GenScript Gene & Cell Engineering Virtual Summit

    2021年7月22日 11:00-18:00 (美國東部時間, EDT)
    July 22nd, 2021. 11:00AM – 6:00PM EDT




    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

    Director, Biomarker Program, Parker Institute for Cancer Immunotherapies, UPenn, University of Pennsylvania

    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.

    July 22, 2021 11:00AM - 11:05AM EDT
    Opening Remark
    July 22, 2021 11:05AM - 12:00PM EDT
    Keynote Presentation (Host)
    Alexander Marson, MD, PhD
    July 22, 2021 11:05AM - 12:00PM EDT
    Reprogramming Human T Cells with CRISPR.?
    Alexander Marson, MD, PhD,
    Director, Gladstone-UCSF Institute of Genomic Immunology
    See Abstract
    Human T cells are critical effectors of immune protection from infections, autoimmune pathology, and cancer immunotherapy. We use CRISPR-mediated gene editing in primary human T cells to systematically identify genetic targets that modulate the functions of T cells in contexts ranging from immunosuppression to cancer killing. By developing and applying CRISPR based methodologies such as pooled knock-in screening, CRISPR activation, and CRISPR interference, we are pinpointing the regulatory networks controlling T cell phenotypes as well as synthetic genetic programs that can be engineered into T cells to improve their utility as cell-based therapies for disease. We are working towards a range of genetically engineered cell therapies for cancer, autoimmunity, infections and other diseases.
    July 22, 2021 12:00PM - 5:00PM EDT
    Track 1: Expanding CRISPR Toolbox
    Ben Kleinstiver, PhD / John Zuris, PhD / Shondra M. Pruett-Miller, PhD / Sam Sternberg, PhD / Niren Murthy, PhD / Rama Shivakumar, PhD?
    July 22, 2021 12:00PM - 12:45PM EDT
    Building More Useful CRISPR-Cas Technologies
    Ben Kleinstiver, PhD,
    Assistant Professor, Massachusetts General Hospital and Harvard Medical School
    See Abstract
    • Engineered CRISPR-Cas variants overcome natural limitations of wild-type enzymes
    • The genome is now nearly completely editable
    July 22, 2021 12:45PM - 1:30PM EDT
    An Engineered AsCas12a nuclease facilitates the rapid generation of therapeutic cell medicines
    John Zuris, PhD,
    Associate Director, Editing Technologies at Editas Medicine
    See Abstract
    Though AsCas12a fills a crucial gap in the current genome editing toolbox, it exhibits relatively poor editing efficiency, restricting its overall utility. In collaboration with Integrated DNA Technologies, we showed that this engineered variant we refer to as AsCas12a Ultra, increased editing efficiency to nearly 100% at all sites examined in HSPCs, iPSCs, T cells, and NK cells. We showed that AsCas12a Ultra maintains high on-target specificity thereby mitigating the risk for off-target editing and making it ideal for complex therapeutic genome editing applications. We achieved simultaneous targeting of three clinically relevant genes in T cells at >90% efficiency and demonstrated transgene knock-in efficiencies of up to 60%. We demonstrate site-specific knock-in of a CAR in NK cells, which afforded enhanced anti-tumor NK cell recognition, potentially enabling the next generation of allogeneic cell-based therapies in oncology. AsCas12a Ultra is an advanced CRISPR nuclease with significant advantages in basic research and in the production of gene edited cell medicines.
    ? 基因和細胞治療

    ? 蛋白工程

    ? 免疫治療

    ? 生物技術

    ? 疫苗開發

    ? 科研進展

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