-concept in retinal gene therapy studies. He has trained numerous investigators at dozens of institutions nationally and internationally on technical aspects of these procedures. His research over the past two-and-a-half decades, resulted in implementation of the first retinal gene therapy clinical trial in the USA (2007, for Leber Congenital Amaurosis. This was also the first gene therapy trial for a non-lethal pediatric disease. Dr. Maguire also directs a gene therapy clinical trial for choroideremia, a blinding disease.

Dennis Nielson, MD, PhD

Dr. Dennis Nielson is a Professor of Clinical Pediatrics at UCSF and the Director of the UCSF Cystic Fibrosis Center since 2001. He served as the Chief of the UCSF Division of Pediatric Pulmonary Medicine from 2001 to 2015. He received his MD and PhD in Physical Chemistry from the University of Utah. Dr. Nielson's scholarly work has covered a wide range of topics: from epithelial ion transport to clinical studies of cystic fibrosis and other chronic lung disorders. He has been the principal investigator or participating investigator on a variety of grants from NIH, CF Foundation, and other agencies. Dr. Nielson's current focus is on increasing our understanding of how to match CF patients with the new emerging small molecule therapies for CF that increase throughput and insertion into epithelial cell membranes and augment function of defective CF proteins.

Stuart H. Orkin, MD

Stuart H. Orkin, MD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center, has been a leading researcher of sickle cell disease and other hematalogic disorders for three decades. He is also the David G. Nathan Professor of Pediatrics at Harvard Medical School and an investigator of the Howard Hughes Medical Institute. His current research focuses on translating basic findings on the BCL11A switch that silences fetal hemoglobin into new treatments for hemoglobin disorders and leveraging vulnerabilities in epigenetic pathways for cancer therapy. Dr. Orkin is an elected member of the National Academy of Sciences (NAS) and the National Academy of Medicine (NAM). He is recipient of the NAS's Kovalenko Medal for outstanding advances in medical research. He earned his BS from MIT and MD from Harvard Medical School and trained in pediatric hematology/oncology at Boston Children's Hospital and Dana-Farber Cancer Institute.

Stuart L. Schreiber, PhD

Stuart L. Schreiber, PhD is the Director of the Center for the Science of Therapeutics and one of four founders of the Broad Institute, where he is a Howard Hughes Medical Institute Investigator. He is also the Morris Loeb Professor of Chemistry and Chemical Biology at Harvard University and a member of the National Academy of Sciences. He discovered principles that underlie information transfer and storage in cells, specifically discoveries relating to signaling by the phosphatase calcineurin and kinase mTOR, gene regulation by chromatin-modifying histone deacetylases, small-molecule dimerizers that activate cellular processes by enforced proximity, and small-molecule probes of challenging targets and processes that relate to human disease. His work has contributed to diversity-oriented synthesis (DOS) and discovery-based small-molecule screening in an open data-sharing environment. Schreiber has participated in the founding of several biotech companies, including Vertex Pharmaceuticals (fosamprenavir/Lexiva; telaprevir/Incivek; ivacaftor/Kalydeco), ARIAD Pharmaceuticals (ponatinib/Iclusig; AP26113; ridaforolimus; AP1903), Infinity Pharmaceuticals (retaspimycin; duvelisib), Forma Therapeutics and H3 Biomedicine.

Edwin Stone, MD, PhD

Edwin M. Stone is the Director of the Stephen A. Wynn Institute for Vision Research at the University of Iowa. He received his MD and PhD from the Baylor College of Medicine and his training in vitreoretinal surgery at the University of Iowa where he joined the faculty in 1990. He is known for his work in defining the genetic basis of blinding eye diseases: ranging from two of the most common causes of blindness, macular degeneration and glaucoma, to much rarer conditions such as retinitis pigmentosa and Usher syndrome. Dr. Stone has been active in removing the technical, legal and financial barriers between genetic discoveries and the patients who could benefit from them. He founded the Carver Nonprofit Genetic Testing Laboratory at the University of Iowa that provides low-cost genetic tests for inherited eye diseases to patients in more than 60 countries. He has received the Doyne Medal from the Oxford Congress of Ophthalmology, the Gregg Medal from the Royal Australian College of Ophthalmology, and the Cogan Award from the Association for Research in Vision and Ophthalmology. He holds the Seamans-Hauser Chair of Molecular Ophthalmology in the University of Iowa Carver College of Medicine.

Sonia Vallabh and Eric Minikel

In 2010, Sonia Vallabh watched her 52-year-old mother die of a rapid and mysterious, neurodegenerative disease. One year later, Sonia learned the cause--a genetic prion disease, and that she herself had inherited the deadly mutation. No treatment or cure is available, and without a medical breakthrough, she will almost certainly suffer her mother's fate within 20 years. Sonia and her husband Eric Minikel had no biology training: she was a recent Harvard Law School graduate, and he was an MIT-trained city planner and engineer. Nevertheless, the couple set out to re-train themselves as scientists and devote their lives to searching for a treatment or cure for her disease. They started a scientific blog, began taking night classes and attending conferences, found entry-level jobs in research labs, and eventually enrolled as PhD students in biology at Harvard Medical School. They are now based in Stuart Schreiber's laboratory at the Broad Institute of MIT and Harvard. Here, under the guidance of an extensive team of Broad scientists including founding director Eric Lander, they have launched a new therapeutic initiative to discover drugs for her disease. In February 2016, Sonia was invited to share her journey with President Obama upon the first anniversary of the Precision Medicine Initiative. Their conversation can be viewed on the White House website. A recent telling of Sonia and Eric's story can also be found in the Boston Globe Magazine.

Feng Zhang

Feng Zhang is a Core Member at the Broad Institute of MIT and Harvard, an Investigator at the McGovern Institute for Brain Research at MIT, and an Assistant Professor in the Department of Brain and Cognitive Sciences. He obtained an AB in Chemistry and Physics from Harvard University in 2004. As a PhD student in the Chemistry Department at Stanford University, Zhang worked with Karl Deisseroth to develop optogenetics technologies for dissecting brain circuits, using light-sensitive proteins from microbes to control of neuronal activity in living organisms with light. After finishing his PhD in 2009, Zhang joined the Harvard Society of Fellows as a Junior Fellow (2009-2010), focusing on developing gene editing tools based on transcription activator-like effectors (TALEs). In 2011, Zhang began his own laboratory at the Broad and McGovern Institutes, where he and his team pioneered the use of microbial CRISPR-Cas systems for gene editing in eukaryotic cells. His lab continues to play a critical role in the development of gene editing technologies and applications that are accelerating research around the world.

Production

John Rubin

Executive Producer

John Rubin is a Peabody-winning and multiple Emmy-winning filmmaker who turned to documentaries after completing his PhD in cognitive science at MIT. His films often explore the intersection of natural history and science.

Rob Whittlesey

Director, Producer

For over thirty years Rob Whittlesey has been making critically acclaimed, award-winning documentary films, for distribution via PBS broadcast and direct to classroom. Through those decades, the tools he's employed have evolved from 16mm film to digital high definition, but what hasn't changed is his passion for sharing real people's stories in a visual and memorable way. Rob's many producer-director credits include programs for the PBS mini-series Strange Days on Planet Earth, Rx for Survival, Evolution, and A Science Odyssey. He's also made ten films for the PBS series NOVA, as well as episodes of PBS' Frontline, Nature, and Adventure series. The New York Times called his PBS special Apollo 13: To the Edge and Back (a National Emmy nominee) "gripping," and Variety added, "an upcoming feature film, starring Tom Hanks and Kevin Bacon…will have to work hard to match the impact of this docu."

Among the many accolades his films have received are five National News and Documentary Emmy nominations and top prizes at competitions as varied as Wildscreen, Jackson Hole Wildlife Film Festival, and the Explorer's Club Film Festival.