The Story Unfolds on Protein Folding: Intriguing Updates on Therapeutic Applications and Investment Opportunities
Wednesday, April 1st
8:45 am - 10:00 am
Constitutional Ballroom B
Research has led scientists to observe that Alzheimer’s disease, cystic fibrosis, and many cancers may result from an accumulation of misfolded proteins in certain tissues. These misfolded proteins aggregate and form large clumps in the brain, which give way to a group of diseases known as amyloidoses, of which Alzheimer’s is the most recognizable, and other neurodegenerative diseases. Given the promise of targeting diseases caused by protein misfolding and the vast number of possible therapeutic applications, protein folding is quickly becoming a focus of attention in biopharmaceutical research. Hear from several experts in the area who will discuss the therapeutic applications and investment opportunities that protein folding enables.
Moderator
- Christopher K. Mirabelli, PhD; Managing Director, HealthCare Ventures
Panelists
- Andrew Dillin, PhD; Associate Professor and Pioneer Developmental Chair of Molecular and Cell Biology Laboratory, Salk Institute
- Gregory J. LaRosa, PhD; Vice President, Biology, BIKAM Pharmaceuticals, Inc.
- Richard Labaudinière, PhD; President and CEO, FoldRx Pharmaceuticals, Inc.
- Dennis J. Selkoe, MD; Vincent and Stella Coates Professor of Neurological Diseases, Harvard Medical School
Who's Who
Christopher K. Mirabelli, PhD; Dr. Mirabelli joined HealthCare Ventures as a Managing Director in 2000. Dr. Mirabelli was chairman of the board and chief executive officer of LeukoSite, Inc. from 1993 through 1999, a company in which HealthCare Ventures was the lead investor. Dr. Mirabelli was a co-founder of Isis Pharmaceuticals and served with SmithKline and French Laboratories R&D Division. Dr. Mirabelli received his Doctorate degree in molecular pharmacology from Baylor College of Medicine.
Andrew Dillin, PhD; Dr. Dillin is Pioneer Developmental Chair and an associate professor in the Molecular and Cell Biology Laboratory, uses the tiny roundworm Ceanorhabditis elegans to study the process of aging by looking at a hormone that is most widely recognized for its role in diabetes among humans: insulin. The insulin signaling pathway in worms is not only almost identical to that found in humans, but Dr. Dillin discovered that insulin also controls many physiological aspects in the worm's body, including reproduction and aging. In humans, interfering with insulin/IGF-1 signaling to generate a life-prolonging benefit would lead to type 2 diabetes and possibly cancer. In worms, larval development and reproduction are affected along with longevity. Some of Dr. Dillin's earlier research had hinted at the possibility to genetically manipulate one element of the pathway without disrupting its additional functions, this led him to search for "specificity" factors that may control how and if insulin and IGF-1 impact a wide range of target genes. Recently, he and his team pinpointed a protein specifically responsible for extending lifespan and youthfulness without disrupting the worms' response to some forms of stress, development and fertility controlled by the insulin signaling pathway. Additionally, Dr. Dillin is interested in age-onset neurodegenerative diseases. Like most neurodegenerative diseases, Alzheimer's disease usually appears late in life, raising the question of whether it is a direct and disastrous consequence of aging or if the toxic protein aggregates that cause the disease simply take a long time to form. He discovered that the harmful beta amyloid aggregates accumulate when aging impedes two molecular clean-up crews from getting rid of these toxic species.
Gregory J. LaRosa, PhD; Dr. LaRosa.is Vice President of Biology at Bikam Pharmaceuticals, Inc. joining the company in January 2008. He has over 20 years of experience in inflammation drug discovery and development, in start-up and larger biotechnology companies. At Bikam Dr. LaRosa is responsible for managing all biology activities, both internally and through external contract or collaboration. Internally, Bikam has a small research biology group charged with the discovery and validation of novel small molecules that act as pharmacologic chaperones for the rod cell visual pigment apo-protein, opsin. The group has been focusing on the development and utilization in vitro and in vivo models, related to retinitis pigmentosa (RP) and age-related macular degeneration (AMD), to identify new small molecules as candidates for progression into development for RP and AMD clinical trials. Prior to arriving at Bikam, Dr. LaRosa served as Vice President of Discovery Research at Critical Therapeutics, Inc., Lexington, MA, from 2003 until 2008, as Senior Director of Immunopharmacology at Millennium Pharmaceuticals, Inc. from 1999 to 2003, and held several senior research positions at LeukoSite, Inc., and Repligen, Corp. Dr. LaRosa received a Ph.D. from Harvard University (Division of Medical Sciences) in Molecular, Cellular, and Developmental Biology.
Richard Labaudinière, PhD; Dr. Labaudinière is President and CEO of FoldRx Pharmaceuticals, Inc., and brings over 19 years of international experience in drug discovery and development, including 16 years in multinational pharmaceutical companies, such as Glaxo, RPR and Aventis. Prior to FoldRx, Dr. Labaudinière was Senior Vice-President of Research and Development at Genome Therapeutics, now Oscient Pharmaceuticals. At Genome Therapeutics, he was responsible for building and directing all drug discovery and development activities. He oversaw the development of Genome Therapeutics' lead product candidate, Ramoplanin, for the prevention, treatment and control of serious hospital-based infections and Genome Therapeutics' biopharmaceutical portfolio of discovery and development alliances with pharmaceutical companies including Amgen, AstraZeneca, bioMérieux, Schering-Plough and Wyeth, in the field of osteoporosis, asthma and infectious diseases.
Before joining GenomeTherapeutics, Dr. Labaudinière held numerous senior management positions at Glaxo, RPR and Aventis, having full oversight for discovery and development programs, in many different therapeutic areas, from target selection to drug candidate selection and clinical development programs. His R&D efforts have resulted in several therapeutics reaching clinical development and the market.
Dr. Labaudinière is a graduate of Ecole Nationale Supérieure De Chimie in Montpellier, France and is author of over 70 publications and patents.