February 26-28, 2018

Boston, USA

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Speakers

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Kevin Holden
Head of Synthetic Biology
Synthego

Kevin Holden is Head of Synthetic Biology at Synthego in Redwood City, California where he is responsible for integrating synthetic biology workflows, such as CRISPR genome engineering, into novel engineering and automation platforms.  He is also responsible for overseeing academic and industrial collaborations with key opinion leaders within the CRISPR community. He has over 10 years of biotechnology experience that includes synthetic biology, engineering metabolic pathways in microbes and collaborative research.  Kevin earned his PhD in Microbiology from University of California, Davis.

Daniel G. Anderson
Assistant Professor
MIT

Dr. Daniel G. Anderson is a Professor at the Massachusetts Institute of Technology, and a member of the Department of Chemical Engineering, the Institute of Medical Engineering and Science, and the David H. Koch Institute for Integrative Cancer. He received his PhD in molecular genetics from the University of California at Davis. At MIT, he pioneered the use of robotic methods for the development of smart biomaterials for drug delivery and medical devices. His work has resulted in the publication of over 350 papers, patents and patent applications.

Day One

Monday February 26th, 2018

09.10 | In Vivo Genome Editing with CRISPR/Cas9 Nano Formulations

Alexander Marson
Assistant Professor
University of California

Alex Marson completed medical school at Harvard, PhD training at the Whitehead Institute/MIT with Richard Young and Rudolf Jaenisch, Internal Medicine residency at the Brigham and Women’s and clinical training in Infectious Diseases at UCSF. He was a UCSF Sandler Faculty Fellow from 2013–2016. Marson is now an assistant professor in the UCSF Department of Microbiology and Immunology, with joint appointments in the Department of Medicine and the Diabetes Center. He is also affiliated with the UCSF Helen Diller Cancer Center and the Innovative Genomics Initiative (IGI). His lab integrates systems-scale investigations of human T cell circuitry with functional perturbation studies, including genome editing in primary T cells.

Day One

Monday February 26th, 2018

09.40 | Engineering T Cell Circuitry

Devon Shedlock
Vice President, Preclinical Development
Poseida

Devon J. Shedlock, Ph.D., is Director of Immuno-Oncology at Poseida Therapeutics.  Dr. Shedlock has extensive experience in the field of T cell immunology and was formerly an Adjunct Assistant Professor of Pathology & Laboratory Medicine at the Perelman School of Medicine and the Associate Director of the T-Cell Engineering Laboratory that is part of Carl June's group at the University of Pennsylvania.  Prior to his work developing engineered T lymphocyte-based therapies for treating cancer, he led a team in the development of DNA vaccines against numerous pathogens from the preclinic to clinical trials including those targeting the human herpes viruses and a vaccine for Ebola.  He also established numerous animal models for evaluation of vaccine immunogenicity and efficacy, molecular adjuvants, genetic optimization strategies, and advanced delivery technologies.  Prior to this work he studied the mechanisms of antibody-mediated neutralization of Ebola as a Postdoctoral Fellow at the Vaccine Research Center, National Institutes of Health.  Dr. Shedlock received his PhD from the University of Pennsylvania investigating the role of helper T cells in the generation of immunity and was awarded the Saul Winegrad Award for exceptional dissertation and significant contribution to biomedical science.  

Guru Channabasavaiah
Director
UNMC

Janice Chen
Graduate Student
Jennifer Doudna's Lab, Berkeley

I am a graduate student in Jennifer Doudna’s lab at the University of California, Berkeley. My thesis work investigates the relationships between structure, dynamics and function of RNA-guided interference proteins from class 2 CRISPR-Cas systems. These proteins are critical for antiviral defense in bacterial host immunity, and many have also been harnessed for genome engineering applications. I am particularly fascinated by how Cas9 and related class 2 CRISPR interference proteins integrate many functions as single, multi-domain endonucleases. Towards that interest, my research addresses how Cas9 conformational changes couple target recognition and cleavage, and how nuclease specificity is both naturally achieved and artificially improved through protein engineering. I am also broadly interested in the convergent evolution of DNA target cleavage mechanisms across the class 2 CRISPR-Cas universe.

Cédric Cleyrat
Assistant Research Professor & Genome Editing Consultant
UNM Cancer Centre

Cédric is an experienced cellular-molecular biologist focused on discovering unique protein signaling and trafficking pathways with the goal to develop novel therapeutic approaches in the field of hematologic malignancies. Cédric holds two M.Sc. in Biotechnology Engineering and Molecular-Cellular Biology. He obtained his Ph.D. in Hematology-Oncology from the University of Nantes (France), studying the inflammatory profile of hematopoietic progenitors in myeloid cancers. He then received a postdoctoral fellowship from the University of New Mexico Systems Biology Center (the STMC) to study the high affinity receptor for IgE (FcεRI) and its implication in allergies, asthma and mast cell diseases, using cutting-edge imaging technologies. As a faculty at the University of New Mexico Comprehensive Cancer Center, Cédric has integrated CRISPR-Cas9 genome editing to his workflow to generate unique reagents to study intracellular trafficking and signaling networks in myeloid malignancies while also developing novel CRISPR-based CAR therapeutic tools.

Ben Kleinstiver
Instructor
Harvard Medical School

Ben Kleinstiver is an Instructor in Keith Joung’s group at the Massachusetts General Hospital (MGH) and Harvard Medical School (HMS). His research interests are broadly focused on developing and improving genome editing techniques. With a background in protein engineering, he is interested evolving highly targetable nucleases that have enhanced properties. His current work is focused on optimizing, altering, and defining the specificities of CRISPR-Cas nucleases. Ben completed his Ph.D. at the University of Western Ontario, and is currently a Banting Postdoctoral Fellow and a Charles A. King Postdoctoral Fellow at MG.

Brad Merrill
Associate Professor
University of Illinois at Chicago

For about a decade, Brad Merrill's lab  at University of Illinois at Chicago has been using mutations and mutagenesis to examine developmental biology and stem cell biology based questions using the mouse and mouse embryonic stem cells as model systems. His group has heavily used CRISPR-Cas9, and is interested in elucidating how the Cas9 interfaces with the biology of the systems it is used in. Brad also established and directs the Genome Editing Core at UIC, which provides broad Cas9-based genome editing services and DNA sequencing services to the research community.

Dan Tierno
Strategic Implementation Manager
Bayer

Samira Kiani
Assistant Professor
Arizona State University

Samira Kiani is an Assistant Professor in the school of biological and health systems engineering at Arizona State Univeristy. She completed her Postdoctoral training in the center for Synthetic Biology at Massachusetts Institute of Technology, where she worked on developing synthetic gene circuits to reprogram the function and behavior of mammalian cells based on the Clustered Regularly Interspaced short Palindromic Repeats (CRISPR)/Cas9 technology. Her lab is interested in developing synthetic gene circuits using the Cas9/gRNA engineering strategies to edit/modulate endogenous genes or transgenes in human cells towards reprogramming cellular fate/function. She is the recipient of DARPA Young Faculty award for developing synthetic biology tools for gene therapies.

Abraham Scaria
Vice President & Head of Ophthalmology
Casebia

Dr. Scaria obtained his Ph.D. in Molecular Biology from Indiana University School of Medicine in Indianapolis. He did postdoctoral training with Dr. William Wold at St. Louis University School of Medicine working on RNA splicing mechanisms in adenoviruses and virus-host interactions. Dr. Scaria then worked with Dr. Mark Kay as a Senior Fellow at University of Washington School of Medicine in Seattle before joining Genzyme Corporation to work on gene therapy for Cystic Fibrosis. At Genzyme and later at Sanofi-Genzyme, Dr. Scaria held various positions with increasing responsibilities for two decades finally heading up both the gene therapy research group and the ophthalmology research efforts. Dr. Scaria is currently VP & Head of Ophthalmology at Casebia Therapeutics where he is developing gene-editing based therapies for ocular genetic diseases.

Day Two

Tuesday February 27th, 2018

09.40 | Gene Delivery & Gene Editing in the Retina

Ruwanthi Gunawardane
Director, Stem cells and Gene editing
Allen Institute

Ruwanthi (Ru) Gunawardane is the director of stem cells and gene editing at the Allen Institute for Cell science, where a multi-disciplinary team science approach is used to study the fundamentals of cell behavior. Ru leads a group of researchers creating a collection of highly quality gene edited stem cell lines that are used by the institute to study cell organization and activities through live cell imaging and are also made available to the research community. Ru joined the Allen institute after spending 5 years at Amgen where she developed assays to screen for novel therapeutics in oncology, inflammation, and cardiovascular diseases and help advance the early drug discovery pipeline. Prior to Amgen, Ru worked at Ambit Biosciences where she was part of a small team of scientists that identified and characterized AC220, a potent FLT3 kinase inhibitor that is currently in phase 3 trials for AML.

Day Two

Tuesday February 27th, 2018

09.10 | Using CRISPR/Cas9 to Illuminate Stem Cell Organization and Dynamics

Matthias Heidenreich
Research Scientist

Matthias Heidenreich studied Molecular Medicine at the University- and Max Planck Institute of Immunobiology & Epigenetics of Freiburg, Germany. He earned his doctorate with the Freie Universität and the Max-Delbrück-Center for Molecular Medicine in Berlin where he worked on neuronal KCNQ potassium channels. As postdoctoral research fellow in the laboratory of Feng Zhang at Broad Institute of MIT and Harvard he focused on the development of new genome engineering and single cell sequencing technologies for studying the function and dysfunction of the brain. He is now Research Scientist at Vertex Pharmaceuticals in Boston.

Day Two

Tuesday February 27th, 2018

16.15 | Gene Editing By CRISPR-Cas & Cpf1 In The Mouse Brain

Andrew Kernytsky
Head of Computational Biology
CRISPR Therapeutics

Day Two

Tuesday February 27th, 2018

16.45 | Safety Package and the Methods for Progression to CRISPR Clinical Trials

Danilo Maddalo
Laboratory Head
Novartis

Danilo Maddalo obtained a degree in Pharmaceutical Chemistry with honours from the University of Naples (Italy) ‘Federico II’ before moving to Karlsruhe (Germany) where he carried his PhD studies in Biochemistry and Molecular Biology (with honours) focusing on the role of molecular chaperones in cancer. Thereafter he was awarded a grant as Young Investigator from the Helmholtz Society to investigate the impact of cell stress on tumour growth and response to chemotherapy. To further specialize in mouse modeling, he moved to New York City (USA) at Memorial Sloan Kettering Cancer Center where he established a model of chromosomal rearrangements using the CRISPR/Cas9 technology. Dr Maddalo has now moved to the Novartis Institutes for Biomedical Research in Basel (Switzerland) to focus on mouse modeling of disease for drug discovery and target identification.

Samantha Maragh
Human Geneticist & Molecular Biologist
NIST

Samantha leads NIST's programs supporting genome editing technologies. She heads the launching NIST led Genome Editing Consortium which is a public private partnership to address pre-competitive measurement and standards needs for the genome editing field.  Her focus areas are measurement confidence and technology development for genome editing, bioassay and biomarker validation, with applications in engineering biology, precision/regenerative medicine, and cancer biology.  Samantha also represents the U.S. as a technical expert to the International Standards Organizations Technical Committee on Biotechnology (ISO TC276) representing the interests and expertise of the U.S. on standards relating to nucleic acids measurements. Samantha received a B.S. Cellular & Molecular Biology from Loyola University in Baltimore, a M.S. in Biotechnology from Johns Hopkins University in the area of molecular targets and drug discovery, and a Ph.D. in Human Genetics & Molecular Biology from the Johns Hopkins School of Medicine, McKusick-Nathans Institute of Genetic Medicine.

Day One

Monday February 26th, 2018

15.10 | How do you know? Measurements and Standards to Support Confidence in Genome Editing

Daniel Dever
Instructor
Stanford

Daniel Dever is a Research Instructor in the laboratory of Dr. Matthew Porteus at Stanford University, in the Department of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine. During his postdoctoral work in the Porteus group, he (with others) developed a CRISPR/Cas9-based beta-globin gene targeting in CD34+ hematopoietic stem cells as a potential therapeutic strategy to treat severe sickle cell disease. Dr. Dever (along with his collaborators) has now successfully used this methodology to efficiently target >15 genes in primary blood cells that are associated with hematopoiesis, hematopoietic genetic diseases, hematopoietic malignancies, or safe harbor sites. Dr. Dever's primary research interests are to continue to leverage CRISPR/Cas9-based genome editing technologies to study the molecular mechanisms of gene targeting in human hematopoietic stem cells with the ultimate goal of optimizing and further developing novel cell and gene therapies for disease of the blood and the immune system.

T.J. Craddick
Head of Genome Editing
CRISPR Therapeutics

Blythe Sather
Associate Director
Juno

Dr. Blythe Sather received her PhD from the University of Washington, Department of Immunology in 2007. For her thesis, she focused on understanding the role of homing receptors on the function of regulatory T cells in the laboratory of Dr. Danial Campbell at the Benaroya Research Institute in Seattle. She then moved to do her postdoctoral work at Seattle Children’s Research Institute in the laboratory of Dr. David Rawlings, where she focused on developing lentiviral gene therapies for primary immunodeficiency disorders. During this time, she lead a team who did ground breaking work using megaTAL nucleases to insert chimeric antigen receptors (CARs) via gene editing-mediated homologous recombination into the CCR5 locus of primary T cells for HIV therapy. In 2014, her desire to bring these types of therapies to the clinic on a larger scale lead her to move to Juno Therapeutics to help develop their research division. Since at Juno, she been instrumental in building their CAR T cell development platform, leading the multiple myeloma CAR T cell program, as well leading the collaboration with Editas medicine to bring CRISPER-mediated gene editing to CAR and TCR T cell products. She is currently an associate director in the early pipeline group of the research department and Juno.

Day Two

Tuesday February 27th, 2018

10.10 | Using CRISPR to Modulate Immunosuppressive Pathways in CAR T Cell Products

Jack Heath
Principal Research Associate
Editas

Shantanu Kumar
Senior Scientist
Gene Editing

Shantanu completed his PhD in Microbiology in 2004. During his postdoctoral work at The Scripps Research Institute (TSRI) and University of California San Diego (UCSD), he received training in the area of gene therapy, epigenetics, cancer biology, and stem cell biology. Since past 4 years, his focus has been on genome editing technology (CRISPR/Cas9) and validation of engineered cell lines using advanced technologies. In addition to R&D work, he also leads first genome editing training course work at EMBL, Heidelberg, Germany. At Juno Therapeutics, he is working with research, analytical and process development (AD&PD) teams to integrate T cells editing at manufacturing scale for early phase clinical trials.

Jonathan S. Gootenberg
Ph.D. Candidate
Broad Institute of MIT and Harvard

Jonathan S. Gootenberg is currently a Ph.D. candidate in the Harvard Systems Biology program, co-advised by Feng Zhang and Aviv Regev of the Broad Institute of MIT and Harvard. Jonathan's research combines computational and molecular approaches to discover and characterize new biological tools, with a specific focus on CRISPR/Cas proteins. These tools have diverse basic science and translation applications, including nucleic acid detection, genome-scale screening, and live-cell imaging.

Omar O. Abudayyeh
Ph.D. Student
MIT

Omar O. Abudayyeh is an M.D.–Ph.D. student in the Harvard–Massachusetts Institute of Technology (MIT) Health Sciences and Technology program. In the laboratory of Feng Zhang at the Broad Institute of MIT and Harvard, Cambridge, Massachusetts, USA, his doctoral research focuses on the discovery and characterization of novel CRISPR proteins, such as Cpf1, C2c1 and Cas13a/C2c2, in bacteria for the purpose of expanding the genome-editing toolbox and studying mammalian biology. His recent work has focused on using the RNA-targeting CRISPR system Cas13 for building a transcriptome engineering toolbox for applications in diagnostics and therapeutics.  

Xiaobo Zhou
Assistant Professor
Harvard

Dr. Zhou is a principal investigator leading the Functional Genomics program in respiratory diseases at BWH/HMS, aiming to translate human GWAS discoveries to lung biology by multi-pronged approaches. Her group has pioneered functional variants identification in COPD (chronic obstructive pulmonary diseases) and asthma by interrogating chromatin structure and modification. Two major areas of research interest in her group include assigning causal genes to non-coding GWAS loci and developing in vitro and in vivo models to screen function of GWAS genes associated with COPD and asthma. Her recent work has also applied single cell RNA sequencing and genome-editing methods to determine roles of the GWAS genes in regulating inflammation and cellular signaling pathways. Before Dr. Zhou established Functional Genomics Laboratory in BWH, she obtained her Ph.D in Chinese Academy of Medical Science in Beijing and finished her post-doctoral training at Harvard Medical School. She currently has four NIH funded projects as the Principal Investigator or Director and another four collaborative projects as the co-Investigator.

Jon Terret
Head of Immuno/Oncology
CRISPR Therapeutics

Seshi Police
Head of AAV Delivery
CRISPR Therapeutics

Leon Song
Director
Genscript

Greg Gocal
EVP & CSO
Cibus

Shawn Shafer
Director of Advanced Genomics
Millipore Sigma

Day One

Monday February 26th, 2018

15.40 | Knockout Screening with the Sanger Whole Genome (Talk details Subject to Change) Arrayed CRISPR Libraries

Jason Potter
Senior Scientist
Thermo Fisher Scientific

Mark A. Behlke
Chief Scientific Officer
Integrated DNA Technologies