The scientific advisors of Proteostasis Therapeutics, Inc. include thought leaders in the proteostasis field and in the biological pathways and processes that regulate the Proteostasis Network.
Dr. Balch has been working on understanding protein folding and membrane trafficking through the secretory pathway of eukaryotic cells in response to inherited human disease. Using a wide range of structural (x-ray/cryo-electron microscopy/NMR), biochemical, molecular and systems biology (bioinformatics, gene expression profiling and mass spectrometry) approaches, he has focused on dissecting the basic principles of the protein folding and trafficking problem to a wide variety of misfolding diseases including cystic fibrosis (CFTR), Gaucher Disease (beta-glucocerebrosidase), childhood emphysema (alpha-1-antitrypsin), and amyloid pathologies (Alzheimer's and Parkinson's). These studies provide new insight into the physiological and pathophysiological role of protein homeostasis (proteostasis) program in the function and maintenance of the mammalian cell in complex tissue environments encoded by the genome and reprogrammed by the epigenome.
Jeffrey Brodsky, Ph.D. [View Bio]
Professor, Avinoff Chair, Department of Biological Sciences, University of Pittsburgh
Dr. Brodsky received his Ph.D. in Biochemistry and Molecular Biology at Harvard University and then performed post-doctoral work with Randy Schekman at the University of California, Berkeley. Ongoing research in the Brodsky laboratory is geared toward understanding the ER associated degradation (ERAD) pathway. More recent efforts have been devoted to the identification of small molecule and genetic modulators of molecular chaperones and human diseases.
Daniel Finley, Ph.D. [View Bio]
Professor of Cell Biology, Harvard Medical School
Dr. Finley was appointed an Assistant Professor at Harvard Medical School in 1988, after completing his postdoctoral research at Massachusetts Institute of Technology. He graduated from Harvard University in 1980 and received his Ph.D. from Massachusetts Institute of Technology in 1984. His laboratory studies the proteasome, the major protease in the cell, which is critical for protein homeostasis. Dr. Finley’s group has made major contributions to our understanding of the structure, mechanism, regulation, and assembly of this intricate enzyme.
Judith Frydman, Ph.D. [View Bio]
Associate Professor, Department of Biology and BioX Program, Stanford University; Co-director, Center on Protein Folding, NIH Nanomedicine Roadmap Initiative
Dr. Frydman majored in chemistry and received her Ph.D. from the University of Buenos Aires, Argentina. She carried out her postdoctoral training with Ulrich Hartl at the Sloan Kettering Institute in New York, where she discovered the eukaryotic chaperonin TRiC and investigated the basic principles of how proteins fold following translation in eukaryotic cells. Her research program aims to understanding the biological mechanisms controlling cellular protein folding, aggregation and quality control and the relationship of these mechanisms to the genesis of proteotoxic diseases.
Stephen Frye, Ph.D. [View Bio]
Professor, Director of the Center for Integrative Chemical Biology and Drug Discovery, University of North Carolina Eshelman School of Pharmacy
Dr. Frye is an expert in the science of drug discovery as driven by synthetic organic chemistry. Before joining UNC in 2007, he served as worldwide Vice President for High Throughput and Discovery Medicinal Chemistry at GlaxoSmithKline. During his 20 years at GSK, his creation and leadership of a department focused on oncology and protein kinases resulted in the discovery of several drugs, one of which has been approved for the treatment of metastatic breast cancer and another that is in Phase III trials for kidney cancer. He is also the inventor of Avodart, GlaxoSmithKline’s drug used for treatment of benign prostate disease, which is currently under investigation for the prevention of prostate cancer. Dr. Frye earned his B.S. in chemistry at North Carolina State University and his Ph.D. in organic chemistry at UNC-Chapel Hill. He also completed off-campus graduate research at the Institut de Chemie Organique de Université de Lausanne, Switzerland.
Randal J. Kaufman, Ph.D. [View Bio]
Investigator, Howard Hughes Medical Institute; Professor of Biological Chemistry and Warner-Lambert/Parke-Davis Professor of Medicine, University of Michigan Medical School
Dr. Kaufman received his B.A. degree in molecular, cellular, and developmental biology from the University of Colorado and his Ph.D. degree in pharmacology from Stanford University, where he studied gene amplification as a mechanism by which cells become resistant to anticancer agents. He was a Helen Hay Whitney fellow with Phillip Sharp at the Center for Cancer Research at the Massachusetts Institute of Technology, where he developed gene transfer technologies based on gene amplification and expression in mammalian cells. After postdoctoral studies, Dr. Kaufman was a founding scientist at Genetics Institute Inc., where he engineered mammalian cells for high-level expression of therapeutic proteins, such as clotting factors that are now used to treat individuals with hemophilia. Since his move to the University of Michigan, Dr. Kaufman has focused on mechanisms by which mammalian cells regulate protein folding and secretion and a cellular response known as the unfolded protein response (UPR). He currently uses mouse models to elucidate the significance of the UPR in health and disease.
David Ron, M.D. [View Bio]
The Julius Raynes Professor of Cell Biology and Medicine, Skirball Institute, New York University School of Medicine
Dr. Ron received his medical degree from the Technion in Haifa, Israel. After completing clinical training in Internal Medicine at Mount Sinai Hospital in NYC and Endocrinology and Metabolism at the MGH in Boston, he conducted research on gene expression in adipose tissue under the supervision of Joel Habener. In 1992 he took a faculty position at New York University School of Medicine. Work in his laboratory at the Skirball Institute of Biomolecular Medicine has focused on the cellular adaptations to protein misfolding, especially as they pertain to mammalian pathophysiology.
Marc Vidal, Ph.D. [View Bio]
Professor of Genetics, Harvard Medical School; Director of the Center for Cancer Systems Biology (CCSB), Dana-Farber Cancer Institute
Originally trained as a bioengineer and a geneticist, Dr. Vidal pioneered the concept of "interactome network modeling." Having developed interdisciplinary strategies together with collaborators from the fields of physics, computer science, mathematics, genomics and human genetics, Dr. Vidal and his team have been charting the human interactome network, the complex system of interacting macromolecules operating inside cells of the body. Having discovered systems properties in the interactome, they are now starting to unravel fundamental relationships between cellular systems and human disease. Dr. Vidal was elected Associate Member of the Royal Academy for Science and the Arts of his native country Belgium and received several awards including a prestigious Chair from the Francqui Foundation and an Abbott Bioresearch Award. Dr. Vidal’s work has been featured in several documentaries (e.g.,"Connected: How Kevin Bacon cured cancer" originally broadcasted on ABC) and on the Web, including recent interviews on "Futures in Biotech" with Leo Laporte.
Jonathan S. Weissman, Ph.D. [View Bio]
Investigator, Howard Hughes Medical Institute; Professor, Cellular and Molecular Pharmacology, Biochemistry and Biophysics, University of California, San Francisco
Dr. Weissman is researching how cells ensure that proteins fold into their correct shape, as well as the role of protein misfolding in disease and normal physiology. He is also developing experimental and analytical approaches for exploring the organizational principles of biological systems. Dr. Weissman received his undergraduate physics degree from Harvard College. After obtaining a Ph.D. in physics from the Massachusetts Institute of Technology, where he worked with Peter Kim, Dr. Weissman pursued postdoctoral fellowship training in Arthur Horwich's laboratory at Yale University School of Medicine. He was recently awarded the Raymond and Beverly Sackler International Prize in Biophysics and elected to the National Academy of Sciences.
