Scientific Advisory Board
Greg A. Petsko, Ph.D., ChairmanArthur J. Mahon Professor of Neurology and Neuroscience, Weil Cornell Medical College. Director of the Helen and Robert Appel Alzheimer’s Disease Research Institute
Gregory A. Petsko is the Arthur J. Mahon Professor of Neurology and Neuroscience at Weill Cornell Medical College in New York City, and also Director of the Helen and Robert Appel Alzheimer’s Disease Research Institute. He also holds appointments as Adjunct Professor of Biomedical Engineering at Cornell University, Adjunct Professor of Neurology at Harvard Medical School, and Tauber Professor of Biochemistry and Chemistry, Emeritus, at Brandeis University. He has been elected to the National Academy of Sciences, the National Academy of Medicine, the American Academy of Arts and Sciences, and the American Philosophical Society.
He is the co-founder of several publicly-traded biotechnology companies and is one of the founding editors of the PLoS family of journals. His research interests include protein structure and function and the development of methods to treat age-related neurodegenerative diseases, including ALS (Lou Gehrig’s), Alzheimer’s and Parkinson’s diseases.
Robin Ali, Ph.D.Professor of Human Molecular Genetics at UCL Institute of Ophthalmology, London. Head of Department of Genetics & Theme Leader for Gene Therapy at NIHR Biomedical Research Centre for Ophthalmology, Moorfields Eye Hospital, London
Dr. Robin Ali is the Chief Scientific Officer at MeiraGTx. As chief investigator, Robin established the world’s first clinical trial of gene therapy for retinopathy. In 2007 he was elected to the UK Academy of Medical Sciences and in 2009 appointed Senior Investigator of The UK National Institute for Health Research. He is President of the European Society of Gene and Cell Therapy and has served on the advisory boards of several funding bodies including the UK Medical Research Council (Neurosciences and Mental Health Board and the Regenerative Medicine Research Committee), Research to Prevent Blindness USA and Fighting Blindness Ireland, as well as the advisory boards of pharmaceutical and biotech companies, including Alcon/Novartis.
Robin Ali obtained his BSc (1988) and PhD in Genetics (1993) at University College London, continued by postdoctoral training at the MRC National Institute for Medical Research and UCL Institute of Ophthalmology and established his laboratory at UCL Institute of Ophthalmology in 1999. Dr. Ali is also a visiting Professor at The Kellogg Eye Center, University of Michigan and Director of the Wolfson Gene Therapy Unit, UCL’s facility for GMP manufacture of viral vectors.
Thomas Shenk, Ph.D.James A. Elkins Jr. Professor of Life Sciences, Department of Molecular Biology, Princeton
Thomas E. Shenk is the James A. Elkins Jr. Professor of Life Sciences in the Department of Molecular Biology at Princeton University. He is a virologist who has investigated gene functions and pathogenesis of adenovirus, a DNA tumor virus, and human cytomegalovirus, a member of the herpes family of viruses. Prof. Shenk is a fellow of the American Academy of Arts and Sciences and a member of the U.S. National Academy of Sciences and the U.S. Institute of Medicine. He is a past president of the American Society for Virology and the American Society for Microbiology, and served on the board of directors of Merck and Company for 11 years. Prof. Shenk currently serves as a board member of Forge Life Science, Kadmon Corporation, the Fox Chase Cancer Center and the Hepatitis B Foundation.
Arnold J. Levine, Ph.D.Professor Emeritus, School of Natural Sciences Biology, Institute for Advanced Study, Princeton
Dr. Arnold Levine is a widely acclaimed leader in cancer research. In 1979, Levine and others discovered the p53 tumor suppressor protein, a molecule that inhibits tumor development. He established the Simons Center for Systems Biology at the Institute, which concentrates on research at the interface of molecular biology and the physical sciences: on genetics and genomics, polymorphisms and molecular aspects of evolution, signal transduction pathways and networks, stress responses, and pharmacogenomics in cancer biology. From 1998 to 2002, Dr. Levine served as President and CEO and Robert Harriet HeilbrunnProfessor of Cancer Biology at The Rockefeller University. Prior to his post at Rockefeller, he was chairman of the Department of Molecular Biology at Princeton University (1984-1996), and, prior to that, Chair of the Department of Microbiology at the Stony Brook University Medical Center School of Medicine. Numerous distinctions include:American Association for Cancer Research Academy, Fellow; National Academy of Sciences, Member; Memorial Sloan-Kettering Cancer Center, Medal for Outstanding Contributions to Biomedical Research 2000; Keio University Medical Science Fund, Japan, Keio Medical Science Prize 2000; Albany Medical Center Prize in Medicine and Biomedical Research 2001; American Association for Cancer Research, Kirk A. Landon-AACR Prize for Basic Cancer Research 2008; Dart/NYU Biotechnology Achievement Award in Basic Biotechnology 2008; American Cancer Society, Medal of Honor 2009; Lars Onsager Medal 2012.
James Alwine, Ph.D.Professor of Cancer Biology, School of Medicine, University of Pennsylvania
James C. Alwine is Emeritus Professor of Cancer Biology and former Associate Director of Abramson Cancer Center at the Pearlman School of Medicine of the University of Pennsylvania. He is also Visiting Professor of Immunobiology at the University of Arizona. He is a virologist who has studied the molecular pathogenesis and RNA biology of DNA tumor viruses and herpesviruses. He is a Fellow of the American Academy of Microbiology and the American Association for the Advancement of Science, and an Alumni Fellow of Pennsylvania State. He is co-founder of ExcaliVir, a molecular diagnostic company.
Scott A. Small, M.D.Boris and Rose Katz Professor of Neurology (in the Taub Institute for Research on Alzheimer's Disease and the Aging Brain, the Gertrude H. Sergievsky Center and in Radiology) and Director, Alzheimer's Disease Research Center, Columbia University
Dr. Scott A. Small is the Boris and Katz Professor of Neurology at Columbia University, where he is the Director of the Alzheimer’s Disease Research Center. With an expertise in Alzheimer’s disease and cognitive aging, Dr. Small’s research focuses on the hippocampus, a circuit in the brain targeted by these and other disorders, notably schizophrenia. He has pioneered the development and application of high-resolution functional MRI techniques that can pinpoint parts of the hippocampus most affected by aging and disease. His lab then uses this information to try to identify causes of these disorders. Over the years and together with various collaborations, this ‘top-down’ approach has identified novel pathogenic drivers of Alzheimer’s disease, cognitive aging, and schizophrenia. More recently, his lab has used this insight for drug discovery and to develop therapeutic interventions, some of which are currently being tested in clinical trials. Dr. Small has co-authored over 120 articles and his neuroimaging and molecular work has led to numerous patents. Dr. Small is the recipient of numerous awards, including the Beeson Scholar Award in Aging Research from the American Federation on Aging, the McKnight Neuroscience of Brain Disorders Award, the Derek Denny-Brown Young Neurological Scholar Award from the American Neurological Association, and the Lamport Award for Excellence in Clinical Science Research from Columbia University.
Michel Michaelides, M.D.Consultant ophthalmic surgeon, Moorfields Eye Hospital, Professor of Ophthalmology, UCL Institute of Ophthalmology
Dr. Michel Michaelides (BSc MB BS MD(Res) FRCOphth FACS) is a Founding Member of MeiraGTx and Professor of Ophthalmology, UCL Institute of Ophthalmology in Dept. of Genetics. He is a Consultant Ophthalmologist at MEH in Depts. Of Inherited Eye Disease, Medical Retina and Paediatric Ophthalmology.
He was awarded a Medical Retina and Genetics clinical fellowship at MEH (2008-2009) and a combined Ophthalmic Genetics and Paediatric Ophthalmology Clinical & Research fellowship at Casey Eye Institute, USA (2009-2010). He is a recipient of a career development award from the Foundation Fighting Blindness (USA) – an award which is rarely given to non-US applicants; and has gained membership of the highly prestigious Macular Society and Retina Society in the USA. He is currently the Principal Investigator of 3 active interventional clinical trials and has 10 on-going ethically approved studies, including 6 in-house natural history studies and a multi-centre international natural history study.
Michael Kaplitt, M.D., Ph.D.Vice Chair, Research, Neurological Surgery; Director, Movement Disorders and Pain, Weill Cornell Brain and Spine Center
Dr. Kaplitt graduated magna cum laude from Princeton University in 1987 with a degree in Molecular Biology and Russian Studies. He enrolled in the Tri-Institutional M.D.-Ph.D. Program and received his Ph.D. in Molecular Neurobiology in 1993 from The Rockefeller University and his M.D. in 1995 from Cornell Medical College. Following his Neurosurgery residency and chief residency at NewYork-Presbyterian/Weill Cornell, he completed a fellowship in Stereotactic and Functional Neurosurgery with Dr. Andres Lozano at the University of Toronto prior to joining our staff as Assistant Professor of Neurological Surgery in July 2001
Dr. Kaplitt combines surgical expertise with advanced training in state-of-the-art stereotactic techniques to provide patients with effective, minimally invasive treatments for degenerative disorders, including Parkinson’s disease, essential tremor, and dystonia
Dr. Kaplitt has pioneered human gene therapy for neurodegenerative disorders, having performed as a student the first rodent study that used the adeno-associated virus (AAV) vehicle for gene transfer to the brain. He performed the world’s first human gene therapy procedure for Parkinson’s disease, and more recently helped to guide the first successful randomized, double-blind, sham-controlled trial of this experimental treatment, which was the first positive trial of this kind for gene therapy in any brain disorder.
Stefano Rivella, Ph.D.Kwame Ohene-Frempong Chair on Sickle Cell Anemia and Professor of Pediatrics, The Children's Hospital of Philadelphia Research Institute
Dr. Rivella is a Professor of Pediatrics with tenure at the Children’s Hospital of Philadelphia and University of Pennsylvania and holds the Kwame Ohene-Frempong Chair on Sickle Cell Anemia. After he completed his studies at the University of Pavia, in Italy, and did his postdoctoral work at Memorial Sloan Kettering Cancer Center in New York City, he opened his laboratory at Weill Cornell Medical College in New York City, where he was Director of the Stanley Jaffe Gene Therapy Lab. Dr. Rivella is an expert in the pathophysiology of erythroid and iron disorders and in the generation of lentiviral vectors for the cure of hemoglobinopathies and has been providing expertise on mouse models important for red blood cell and macrophage biology as well as inflammation and iron metabolism to many US laboratories and pharmaceuticals interested in learning these subjects and/or testing new drugs. As such he has collaborated or provided consultation to many companies, biotechs and pharmaceuticals, such as Rana Therapeutics, Keryx Pharmaceuticals, Nektar Therapeutics, Novartis Pharmaceutical, Ionis Pharmaceutical, Merganser Biotech, Bayer Pharmaceutical, Megenics LLC, Biomarin Pharmaceutical, and Alexion Pharmaceutical. He is presently also a member of the Scientific Advisory Board (SAB) of Merganser Biotech and Ionis Pharmaceutical.
Chuan He, Ph.D.John T. Wilson Distinguished Service Professor in the Department of Chemistry, Department of Biochemistry and Molecular Biology, and Director of the Institute for Biophysical Dynamics at the University of Chicago
Chuan He, Ph.D., is the John T. Wilson Distinguished Service Professor in the Department of Chemistry, Department of Biochemistry and Molecular Biology, and Director of the Institute for Biophysical Dynamics at the University of Chicago. He was born in P. R. China in 1972 and received his B.S. (1994) from the University of Science and Technology of China. He received his Ph. D. degree from Massachusetts Institute of Technology in chemistry in 2000 with Professor Stephen J. Lippard. After being trained as a Damon-Runyon postdoctoral fellow with Professor Gregory L. Verdine at Harvard University from 2000-2002, he joined the University of Chicago as an assistant professor, and was promoted to associate professor in 2008 and full professor in 2010. He was selected as an as an Investigator of the Howard Hughes Medical Institute in 2013. He is also a member of the Cancer Research Center at the University of Chicago. His research spans a broad range of epigenetics, chemical biology, biochemistry, molecular biology, cell biology, and genomics. His recent research concerns reversible RNA and DNA methylation in biological regulation. Chuan He’s laboratory has spearheaded development of enabling technologies to study the biology of 5-hydroxymethylcytosine (5hmC) in mammalian genomes. His laboratory discovered reversible RNA methylation as a fundamental new mechanism of gene expression at the post-transcriptional level in 2011.
Kevin Weeks, Ph.D.Kenan Distinguished Professor, Department of Chemistry at the University of North Carolina
Kevin Weeks, Ph.D., is the Kenan Distinguished Professor in the Department of Chemistry at the University of North Carolina. Dr. Weeks received his Ph.D. from Yale University in 1992. Research in the Weeks group lies at the interface of chemistry, biology and genomics. One of the most amazing discoveries of recent years has been the profound role of RNA in regulating all areas of biology. For example, a much larger fraction of the human genome is transcribed into RNA than codes for protein synthesis. Further, the functions of many RNA molecules require that an RNA fold back on itself to create intricately and complexly folded structures. Until recently, however, we had little idea of the broad contributions of RNA structure and function because there simply did not exist rigorous chemical tools for understanding RNA molecules in cells and viruses.
The vision of our laboratory is therefore, first, to invent novel chemical microscopes that reveal quantitative structure and function interrelationships for RNA and, second, to apply these RNA technologies to broadly important problems in biology.
Our work is highly interdisciplinary. Projects in the laboratory meld fundamental basic science chemistry, computational chemistry, and biotechnology development and extend to practical applications in virology, high-throughput RNA structure analysis, and understanding biological processes in cells.
Current projects focus on (i) RNA folding and protein assembly reactions central to the replication of human viruses, including HIV and Dengue, (ii) function of biomedically important RNA-protein complexes inside living cells, and (iii) discovery of small molecule ligands, potential drugs, targeted against medically important RNAs.
Robert Batey, Ph.D.Professor of Chemistry and Biochemistry, University of Colorado, Boulder
Dr. Batey is a Professor of Chemistry and Biochemistry at the University of Colorado, Boulder, where he has been since 2001. He received his Ph.D. degree in Biology from the Massachusetts Institute of Technology in 1997 with Professor Jamie Williamson and was a Jane Coffin Childs postdoctoral fellow at Yale University with Professor Jennifer Doudna between 1997 and 2001.
The Batey laboratory seeks to understand how structured RNA elements directs gene expression. In 2004, his research team was the first to reveal the structural basis for small molecule binding by a naturally-occurring regulatory element called a riboswitch. These sequences, frequently found in bacterial mRNAs, directly bind a small molecule effector to an “aptamer” that directs a structural switch that in turn informs the expression machinery. Since these first insights, the Batey laboratory has worked extensively on the structural and mechanism of spectrum of riboswitches that bind diverse small molecules including guanine, S-adenosylmethionine, lysine, tetrahydrofolate, vitamin B12, and the purine biosynthetic intermediate ZMP. Using a combination of structural, biochemical and cell-based approaches, the Batey laboratory has provided many of the key insights into how small molecule binding by RNA can be harnessed to regulate mRNA expression.
More recently, we have sought to leverage our knowledge of natural aptamers to develop new approaches to evolving synthetic aptamers in vitro for cellular applications. We have shown that certain RNA architectures can act as privileged scaffolds, capable of hosting novel binding activities and retain robust intracellular function. These efforts are ongoing with the goal of developing a broad range of synthetic aptamers for a range of diagnostic and therapeutic applications.
Brian Shoichet, Ph.D.Professor of Pharmaceutical Chemistry, UCSF School of Pharmacy
Brian Shoichet is a professor of Pharmaceutical Chemistry in the UCSF School of Pharmacy. He received his Ph.D. from UCSF in 1991.
The Shoichet lab seeks to discover chemical reagents that can illuminate biological problems. A longstanding effort to do so is by exploiting protein structures to predict new reagents and therapeutic leads (structure-based ligand discovery). Two ongoing projects are: Developing new computational methods for ligand discovery and applying these to G-Protein Coupled Receptors (GPCRs), which are the single largest family of signaling receptors in human cells.
Allied with this effort is an experimental research program that tests the new methods in well-controlled systems, determining x-ray crystal structures and measuring binding thermodynamics. The experimental program has led to unexpected discoveries, including the observation that many drugs and reagents can form colloidal aggregates in solution. This has led us to investigate how the physical organic chemistry of drugs affects their behavior in vitro and in vivo, influencing drug delivery and formulation.
A new effort turns the entire structural view on its head, 5. Developing computational methods to relate receptors by the similarity of their ligands, rather than by protein sequence or structure. This changes pharmacological relationships dramatically—targets that would normally be considered sequence neighbors are pushed far apart (because their ligands are dissimilar), whereas other targets that supposedly have nothing to do with one another become neighbors (because their ligands are very similar). Since the new relationships are articulated by ligands, they may be directly tested both on isolated receptors and, increasingly, against model whole organisms, such as zebra fish, C. elegans and mice. This project seeks to discover the integrated chemical circuits through which drugs and reagents affect whole organisms.”