Human Genetics: Unmet Needs are in the Genes - Treatments for Rare Genetic Disorders
Tuesday, February 10th
9:30 am - 10:40 am
East Foyer
In an era when biotech drugs are under intense reimbursement pressure, it may come as a surprise that exceptionally high cost treatments for rare genetic disorders continue to attract the attention of industry and investors. The promise of market exclusivity, molecularly defined patient populations and clearly understood mechanisms of action are ideal profile in today’s challenging regulatory, pharmacoeconomic and capital environment. With 2008 sales of over $2 billion, genetic disorders known as Lysosomal Storage Diseases (LSDs), continue to be an area of high unmet medical need. Join us as leading physicians and companies specializing in this unique field discuss how best to address these development and commercialization challenges.
Moderator
- Salveen Kochnover, CFA, Senior Analyst, Biotechnology Equity Research, Collins Stewart, LLC
Panelists
- Barbara Burton, MD; Director, PKU Program, Professor of Pediatrics, Northwestern University's Feinberg School of Medicine
- Daniel P. Maher; Vice President of Product Development, BioMarin
- Sylvie Gregoire, PharmD; President, Shire Human Genetic Therapies
- Chester "Chet" Whitley, MD, PhD; Professor, Gene Therapy Center, University of Minnesota
Who's Who
Barbara Burton, MD; Dr. Burton is Professor of Pediatrics at Northwestern University Medical School and is the Director of the PKU and Metabolic Disease Clinic at Children's Memorial Hospital in Chicago, IL. Dr. Burton's research interests include: Marfan Syndrome, Ehlers-Danlos syndrome, PKU and other metabolic disorders, and neurogenetic and opthalmic genetic disorders. Dr. Burton is Board certified in Pediatrics, Clinical Genetics and Clinical Biochemical Genetics. She is the past President of the Chicago Pediatric Society, and currently is President of the Society for Inherited Metabolic Disorders. She is Chairman of the Genetic and Metabolic Advisory Committee to the Illinois Department of Public Health. Dr. Burton is also a member of the Propionic Acidemia Foundation, the International Advocate for Glycoprotein Storage Diseases, and a member of numerous national societies including the American Society of Human Genetics and the American College of Medical Genetics. She was extensively involved in initiating expanded newborn screening in Illinois. Dr. Burton has contributed over 100 articles and chapters to the medical literature, many of them focusing on the diagnosis and treatment of inborn errors of metabolism and on the prenatal diagnosis of genetic disorders.
Daniel P. Maher; Mr. Maher joined BioMarin in August 2003. He is currently responsible for program management, and oversees all product development activities at BioMarin. From 1998 to 2003, Mr. Maher worked at Aradigm Corporation serving as vice president, program management. Prior to Aradigm, he served as director of clinical operations at Covance, Inc., and as vice president of operations at Spectra BioMedical, Inc. Prior to Spectra, Mr. Maher acted as director, therapeutics project management, at Chiron Corporation. Mr. Maher started his career at Genentech in 1981, serving in several capacities in manufacturing and process development, and finally as Manager of Product Development. Mr. Maher received a M.B.A. in health services management from Golden Gate University and a B.A. in biology from San Francisco State University.
Sylvie Gregoire, PharmD; Dr. Sylvie Gregoire joined Shire in September 2007. She is President of Shire Human Genetic Therapies and a member of Shire's Management Committee. Dr Gregoire has over 20 years of pharmaceutical and biotechnology experience. She most recently served as Executive Chairwoman of the Board of IDM Pharma, a biotechnology company in California. Prior to this she was CEO of GlycoFi, and has also held numerous leadership positions at Biogen Inc., in the United States and France. She also worked for Merck & Co. in various positions in clinical research and in European regulatory affairs both in the US and abroad. She received her Doctor of Pharmacy degree from the State University of New York at Buffalo, and her pharmacy degree from Universite Laval, Quebec City, Canada.
Chester "Chet" Whitley, MD, PhD; Dr. Whitley is Professor, Gene Therapy Center, Department of Pediatrics and Institute of Human Genetics at University of Minnesota. He is Director, PKU Clinic, University of Minnesota, treating all patients with phenylketonuria in the region. He currently evaluates tetrahydrobiopterin (Kuvan, BioMarin). His expertise and clinical practice is in lysosomal storage diseases (e.g. Gaucher Disease, Hurler Syndrome, Hunter Syndrome, Sanfilippo Syndrome, Fabry Disease, Pompe Disease) and is knowledgable of Aldurazyme, Fabrazyme, Elaprase, Cerezyme, Naglazyme, Myozyme, Zavesca and experimental work. Dr. Whitley is Pl of the NIH program project grant "Gene Therapy for Metabolic Disorders". He uses molecular genetics to study mecopolysaccharidosis (MPS) diseases. His diagnostic lab at the University offers mutation analysis by DNA sequencing for these conditions. Testing provides information that may help predict clinical outcomes of disease. Dr. Whitley is President and CEO of Zebraic Corporation, a company specializing in early diagnostic testing for mucopolysaccharidosis disease using the patentes "MPStest".