Our Company
Leadership Team
Our team is built with experts in the fields of gene therapy, manufacturing and drug development. Our driven and compassionate team comes to work each day to make progress towards our goal of delivering cures to patients living with devastating diseases.
Scientific Advisory Board
Dr. 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.
Dr. Thomas Shenk has served as a member of our board of directors since June 2015. Dr. Shenk has been the James A. Elkins Jr. Professor of Life Sciences in the Department of Molecular Biology at Princeton University since 1984. Dr. Shenk served on the board of directors of Merck and Co. Inc., a pharmaceutical company, from 2001 to 2012. Dr. Shenk currently serves as a director of Vical Incorporated, a biopharmaceutical company, and Kadmon Holdings, Inc., a biopharmaceutical company. He received a B.S. from University of Detroit and a Ph.D. from Rutgers University.
Dr. Arnold Levine has served as a member of our board of directors since February 2016. Dr. Levine currently serves as a professor emeritus at the Institute for Advanced Study at Princeton University. He was a professor in the Department of Molecular Biology at Princeton University from 1984 until 1996, where he was named the Harry C. Wiess Professor in Life Sciences and was the chairman of the department. Dr. Levine received a B.A. from Harpur College, State University of New York at Binghamton and a Ph.D. in microbiology from the University of Pennsylvania.
Dr. 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.
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 New York-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.
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.
Dr. Chuan He 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 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.
Dr. Kevin Weeks, 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, as rigorous chemical tools for understanding RNA molecules in cells and viruses did not exist.
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.
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 from 1997 to 2001.
The Batey laboratory seeks to understand how structured RNA elements direct 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.
Dr. 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.”
Dr. Lynne Maquat is the J. Lowell Orbison Endowed Chair, Professor of Biochemistry & Biophysics, Founding Director of the Center for RNA Biology, and Founding Chair of Graduate Women in Science at the University of Rochester, Rochester, NY. After obtaining her Ph.D. in Biochemistry from the University of Wisconsin-Madison and undertaking post-doctoral work at the McArdle Laboratory for Cancer Research, she joined Roswell Park Cancer Institute before moving to the University of Rochester. Dr. Maquat’s research focuses on the molecular basis of human diseases, with particular interest in mechanisms of mRNA decay. Dr. Maquat discovered nonsense-mediated mRNA decay (NMD) in 1981 and, subsequently, the exon-junction complex (EJC) and how the EJC marks mRNAs for a quality-control “pioneer” round of protein synthesis. She also discovered Staufen-mediated mRNA decay, which mechanistically competes with NMD and, by so doing, new roles for short interspersed elements and long non-coding RNAs. She is an elected Fellow of the American Association for the Advancement of Science (2006), and an elected Member of the American Academy of Arts & Sciences (2006), the National Academy of Sciences (2011), and the National Academy of Medicine (2017), and a Batsheva de Rothschild Fellow of the Israel Academy of Sciences & Humanities (2012-3). She received the William C. Rose Award from the American Society for Biochemistry & Molecular Biology (2014), a Canada Gairdner International Award (2015), the international RNA Society Lifetime Achievement Award in Service (2010) and in Science (2017), the FASEB Excellence in Science Award (2018), the Vanderbilt Prize in Biomedical Science (2017), the Wiley Prize in Biomedical Sciences (2018), and the International Union of Biochemistry and Molecular Biology Medal (2019).
Dr. 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.
Dr. Thomas Shenk has served as a member of our board of directors since June 2015. Dr. Shenk has been the James A. Elkins Jr. Professor of Life Sciences in the Department of Molecular Biology at Princeton University since 1984. Dr. Shenk served on the board of directors of Merck and Co. Inc., a pharmaceutical company, from 2001 to 2012. Dr. Shenk currently serves as a director of Vical Incorporated, a biopharmaceutical company, and Kadmon Holdings, Inc., a biopharmaceutical company. He received a B.S. from University of Detroit and a Ph.D. from Rutgers University.
Dr. Arnold Levine has served as a member of our board of directors since February 2016. Dr. Levine currently serves as a professor emeritus at the Institute for Advanced Study at Princeton University. He was a professor in the Department of Molecular Biology at Princeton University from 1984 until 1996, where he was named the Harry C. Wiess Professor in Life Sciences and was the chairman of the department. Dr. Levine received a B.A. from Harpur College, State University of New York at Binghamton and a Ph.D. in microbiology from the University of Pennsylvania.
Dr. 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.
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 New York-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.
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.
Dr. Chuan He 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 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.
Dr. Kevin Weeks, 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, as rigorous chemical tools for understanding RNA molecules in cells and viruses did not exist.
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.
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 from 1997 to 2001.
The Batey laboratory seeks to understand how structured RNA elements direct 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.
Dr. 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.”
Dr. Lynne Maquat is the J. Lowell Orbison Endowed Chair, Professor of Biochemistry & Biophysics, Founding Director of the Center for RNA Biology, and Founding Chair of Graduate Women in Science at the University of Rochester, Rochester, NY. After obtaining her Ph.D. in Biochemistry from the University of Wisconsin-Madison and undertaking post-doctoral work at the McArdle Laboratory for Cancer Research, she joined Roswell Park Cancer Institute before moving to the University of Rochester. Dr. Maquat’s research focuses on the molecular basis of human diseases, with particular interest in mechanisms of mRNA decay. Dr. Maquat discovered nonsense-mediated mRNA decay (NMD) in 1981 and, subsequently, the exon-junction complex (EJC) and how the EJC marks mRNAs for a quality-control “pioneer” round of protein synthesis. She also discovered Staufen-mediated mRNA decay, which mechanistically competes with NMD and, by so doing, new roles for short interspersed elements and long non-coding RNAs. She is an elected Fellow of the American Association for the Advancement of Science (2006), and an elected Member of the American Academy of Arts & Sciences (2006), the National Academy of Sciences (2011), and the National Academy of Medicine (2017), and a Batsheva de Rothschild Fellow of the Israel Academy of Sciences & Humanities (2012-3). She received the William C. Rose Award from the American Society for Biochemistry & Molecular Biology (2014), a Canada Gairdner International Award (2015), the international RNA Society Lifetime Achievement Award in Service (2010) and in Science (2017), the FASEB Excellence in Science Award (2018), the Vanderbilt Prize in Biomedical Science (2017), the Wiley Prize in Biomedical Sciences (2018), and the International Union of Biochemistry and Molecular Biology Medal (2019).