Dr. Frank Bennett is responsible for preclinical antisense drug discovery and antisense technology research at Ionis Pharmaceuticals. He is also the franchise leader for neurological programs at Ionis. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics and medicinal chemistry. Dr. Bennet is a co-recipient of the 2019 Breakthrough Prize in Life Sciences for his contributions to the discovery and development of SPINRAZA® (nusinersen) and the 2018 Hereditary Disease Foundation’s (HDF) Leslie Gehry Brenner Prize for Innovation in Science for his leadership and continued commitment to developing antisense therapies for Huntington’s disease (HD). Dr. Bennett has published more than 200 papers in the field of antisense research and development, and he is an inventor on more than 175 issued patents.
Dr. Marlene Haffner was the Director of the Office of Orphan Products Development at the Food and Drug Administration (FDA) for 20+ years. During her tenure in that program, it became THE orphan products program in the world. In addition to assisting in the development of more than 300 products in the US, she is credited with assisting in the development of programs in Japan, the European Union, Australia, and beyond. Her extensive speaking and writing brought the term “orphan products” to the mind of many in the pharmaceutical world.
Marlene is a problem solver and creative thinker. She founded Haffner Associates in 2009 in order to continue to apply her extensive experience in development of orphan products by working with small to large biotech/pharmaceutical companies, patient advocacy groups, and government agencies. In addition to her work at Haffner Associates, Marlene serves as an expert advisor and board member to companies throughout the world.
Trained as an internist and hematologist, Marlene graduated from the George Washington University School of Medicine and did further training at the Columbia University School of Medicine and the Albert Einstein College of Medicine, in New York City. She received her Masters of Public Health from the Johns Hopkins Bloomberg School of Public Health in Baltimore. Following her tenure at FDA and prior to forming Haffner Associates, Marlene served as Executive Director, Global Regulatory Intelligence and Policy for two years at Amgen, the largest biotech company in the world.
Dr. Yu completed his undergraduate degree in biochemistry and molecular biology at Harvard College, his M.D. and Ph.D. degree (in Neuroscience) from the University of California at San Francisco, clinical neurology training at Massachusetts General Hospital and Brigham and Women’s Hospital, and a fellowship in neurodevelopmental genetics at MGH and Boston Children’s Hospital. He joined the faculty in the Division of Genetics and Genomics as Instructor in 2010 and Assistant Professor in 2013.
The Yu lab conducts genome-wide and world-wide searches to find the genes that are responsible for autism spectrum disorders (ASDs) and other neurodevelopmental conditions. We have had a particular emphasis on biallelic/recessive mutations (i.e., impacting both copies of a gene) in ASD, and have uncovered a striking enrichment of biallelic gene disruptions in affected individuals, especially in girls. We are passionate about finding ways to bring genomic tools to the bedside to help care for patients with genetic disorders. These projects range from the deployment of genome sequencing in the neonatal intensive care unit to the design and delivery of genome-guided therapeutics for sick children.
Dr. Margie Frazier has diverse healthcare-related experience in both government and non-profit sectors, working in policy as well as a clinical social worker in hospitals and outpatient settings. She currently is a consultant in rare disease, helping both industry and non-profit stakeholders navigate the drug development process as it relates to patients and families. As former executive director of the Batten Disease Support and Research Association, she grew the organization’s capacity to facilitate research and trial development, resulting in the first FDA-approved drug for CLN2 disease. She has served as a member of the NINDS Advisory Council, the chair of the Council of Patient Advocacy for the Lysosomal Storage Disease RDCRN Consortia and has been a frequent speaker and panelist on patient engagement and advocacy in rare disease. Prior to her rare disease focus, she worked as an advocate for other vulnerable populations in pediatric and adult mental health, charitable health care networks and in HIV/AIDs care provision. As part of a Ryan White Title lll primary care planning initiative, she worked closely with the NIH/HRSA to train community organizations across the U.S. to develop care networks for those with HIV/AIDS. Margie earned her Ph.D. from the Crown Family School of Social Service Administration at the University of Chicago, where she was a National Institute of Mental Health Fellow. She lives in Columbus, Ohio.
Dr. Post has more than 30 years of experience with all stages of drug development, from early discovery through FDA approval. He was a Founder and Chief Scientific Officer of LEAD Therapeutics, which was later acquired by BioMarin Pharmaceuticals where he then served as the Chief Scientific Officer. At LEAD and BioMarin, he worked on the discovery and early development of the PARP inhibitor talazoparib. Previously, he served as Senior Vice President of Research and Development for Onyx Pharmaceuticals from 2000 to 2006. In this role, he was responsible for the co-development of Nexavar through to FDA approval for renal cancer. Leonard received a PhD in Biochemistry from the University of Wisconsin, Madison. He is now the Chief Scientific Officer of Vivace Therapeutics, an oncology-focused portfolio-based drug discovery and development company.
Dr. Kaufmann is Senior Vice President of Clinical Development/Analytics & Translational Medicine with Novartis Gene Therapies. Previously she was director of the Division of Clinical Innovation at the National Center for Advancing Translational Sciences (NCATS). In this capacity, she oversaw the Clinical and Translational Science Awards (CTSA) program, which supports a national consortium of medical research institutions working to transform the way biomedical research is conducted. The program is designed to help accelerate the translation of laboratory discoveries into treatments for patients, train a new generation of clinical and translational researchers, and engage communities in clinical research efforts. In partnership with other NIH Institutes and Centers, government agencies, patient groups, nonprofits, and industry, the CTSA program supports progress across a broad range of diseases and conditions, including cancer, neurological disorders, heart disease and multiple rare diseases.
Before joining NCATS, Kaufmann was the director of the Office of Clinical Research at the National Institute of Neurological Disorders and Stroke (NINDS), where she worked with investigators to plan and execute a large portfolio of clinical research studies and trials in neurological disorders, many of them in rare diseases. Prior to joining NINDS, Dr. Kaufmann was a tenured associate professor of neurology at Columbia University in New York City. A native of Germany, she studied in Bonn, London and Paris before earning her medical degree from the University of Bonn, Germany, and a Master of Science degree in biostatistics from Columbia’s Mailman School of Public Health. She completed an internship in medicine at St. Luke’s/Roosevelt Hospital in New York City, and trained in neurology and clinical neurophysiology at Columbia University.
Dr. Kaufmann is board-certified in neurology, clinical neurophysiology, neuromuscular medicine, and electrodiagnostic medicine. She did a postdoctoral fellowship in molecular biology of mitochondrial diseases at Columbia’s H. Houston Merritt Center for Muscular Dystrophy and Related Diseases. While on the faculty of Columbia University, she worked clinically in the neuromuscular division, the electromyography laboratories, and the pediatric neuromuscular clinic. She currently sees patients in the Muscular Dystrophy Association Clinic at Children’s National Medical Center in Washington, DC. Her research focus is on the clinical investigation of spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS) and mitochondrial diseases.
Dr. Pavlath has a history of scientific leadership with experience across the biotech, non-profit and academic sectors. She is Vice President of Research at Aavanti Bio focused on building a pipeline of precision genetic therapies for rare disease patients. Previously Dr. Pavlath was Chief Research Officer at the Muscular Dystrophy Association (MDA) spearheading innovations in science by providing strategic and operational leadership of MDA's discovery, translational and clinical research portfolio. Prior to joining MDA, she led an internationally known laboratory as a tenured professor of Pharmacology at Emory University focused on cellular and molecular mechanisms involved in muscle regeneration and muscular dystrophy. She earned her doctorate in Pharmacology at Stanford University and then completed postdoctoral training at Stanford University and the University of Arizona.