Gene and cell engineering advances have influenced almost all branches of life sciences. From the lab to the clinic, innovative gene synthesis technologies and gene editing tools have transformed basic, translational, and biomedical research. GenScript has partnered with pioneers pushing the boundaries in gene and cell engineering by providing its deep expertise in gene synthesis technologies. This first, GenScript Gene & Cell Engineering Virtual Summit will showcase cutting-edge research from leaders leveraging synthetic biology technologies in drug-discovery, de novo protein design, and genome-editing.
One Day of exceptional scientific sessions entirely online with free registration.
Engage and network with leaders in the field without travel hassles.
Learn about the latest discoveries, deep insights and visions for the future from experts in the field.
Earn continuing education credit (CEC).
Alexander Marson, MD, PhD
Director, Gladstone-UCSF Institute of Genomic Immunology
Ben Kleinstiver, PhD
Assitant Professor, MGH, HMS- Center for Genomic Medicine
Gal Cafri, PhD
Immunotherapy and Genetic Engineering Group Leader, Sheba Medical Center
Alex Marson is Director of the Gladstone-UCSF Institute of Genomic Immunology and Associate Professor in the UCSF Department of Medicine, Division of Infectious Diseases. He serves as the scientific director for Human Health at the Innovative Genomics Institute (IGI) and is a member of the Parker Institute for Cancer Immunotherapy and a Chan Zuckerberg Biohub investigator. Work in Dr. Marson’s lab aims to understand the genetic programs controlling human immune cell function in health and disease, with an emphasis on developing and applying CRISPR genome engineering tools to primary immune cells, especially T cells. Combining genomics and gene editing approaches, the lab works to assess the consequences of coding and noncoding genetic variation on immune cell function and autoimmune disease risk and to genetically engineer human immune cells to target cancer, autoimmunity, and infectious diseases.
Ben Kleinstiver is a biochemist with interests in genome editing technology development and scalable protein engineering. He received his Ph.D in Biochemistry from the University of Western Ontario, and completed his postdoctoral studies at Massachusetts General Hospital and Harvard Medical School. Within the Center for Genomic Medicine at MGH, the Kleinstiver laboratory is focused on accelerating the development of CRISPR technologies. The major research goals in his laboratory are to address limitations of existing technologies, to develop new capabilities that solve outstanding needs in the genome editing field, all with the hope of transforming these technologies into genetic therapies to providing safe and effective treatments for patients.
Dr. Cafri specialized in tumor immunology and cancer immunotherapy. For the last 17 years, Dr. Cafri studies the interactions between tumors and the immune system. He began his career working on chimeric molecules to enhance vaccine activity against skin cancer at the Weizmann Institute of Science. Later on, Dr. Cafri spent 4.5 years at the National Cancer Institute (NCI) in Bethesda, Maryland, specializing in treating patients with immune cells directed against cancer mutations. During his time at the NCI, Dr. Cafri was responsible for developing two clinical trials aiming to vaccinate cancer patients with their tumor mutations. Dr. Cafri also developed a method to isolate tumor-specific immune cells from patients' blood - a technique that can bypass the need for tumor resection to develop effective cancer immunotherapies. Dr. Cafri lab develops T-cell receptor therapy for patients with common epithelial cancers and innovative genetic engineering approaches to introduce genes into human T-cells.
J Joseph Melenhorst, PhD
Professor, Pathology and Laboratory Medicine, University of Pennsylvania and Director, Biomarker Program, Parker Institute for Cancer Immunotherapy, UPenn
John Zuris, PhD
Associate Director, Editing Technologies at Editas Medicine
Karla Camacho Soto, PhD
Senior Scientist, Merck
Dr. Jan Joseph (Jos) Melenhorst Professor of Pathology & Laboratory Medicine and Director of the Biomarker Program at the University of Pennsylvania. He obtained his PhD at the University of Leiden, Netherlands (Department of Hematology) on the immune etiology of Aplastic Anemia. In 1998 he moved to the National Institutes of Health in Bethesda, Maryland, where he did his research ‐ first as a postdoc, later as a staff scientist ‐ on the immunobiology of marrow failure syndromes, leukemic disorders, and allogeneic stem cell transplantation. In 2012 he was recruited by Dr. Carl June to the University of Pennsylvania, first as Deputy Director of their clinical manufacturing (cGMP) facility. After a year he was promoted to Director of Product Development & Correlative Sciences and Adjunct Associate Professor Pathology & Laboratory Medicine. In this role, he was at the cusp of the first ever CAR T cell therapy approved by FDA: Kymriah. Further, Dr. Melenhorst led to the development of correlative assay pipeline for the first triple CRISPR/Cas9 genome edited, TCR tumor-redirected T cell product in the USA, published early January of 2020 in Science magazine. In 2020 he was promoted to full Professor at the Department of Pathology & Laboratory Medicine where he now fully focuses his effort on the translational sciences of immunogene therapies. His laboratory’s goals are to enhance our understanding and improvement of the anti-tumor efficacy and safety of adoptively transferred chimeric antigen receptor-modified T cells through correlative, mechanistic, and functional genomics approaches
John Zuris has spent the last six years at Editas Medicine, a genome editing company focused on using CRISPR to potentially cure genetic diseases. As Associate Director of Editing Technologies, he focuses on leveraging both the CRISPR-Cas12a and the CRISPR-Cas9 nuclease modalities to achieve the best editing outcome for a chosen indication. Prior to joining Editas Medicine, John completed his postdoctoral training in the laboratory of Dr. David Liu (Harvard University-Broad Institute) where he developed a lipid nanoparticle delivery system for CRISPR-Cas9 RNPs which allows for highly efficient genome editing in ex vivo and in vivo applications. This delivery technology was later utilized to successfully treat a rare form of deafness in an animal model. Before entering the CRISPR-space, John spent his graduate career elucidating the iron-sulfur cluster transfer and redox mechanisms for a metalloprotein involved in Type II diabetes under the supervision of Dr. Patricia Jennings (UC San Diego).
I received my BS in Chemistry from the University of Puerto Rico in 2009 where I synthesized and characterized the formation of supramolecular structures based on G-quadruplexes. I moved to the beautiful Arizonan desert to obtain my PhD in Chemistry from the University of Arizona under the guidance of Dr Indraneel Ghosh. During my PhD I was involved in the design and discovery of bivalent inhibitors of protein kinases using a cyclic peptide phage display approach. I also had the chance to engineer split-kinases and split-phosphatases to study signal transduction in cellular pathways. After receiving my PhD in 2015 I joined a small Biotech company where I developed mammalian cell based assays to profile kinase inhibitors promiscuity and membrane permeability. I joined Merck in 2018 as a Senior Scientist in the Protein Engineering group where I have had the chance to lead enzyme engineering programs to support manufacturing routes.
Killian S. Hanlon, PhD
Research Fellow, Harvard Medical School; Massachusetts General Hospital
Matthew Porteus, MD, PhD
Professor, Stanford University, School of Medicine
Niren Murthy, PhD
Professor, Department of Bioengineering UC Berkeley- innovative Genomics Institute
Matthew Porteus MD, PhD is the Sutardja Chuk Professor of Definitive and Curative Medicine and a Professor in the Department of Pediatrics, Institute of Stem Cell Biology and Regenerative Medicine and Maternal-Child Health Research Institute at Stanford. His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood (such as sickle cell disease) but also of other organ systems as well. He received his undergraduate degree at Harvard in History and Science where his honors thesis studied the recombinant DNA controversy of the 1970s. He then completed his MD and PhD training at Stanford, clinical training in Pediatric Hematology/Oncology at Boston Children’s Hospital, and post-doctoral research training with Noble Laureate David Baltimore at CalTech. He works as an attending physician on the Pediatric Hematopoietic Stem Cell Transplant service at Lucile Packard Children’s Hospital where he cares for children under going bone marrow transplantation for both malignant and non-malignant diseases. His goal is to combine his research and clinical interests to develop innovative curative therapies. He served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NexTRAC advisory committee evaluating the emergence of new technologies.
Dr. Niren Murthy is a professor in the Department of Bioengineering at the University of California at Berkeley. Dr. Murthy’s scientific career has focused on the molecular design and synthesis of new materials for drug delivery and molecular imaging. The Murthy laboratory developed the hydrocyanines in 2009, which are now one of the most commonly used probes for imaging reactive oxygen species and commercially available from multiple sources. The Murthy laboratory has developed several new nanoparticulate technologies for drug delivery, such as the polyketals, which have been used by numerous laboratories to enhance the delivery of small molecules and proteins. The Murthy laboratory has been recently focused on developing non-viral delivery vehicles that can deliver Cas9 protein, gRNA and Donor DNA in vivo. Dr. Murthy received the NSF CAREER award in 2006, and the 2009 Society for Biomaterials Young Investigator Award.
Manager of Technical Applications at MaxCyte Inc.
Ramarao Vepachedu, PhD
Development Scientist IV, Leidos Biomedical Research, Inc.
Sam Sternberg, PhD
Assistant Professor, Columbia University
Rama Shivakumar is the Manager, Technical Applications at MaxCyte Inc., a clinical stage biotechnology company that specializes in a flow electroporation platform. As the company’s knowledge manager, Rama coordinates the training and data generation used by the company’s Sales, Marketing, R&D and Engineering teams to expand the adoption of MaxCyte’s technology and is responsible for developing technical notes and other customer facing materials. With over 20 years of experience in biotech, she has extensive hands on knowledge of Electroporation, Protein Production, Process Development of Viral Vector Production and Protocol Development. Rama received her graduate training in Molecular Biology from Indiana University School of Medicine at Indianapolis.
Dr. Ramarao Vepachedu is currently working as the Process Development Lead for Cell and Gene therapy projects at Leidos Biomedical Research. His areas of focus are Cell and Gene Therapy, viral vectors, viral vaccines, and monoclonal antibodies. Dr. Vepachedu earned his Ph.D. in Biochemistry from Osmania University, India in the year 1997. He conducted post-doctoral research on DNA vaccines at the Indian Institute of Science, Bangalore, India. Later Dr. Vepachedu investigated host-pathogen interactions and immune response mechanisms at Colorado State University and National Jewish Health. Dr. Vepachedu joined AstraZeneca/Medimmune in 2008 and led the process development and clinical production of live attenuated vial vaccine for the respiratory syncytial virus, recombinant proteins, and monoclonal antibodies. Dr. Vepachedu joined Leidos Biomedical at Frederick National Laboratories in 2014 and led the development of the production process for multiple CART cell therapy, AAV gene therapy, and monoclonal antibody drug products. His lab is interested in cell engineering and methods to improve cell and gene therapy drug production.
Sam H. Sternberg, PhD, runs a research laboratory at Columbia University, where he is an assistant professor in the Department of Biochemistry and Molecular Biophysics. He received his B.A. in Biochemistry from Columbia University in 2007, graduating summa cum laude, and his Ph.D. in Chemistry from the University of California, Berkeley in 2014, under the mentorship of Dr. Jennifer Doudna. He earned graduate student fellowships from the National Science Foundation and the Department of Defense, and received the Scaringe Award and the Harold Weintraub Graduate Student Award. Sam's research focuses on the mechanism of DNA targeting by RNA-guided bacterial immune systems (CRISPR-Cas) and on the development of these systems for genome engineering applications. He is the recent recipient of the NIH Director’s New Innovator Award, and is a Sloan Fellow and Pew Biomedical Scholar. In addition to publishing his work in leading scientific journals, he co-authored a popular science book with Jennifer Doudna, entitled A Crack in Creation, about the discovery, development, and applications of CRISPR gene-editing technology.
Shondra M. Pruett-Miller, PhD
Director, St. Jude Children’s Research Hospital Comprehensive Cancer Center
Ye Liu, PhD
Senior Director of Gene Transfer Technologies, REGENXBIO Inc.
Shondra Pruett-Miller, Ph.D. is an Assistant Member in the Department of Cell and Molecular Biology, the Founding Director of the Center for Advanced Genome Engineering (CAGE), and the Associate Director of Shared Resoucres for the Comprehensive Cancer Center at St. Jude Children’s Research Hospital in Memphis, TN. Shondra completed her Ph.D. in Cell and Molecular Biology from The University of Texas Southwestern Medical Center in August 2008. While at UT Southwestern, she worked in Matthew Porteus’ lab on the optimization of zinc finger nucleases for use in mammalian cells. After graduate school, she was recruited to Sigma-Aldrich as a Senior Scientist in R&D working on their CompoZr ZFN technology. In 2012, she returned to academia as the Founder and Director of the Genome Engineering and iPSC Center (GEiC) at Washington University School of Medicine in St. Louis. In 2017, she joined the Faculty at St. Jude Children’s Research Hospital where she established and is directing the Center for Advanced Genome Engineering (CAGE).
Ye Liu, PhD, is the senior director of gene transfer technologies at REGENXBIO Inc, a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Dr. Liu leads a team of multidisciplinary scientists to improve the safety, efficacy and precision of in-vivo gene therapy by developing novel AAV capsid and transgene cassette. Prior to REGENXBIO, Dr. Liu was the director of genetic engineering and vaccine development at NOVAVAX, where he developed nanoparticle vaccines against infectious diseases such as influenza and SARS-CoV. He received his PhD in biochemistry from Wayne State University and completed his postdoctoral training in genetic medicine at Johns Hopkins University with Nobel Laureate Dr. Gregg Semenza.
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Genome editing technologies have enabled the precise manipulation of DNA sequences at targeted sites to achieve therapeutic effects. Engineered endonucleases like CRISPR-Cas9 institute site-specific DNA breaks to either knock out a gene or enhance the homology directed repair (HDR) based gene correction with an intact donor template. High-fidelity editing, multiplexing capacity, and the ease of implementing the CRISPR-Cas9 system have expanded the scope of programmable genetic manipulations to include simultaneous deletions or insertions of multiple DNA sequences in a single round of mutagenesis. Gene editing has many applications in basic and biomedical research, including disease modeling.
A promising cell therapy approach for hemoglobinopathies or cancer immunotherapy involves the ex vivo gene editing of autologous hematopoietic stem cells (HSCs) or T cells before administering patients.
For hemoglobinopathies, gene editing tools like CRISPR-Cas9 can be used to install a double stranded break to either knock out a gene like BCL11A or enhance the knock-in with an intact donor DNA template to correct the mutation in the beta subunit of the hemoglobin gene (HBB).
Cancer immunotherapy applications use CRISPR-Cas9 to knock out the TCR locus or checkpoint modulators to improve the efficacy and clinical outcomes of the living drug.
Here, we describe the use of a clinically validated, regulatory compliant, scalable electroporation platform for the high efficiency, low toxicity gene editing of hCD34+ hematopoietic stem cells (HSCs), T cells, and iPSC cells at a clinical scale for preclinical evaluation and commercial production of cell therapy products for Sickle Cell Disease, TCR therapy or in disease modeling.
AAV peptide display libraries allow for the generation of novel variants capable of high-level transduction. We previously described an AAV peptide display library combined with a sensitive transduction reporter, iTransduce, that enabled the discovery of potent capsids in the mouse brain after only two rounds of selection via intravenous injection. Analogous to drug repurposing, AAV capsids can be used for gene delivery applications other than originally intended. With that in mind, we tested two of the capsids, AAV-F and AAV-S, from our selection in mice to transduce non-human primate (NHP) spinal cord and cochlea, respectively.
We found that intrathecally injected AAV-F mediated higher expression in motor neurons and interneurons compared to AAV9 in the thoracic and lumbar regions, despite lower numbers of genomes per cell in AAV-F-treated animals. AAV-S was tested in the inner ears of mice and cynomolgus macaques, and in both cases drove robust, high-level transduction throughout the cochlea. Further, AAV-S was able to rescue the deafness phenotype of a mouse model of Clarin-1 related Usher Syndrome. Overall, this work serves to highlight that AAV variants selected from our library system can translate effectively to clinically relevant large animals, and provides further evidence for the benefits of capsid repurposing.
✔ Immunotherapy Researchers