Speakers

Katherine High, MD

"Gene Therapy as a Class of Therapeutics"

Dr. Katherine A. High is a physician-scientist with a longstanding interest in gene therapy for genetic disease. As an Investigator of the Howard Hughes Medical Institute at the Children's Hospital of Philadelphia (CHOP) and a faculty member of the University of Pennsylvania School of Medicine, she carried out pioneering basic and clinical studies in gene therapy for hemophilia and for a rare form of retinal dystrophy caused by mutations in the RPE65 gene. As Co-founder, President and Head of R&D at Spark Therapeutics, she led the approval of the first gene therapy for a genetic disease in the United States. Spark has received FDA breakthrough therapy designations for three different therapeutics, including for retinal dystrophy due to RPE65 mutations, and for hemophilia B and hemophilia A. Dr. High is an elected member of the National Academy of Medicine, the American Academy of Arts and Sciences, and is a Fellow of the Faculty of Pharmaceutical Medicine of the Royal College of Physicians (London). In addition to her operational role, Dr. High serves as a member of the Board of Directors of Spark, and also serves on the Board of Directors of CRISPR Therapeutics.

Kiran Musunuru, MD, PhD, MPH, ML

An actively practicing cardiologist and a committed teacher, Kiran Musunuru, MD, PhD, MPH, ML, is an Associate Professor of Cardiovascular Medicine and Genetics at the Perelman School of Medicine at the University of Pennsylvania. He studied and trained at Harvard University, Cornell University Medical College, The Rockefeller University, Brigham and Women’s Hospital, Johns Hopkins University, and the University of Pennsylvania. His research focuses on the genetics of heart disease and seeks to identify genetic factors that protect against disease and use them to develop therapies to protect the entire population. In his recent work he has been using gene editing to create a one-shot “vaccination” against heart attacks, the leading cause of death worldwide. He is a recipient of the Presidential Early Career Award for Scientists and Engineers from the White House, the American Heart Association’s Award of Meritorious Achievement, the American Philosophical Society’s Judson Daland Prize for Outstanding Achievement in Clinical Investigation, the American Federation for Medical Research’s Outstanding Investigator Award, and Harvard University’s Fannie Cox Prize for Excellence in Science Teaching. He is the author of The CRISPR Generation: The Story of the World’s First Gene-Edited Babies.

Marco Ruella, MD

Marco Ruella, MD, is Assistant Professor of Medicine in the Division of Hematology/Oncology and the Center for Cellular Immunotherapies and Scientific Director of the Lymphoma Program at the Hospital of the University of Pennsylvania. His research focuses on the study of the mechanisms of relapse after chimeric antigen receptor T cell (CART) immunotherapies with the goal of rationally designing combined innovative immunotherapies for relapsing/refractory leukemia and lymphoma. He is the author of numerous peer-reviewed publications on targeted immunotherapies for hematological cancers and is an inventor in several patents on CAR T therapy. His work has been recognized through numerous awards including the inaugural SITC EMD-Serono Cancer Immunotherapy Clinical Fellowship (2014), the AACR-BMS Oncology Fellowship in Clinical Cancer Research (2015), the ASH Scholar Award (2016), a NIH K99-R00 award (2017), the “Paola Campese” Award Leukemia Research (2017), the Cancer Support Community Award (2018), and most recently the 2018 American Society of Hematology Joanne Levy, MD, Memorial Award for Outstanding Achievement. Dr. Ruella obtained his medical degree with high honors and completed his specialization in clinical hematology at the University of Torino, Italy. After completing his fellowship, he was an attending physician in the Hematology and Cell Therapy Division of the Mauriziano Hospital and an instructor at the Biotechnology School at the University of Torino. From 2012, he was a postdoctoral fellow, and then an instructor at the University of Pennsylvania in the Center for Cellular Immunotherapies where he worked with Drs. June and Gill until appointment to his current position in 2018.

David Weiner, PhD

Dr. David B. Weiner is Executive Vice President, Director of the Vaccine and Immunotherapy Center, and the W.W. Smith Charitable Trust Professor in Cancer Research at The Wistar Institute.  Professor Emeritus at the University of Pennsylvania School of Medicine.

Dr. Weiner directs a translational molecular immunology research team focused on creating novel approaches for disease prevention and treatment using synthetic nucleic acid technology.  His group is one of the pioneering research teams in establishing the field of DNA Vaccines & Immune Therapies.  His group & collaborators accomplishments include the first clinical studies of DNA vaccines, with a focus on advances in gene optimization and in DNA delivery. This work has revitalized the field, advancing cutting edge synthetic DNA design + modification of EP delivery to the clinic which allows rapid advancement of new advances safely into human studies.  These include the world’s first Zika vaccine, the first MERS vaccine, an advanced Ebola Vaccine, and a novel HIV vaccine, among others.  Additionally, his laboratory has helped to develop cancer immune therapy approaches for HPV disease, prostate and other cancers which are currently in clinical testing. This led to the first clinically efficacious therapeutic DNA vaccine (for HPV CIN) which has moved into a licensure trial (REVEAL).  His laboratory has published over 420 papers/chapters & reviews. Dr. Weiner has received several awards/honors for these accomplishments, including the WW Smith Family Chair in Cancer Research - 2016, the Vaccine Industry Associations Outstanding Academic Research Laboratory (2015 & 2016), Named one of the Top 20 Translational Research Laboratories of the Year (Nature Biotechnology 2016, 2017 &  2018), the Stone family award for Cancer Research 2014, the NIH Directors Translational Research Award 2011, Fellow of the American Association for the Advancement of Science 2011. Fellow of the International Society for Vaccines, President International Society for Vaccines (2018-2020).  Dr. Weiner served as chair of the prestigious and highly successful Gene Therapy and Vaccine Training Program at the University of Pennsylvania (2002-2016).  Dr. Weiner has been an avid trainer, advisor, and advocate for students and fellows and junior faculty as he is highly committed to developing of the careers of young scientists.  He currently serves as the Director of the Wistar Vaccine Immune Therapy Center and serves as Executive Vice President of the Historic Wistar Institute.