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RESEARCH
The Wilson lab focuses on engineering genome editing enzymes for cell-targeted delivery. CRISPR-based genome editing technology has rapidly transformed biomedical research and shows great promise for the development of novel therapeutic applications. Enzymes such as Cas9 already contain several powerful properties: binding to a specific region of the genome and performing a precise cut at that site. This provides the foundation for therapies that may soon be able to correct the genetic defects that give rise to disease. But for this promise to be fully realized, therapeutic enzymes must be delivered to cells safely and efficiently. Our laboratory is working to develop genome-editing enzymes that are readily internalized by cells. Furthermore, we strive to perform targeted delivery of these enzymes, maximizing precision in correcting specific cells, tissues, or organs. A first step towards realizing this approach has been published in Rouet et al. JACS 2018.
Recent advances in genetic and cell-based technology have demonstrated the transformative power of next-generation therapeutics. However, these treatments are incredibly expensive, and are often only available at a handful of cutting-edge research hospitals. Tragically, there are millions of people in the developing world who would benefit from such treatments, but deployment is currently impractical. The Wilson Lab’s efforts are motivated by the pressing need to improve access to life-changing technologies. A central goal of our work is to find affordable and straightforward new ways to genetic medicines, enabling cures globally.
The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.
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BIOGRAPHY
Oxana is an accomplished R&D leader with a strong track record of discovering and developing first-in-class therapeutics for rare genetic diseases. She has served as Dyne’s chief scientific officer since June 2021 after joining Dyne as senior vice president, head of research in January 2020. Prior to joining Dyne, Oxana served as head of musculoskeletal and renal research in Sanofi’s rare disease and neurological unit, advancing a pipeline of drug candidates using multiple therapeutic modalities, including nucleic acids, proteins and small molecules. She is the author of numerous patents, invited reviews, editorials, book chapters and original research articles in major scientific journals. Oxana received her Ph.D. in genetics from Moscow Genetics Institute, followed by postdoctoral fellowship training in neuromuscular diseases at the Howard Hughes Medical Institute at the University of Iowa.
The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.
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BIOGRAPHY
Nicole Gaudelli received her B.S. degree in biochemistry from Boston College in 2006 where she studied the structural and mechanistic underpinnings of neocarzinostatin biosynthesis and a non-heme iron oxygenase involved in vancomycin assembly. She earned her Ph.D. in chemistry from Johns Hopkins University where she studied monocyclic beta-lactam antibiotics and elucidated the mechanism through which they are biosynthesized. She completed her postdoctoral fellowship at Harvard University in the laboratory of Professor David R. Liu where she expanded the capabilities of base editing technology by creating the first adenine base editor (ABE) through 7 rounds of directed evolution and engineering. Both her doctoral and postdoctoral work culminated in prominent Nature publications in the fields of natural product chemistry and gene editing respectively.
Dr. Gaudelli is an inventor on numerous base editing patents and leads the gene editing technologies platform group at Beam Therapeutics where her team advances precision genetic medicines using base editors. Dr. Gaudelli is a recipient of the 2018 American Chemical Society’s “Talented 12” award, recognized as a 2018 STAT News Wunderkind, a 2018 TEDMED Hive honoree, Genetic Engineering and Biotechnology News “Top 10 Under 40 of 2019”, BioSpace’s 2019 “10 Life Science Innovators Under 40 to Watch”, and is MIT’s 2019 Technology Reviews’ 35 Innovators Under 35.
The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.
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RESEARCH
The Corn Lab develops and uses next-​generation genome editing and regulation technologies. We work on both fundamental biological discovery and potential therapies for human genetic diseases. Our focus is the mechanisms by which cells repair their DNA, maintain and differentiate hematopoietic stem cells, and use ubiquitin signaling to propagate cellular signals. Through technology development, mechanistic cellular biochemistry, and translational projects, we are working to unravel complex cellular phenotypes to further biological understanding and improve human health.
BIOGRAPHY
Jacob Corn is the Professor of Genome Biology at the ETH Zurich. His research aims to better understand and treat disease through next-​generation genome editing technologies. Jacob’s career has bridged academia and industry, working in therapeutic areas that include infectious disease, neurobiology, and oncology. His research takes a multidisciplinary approach, combining cellular biochemistry, functional genomics, computational biology, bioengineering, and biophysics. Jacob’s graduate studies at the University of California, Berkeley explored how cells replicate and protect their genomes. His postdoctoral work at the University of Washington computationally designed protein inhibitors from scratch. Jacob began his independent research career as a group leader at Genentech, where his lab discovered biological mechanisms for challenging therapeutic targets. Jacob then moved back to academia as the founding Scientific Director of the Innovative Genomics Institute and faculty at UC Berkeley. In his spare time, Jacob enjoys trips in the mountains and rock climbing.
The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.
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BIOGRAPHY
Prior to co-founding Biomatics Capital Partners in 2016, Ms. Sunderland was director of Program Related Investments for the Bill & Melinda Gates Foundation. She led the foundation’s $1.5 billion strategic investment pool, which focused on global health, global development and education. She funded 50 investments, including 30 in health care, and built a team of 10 investment professionals. Ms. Sunderland also chaired Bill & Melinda Gates Foundation’s investment committee, which reviews all program-related investments. Prior to that role, she advised foundations, development finance institutions and governments on venture capital, SME financing and technical assistance programs. Ms. Sunderland holds a B.A. from Harvard University, an MBA from Wharton Business School and an M.A. from Johns Hopkins School of Advanced International Studies. Ms. Sunderland sits on the Board of Directors for several of Biomatics’ portfolio companies including Aledade, Compass Therapeutics, eGenesis and Verana Health.
The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.
]]>Jonathan Gootenberg
Fellow, McGovern Institute
Massachusetts Institute of Technology
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ABUDAYYEH BIOGRAPHY
Omar Abudayyeh is a McGovern Institute Fellow at the Massachusetts Institute of Technology where he conducts independent research on investigating novel bacterial defense systems for genome editing and gene delivery properties. He previously was at Harvard Medical School and the Harvard-MIT Health Sciences and Technology program as an MD/PhD student. He completed his doctoral work in Feng Zhang’s lab at the Broad Institute of MIT and Harvard, where his research centered on novel CRISPR enzymes for applications in genome editing, therapeutics, and diagnostics. In recognition of his technology developments, Dr. Abudayyeh was recognized as 2018 Forbes 30 under 30 in Science and Health Care and Business Insider 30 under 30. Dr. Abudayyeh graduated from MIT in 2012 with a B.S. in mechanical engineering and biological engineering, where he was a Henry Ford II Scholar and a Barry M. Goldwater Scholar.
GOOTENBERG BIOGRAPHY
Jonathan Gootenberg earned his bachelor’s degree in mathematics and biological engineering at MIT. He received his PhD in Systems Biology from Harvard University, during which he conducted research with Aviv Regev and Feng Zhang at the McGovern Institute and Broad Institute of MIT and Harvard. During his PhD, Gootenberg focused on the development of molecular technologies for treating and sensing disease states, crossing disciplines by utilizing novel computational techniques, microbiology, biochemistry, and molecular biology to uncover new CRISPR tools, including Cas12 and Cas13. He and his co-authors developed Cas13 into a toolbox with uses in fundamental research, therapeutics, and diagnostics. He is one of the first members of the McGovern Institute Fellows program, which supports the transition to independent research for exceptional recent PhD graduates, and has received multiple awards, including Forbes 30 Under 30 and Business Insider 30 and Under.
The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.
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BIOGRAPHY
Prof. Agnieszka Czechowicz is a physician-scientist and biotech entrepreneur with 20 years of experience in stem cell biology and translational research. Dr. Czechowicz is a faculty member in Stanford University’s Department of Pediatrics, Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine Division.
Dr. Czechowicz’s primary clinical interests are in bone marrow failure, aplastic anemia, and other diseases that necessitate stem cell transplantation. Her primary research interests are in transplantation, gene-therapy/gene-editing and regenerative medicine. Multiple pre-clinical and clinical therapies are in development based upon her studies.
Dr. Czechowicz’s pioneering work has led to critical developments within the life sciences ecosystem. She is the scientific co-founder of Magenta Therapeutics, and her inventions on antibody-based conditioning have also been licensed and are being pursued by Jasper Therapeutics and Forty Seven Inc (acquired by Gilead in 2020). Her efforts have also led to critical patents in development at Editas Medicine and Decibel Therapeutics. Previously, Dr. Czechowicz spent nearly a decade as a consultant at Third Rock Ventures, where she assisted and started companies including Global Blood Therapeutics, Bluebird Bio, Editas Medicine, Decibel Therapeutics and Magenta Therapeutics. She has also advised Beam Therapeutics and Spotlight Therapeutics.
Dr. Czechowicz completed her Ph.D. work in the stem cell biology at Stanford University with Prof. Irv Weissman. She then completed her residency in Pediatrics at the Boston Children’s Hospital, and subsequently pursued subspecialty training in Pediatric Hematology and Oncology at the Dana Farber Cancer Institute while simultaneously conducting postdoctoral research with Prof. Derrick Rossi in collaboration with Prof. David Scadden. She received her M.D., Ph.D., and Bachelor’s Degree from Stanford University.
Dr. Czechowicz is a strong physician-scientist and advocate of translational research. She has done pioneering work showing that hematopoietic stem cell depletion is a critical component to donor hematopoietic stem cell engraftment. She has also led several clinical trials exploring new treatments for Fanconi Anemia. She is passionate about mentoring and training future generations of physicians and scientists, and is very supportive of helping diverse trainees on various traditional and non-traditional career paths.
The Gene Editing / Gene Therapy Seminar Series features leading academic, translational, and investor speakers in e-talks through the summer and fall.
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