The Science, Policy, and Potential of Cell and Gene Therapies

Rena Conti, Ph.D., is the associate research director of Biopharma & Public Policy for the Boston University Institute for Health System Innovation & Policy. She is also an associate professor at the Boston University Questrom School of Business. From 2006 through June 2018, Professor Conti was an Associate Professor of Health Economics and Policy at the University of Chicago Medical School and the Harris School of Public Policy. Dr. Conti is a health economist. Her research focuses on the organization, financing and regulation of medical care. She has written extensively on the pricing, demand and supply of prescription drugs.

R. John Glasspool is a senior advisor to the MIT Center for Biomedical Innovation: NEWDIGS and the Financing and Reimbursement of Cures in the US (FoCUS) Project. He is also CEO and a member of the Board of Directors of Anthos Therapeutics. Mr. Glasspool is a member of the Board of Directors of Dalcor Corporation. He was formerly the executive vice president, head of Corporate Strategy and Customer Operations at Baxalta Incorporated (NYSE: BXLT), formerly Baxter BioScience (NYSE: BAX). Prior to joining Baxter in August 2012, Mr. Glasspool was head of Region Europe and Novartis Vaccines & Diagnostics (NYSE: NVS), a member of the V&D Executive Committee. Mr. Glasspool served as a board member, vice President and president of the European Vaccine manufacturers (EVM), from 2010 until December 2012. Previously, Mr. Glasspool was head of Global Pricing & Market Access and Head of Global Commercial Operations in Novartis Pharma, where he led the functions in embedding the needs of customers and consumers for brands at all stages of their lifecycle development, launch, and in market. Mr. Glasspool joined Novartis as head of Marketing for Neuroscience. He was promoted to global head Cardiovascular & Metabolism Business Franchise in 2006, during which time he grew the franchise sales over a three-year period.

Joanne Kurtzberg, M.D., is a faculty member at Duke University where she serves as director of the Marcus Center for Cellular Cures (MC3), the Pediatric Blood and Marrow Transplant Program, and Carolinas Cord Blood Bank as well as the co-director of the Stem Cell Laboratory. She is an internationally renowned expert in pediatric hematology/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood in the emerging fields of cellular therapies and regenerative medicine. Dr. Kurtzberg pioneered the use of umbilical cord blood as an alternative stem cell source for unrelated hematopoietic stem cell transplantation (HSCT). Over the last two decades, Dr. Kurtzberg has established an internationally known pediatric transplant program at Duke, which treats children with cancer, blood disorders, immune deficiencies, hemoglobinopathies and inherited metabolic diseases. In 2010, Kurtzberg established the Julian Robertson Cell and Translational Therapy Program (CT2) at Duke.  CT2 focuses on translational studies from bench to bedside with a focus on bringing cellular therapies in regenerative medicine to the clinic. Recent areas of investigation in CT2, which are funded by the Marcus Foundation, include the use of autologous cord blood in children with neonatal brain injury, cerebral palsy, and autism, as well as preclinical and clinical studies manufacturing microglial oligodendrocyte-like cells from cord blood to treat patients with acquired and genetic brain diseases. Studies of donor cord blood cells and donor cord tissue MSCs in adults with stroke and children with cerebral palsy and autism are also underway. In 2018, Dr. Kurtzberg established and became director of the Marcus Center for Cellular Cures (MC3) at Duke. Dr. Kurtzberg established one of the largest unrelated donor cord blood banks, the Carolinas Cord Blood Bank, in the world at Duke in 1998.  The bank has a current inventory of >40,000 units and has provided cord blood units to over 2,500 patients undergoing unrelated donor HSCT over the past 20 years. Dr. Kurtzberg’s lab has developed novel assays enumerating ALDH bright cells to predict cord blood potency from segments attached to cryopreserved cord blood units, and is performing translational research testing cord blood expansion, cellular targeted therapies and tissue repair and regeneration. In 2012, under the direction of Dr. Kurtzberg, the Carolinas Cord Blood Bank received FDA approval for DuCord, a stem cell product derived from umbilical cord blood, for use in transplants between unrelated donors and recipients. Dr. Kurtzberg currently holds several INDs for investigational clinical trials. Additionally, she is the co-director of the Duke Hospital Stem Cell Transplant Laboratory. Dr. Kurtzberg has published over 300 peer-reviewed papers, multiple chapters and scientific reviews.  She is a member of the American Society of Hematology, the American Association of Blood and Marrow Transplantation, the American Society of Pediatric Hematology/Oncology, the International Society of Cellular Therapies, the Pediatric Blood and Marrow Transplant Consortium (PBMTC), and multiple other organizations.  She has served on the Board of the Foundation of Accreditation of Cellular Therapies, co-chaired the National Marrow Donor Program’s Cord Blood Advisory Group and has served on the Advisory Council of Blood Stem Cell Transplantation to Health and Human Services.  Dr. Kurtzberg was awarded a Lifetime Achievement Award from the PBMTC in 2012. Most recently, she established and is the first president of the Cord Blood Association.

Gillian Woollett, D.Phil, M.A., is the principal research scientist at Avalere Health where she leads the FDA Practice. She provides the “prequel” of scientific and regulatory strategic policy expertise that supports medicinal products gaining approval at the FDA in a manner that allows them to be successful in the public and private reimbursement world. She is building a bridge for Avalere clients from the FDA space into the traditionally separate Centers for Medicare & Medicaid Services and healthcare policy/business world. Prior to joining Avalere, Gillian was chief scientist at Engel & Novitt, LLP, and was vice president, Science and Regulatory Affairs, at the Biotechnology Industry Organization (BIO). She joined BIO after being associate vice president at the Pharmaceutical Research and Manufacturers of America. She has been an appointee on federal advisory committees to the CDC and the Department of Commerce. Trained as a molecular biologist/immunologist, Gillian publishes in peer-reviewed literature on biotechnology topics and is a frequent speaker on emerging biosciences and their ability to support better and more focused therapies. Gillian has a D.Phil in immunology from the University of Oxford and an MA and BA in the natural sciences tripos (biochemistry) from the University of Cambridge.

Moderator

Sarah J. Dash, MPH, is the president and chief executive officer at the Alliance for Health Policy. She drives the mission and vision of the Alliance, as well as advances and maintains the reputation of the organization as the leading nonpartisan resource for policymakers and health leaders in an evolving health policy environment. Sarah joined the Alliance in 2014 as the vice president for policy and became president and CEO in May 2017. Sarah has long been an influential force in shaping health policy, having served as a senior aide on Capitol Hill and as a member of the research faculty at the Georgetown University Health Policy Institute’s Center on Health Insurance Reforms. Sarah holds a master’s degree in public health from Yale School of Public Health and a Bachelor of Science in chemistry and literature from Massachusetts Institute of Technology. Sarah also holds an Executive Certificate in Nonprofit Management from Georgetown University.