Speakers

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Paul Diehl, Ph.D.
Chief Operating Officer
Cellecta

Dr. Paul Diehl joined Cellecta, Inc. in July 2010 where he applies his 19 years of experience in commercial biotechnology and formal background in biochemistry and molecular biology to developing and expanding the company’s commercial and collaborative partnerships and interactions. Prior to joining Cellecta, Dr. Diehl held various marketing and business development positions related to developing, launching, and supporting research focused life science products and technology at B-Bridge International, Arcturus Bioscience, Agilent Laboratories, Display Systems Biotech, and Clontech Laboratories. Dr. Diehl received his B.A. in Biology from LaSalle University in Philadelphia in 1988 and his Ph.D. in Biochemistry from Washington State University in Pullman, WA in 1993. As a graduate student he analyzed the activity of E. coli isocitrate lyase using site-directed mutagenesis.

Day Two

Thursday 13th December 2018

10:10 am | Combining CRISPR Editing and Cell Barcoding to Elucidate Gene Function and Advance Target Discovery

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.

Radu Rapiteanu
Investigator, Functional Genomics
GSK

Scientific Investigator in Functional Genomics (Ph.D., University of Cambridge) with expertise in developing and implementing pooled and arrayed CRISPR-based high-throughput screens in human primary cells and disease models. Interested in building/improving pipelines for CRISPR genome editing at scale coupled to single cell transcriptomics readouts. Experience in hit validation and mechanistic characterization of targets/pathways. Passionate about accelerating the translation of research into novel therapies.

Day Two

Thursday 13th December 2018

12:25 pm | Arrayed and pooled CRISPR screening in a complex primary cell model of the human airway

Abraham Scaria
VP, Head of Ophthalmology
Casebia Therapeutics

Dr. Abraham Scaria is Vice President, Head of Ophthalmology at Casebia Therapeutics. Dr. Scaria has more than 20 years of experience in gene therapy, ranging from discovery research to clinical trials. For much of his career, Scaria has specialized in viral vectors and ocular gene therapy, having served as Senior Scientific Director of Gene Therapy and Ophthalmology at Genzyme, and subsequently Sanofi Genzyme. He most recently served as Head of Gene Therapy Research at Sanofi. He earned his Ph.D. in biochemistry and molecular biology from Indiana University School of Medicine followed by postdoctoral research at St. Louis University School of Medicine and University of Washington in Seattle.

Day Two

Thursday 13th December 2018

2:00 pm | Viral Engineering Strategies to Alleviate Barriers Towards Successful Delivery: A Specific Focus on the Retina

Beeke Wienert
Postdoctoral Researcher
Innovative Genomics Institute, Conklin & Corn labs, Gladstone Institutes, San Francisco

Beeke earned her PhD in Molecular Genetics in 2016 from the University of New South Wales in Sydney, Australia, studying hemoglobinopathies and therapeutic upregulation of fetal hemoglobin using CRISPR. Since 2017 she is a postdoctoral researcher at the Innovative Genomics Institute in the Bay Area and specializes in CRISPR-Cas9 off-target discovery and DNA repair as part of Jacob Corn’s team. In 2018 she joined Bruce Conklin’s team at the Gladstone Institutes in San Francisco to study rare genetic disease in patient-derived stem cells. Her work focuses on the development of safe CRISPR reagents for therapeutic genome editing and new methods for off-target detection in cells and tissues.

Day One

Thursday 13th December 2018

12:10 pm | Unbiased Detection of CRISPR Off-targets in Vivo Using Discover-Seq

Ben Kleinstiver
PhD, Principal Investigator Massachusetts General Hospital Instructor
Harvard Medical School

Ben Kleinstiver is a Principal Investigator at the Massachusetts General Hospital (MGH) and Instructor at Harvard Medical School (HMS). His research interests are broadly focused on developing and improving genome editing technologies for biomedical research and clinical applications. The work in his laboratory is focused on using protein engineering methods to evolve genome editing nucleases that have enhanced properties. His research group is actively developing CRISPR enzymes with improved on-target activities, genome-wide specificities, and targeting ranges, and are also explore the feasibility of applying these engineered technologies for the treatment of genetic diseases.

Day One

Thursday 13th December 2018

2:40 pm | Engineering CRISPR Enzymes to have Superior Properties

Bio-Rad Representative

Day One

Thursday 13th December 2018

11:40 am | Session details to be confirmed. Please check back for speaker and presentation information

George M. Church, PhD.
Professor of Genetics Department of Genetics, Blavatnik Institute
Harvard Medical School

George M. Church, PhD ’84, is professor of genetics at Harvard Medical School, a founding member of the Wyss Institute, and director of PersonalGenomes.org, the world’s only open-access information on human genomic, environmental, and trait data. Church is known for pioneering the fields of personal genomics and synthetic biology. He developed the first methods for the first genome sequence & dramatic cost reductions since then (down from $3 billion to $600), contributing to nearly all “next generation sequencing” methods and companies. His team invented CRISPR for human stem cell genome editing and other synthetic biology technologies and applications – including new ways to create organs for transplantation, gene therapies for aging reversal, and gene drives to eliminate Lyme Disease and Malaria.  Church is director of IARPA & NIH BRAIN Projects and National Institutes of Health Center for Excellence in Genomic Science.  He has coauthored 450 papers, 105 patents, and one book, “Regenesis”. His honors include Franklin Bower Laureate for Achievement in Science, the Time 100, and election to the National Academies of Sciences and Engineering.

Day One

Thursday 13th December 2018

1:40 pm | Multiplex Editing & Differentiation

Dan Tierno
Associate Director, Global Clinical Data Sciences and Analytics
Bayer

Mr. Dan Tierno has over 20 years of experience in pharmaceutical portfolio management, R&D and commercialization.  Mr. Tierno also specializes in cell and gene editing technologies, mHealth, and strategies for use of advanced analytics in clinical trials. Mr. Tierno has spoken on the following topics: training scientists for rare disease therapeutic development, impacts of genomics in clinical trial design for cell and gene therapies, and defining and generating value via portfolio management. In 2019, topics Mr. Tierno will be speaking on include innovative models for cell and gene therapy clinical trials, funding and payer models for novel therapeutics, and the relationship between STEM programs in early education and entrepreneurship. Mr. Tierno holds his B.S. in Nutrition Science from New York Institute of Technology, his Honors M.B.A. from the Zicklin School of Business at Baruch College, and his M.A. in Biotechnology from Columbia University.

Day Two

Thursday 13th December 2018

4:30 pm | Panel Discussion: Standardization and Reproducibility of CRISPR Protocols Across the Industry

David Alvarado
Associate Principal Scientist
Merck

Advances in gene editing technologies have provided new opportunities to interrogate global regulatory mechanisms in an unbiased manner. Genetic variants are frequently associated with elevated risk of complex human diseases; however, the mechanisms are often unknown. In order to develop targeted therapies, a better understanding of which pathway(s) are relevant for disease pathogenesis is needed. To explore modulators of genetic targets, we are using genome-wide pooled CRISPR screens to identify druggable regulators of gene expression levels and gain valuable insights into the genetic mechanisms underlying human disease.

Day Two

Thursday 13th December 2018

8:40 am | How CRISPR Based Screening Systems can be used in Parallel for Target Identification and Validation

Eric Paul Bennett
Associate Professor
University of Copenhagen

Eric P. Bennett has obtained both his master degree in Biochemistry and Molecular Biology (MSc) and doctoral degree (Dr.med) from the University of Copenhagen. His research efforts at the Copenhagen Center for Glycomics, University of Copenhagen, have been concentrated in the field of Glycobiology where he 2 decades ago cloned and identified more than 30 human glycosyltransferase encoded genes (glycogenes). For the last 9 years his efforts have been focused on ways of establishing precision-engineered isogenic cells lacking individual glycogenes. Using the recently emerged nuclease targeting technologies, such as ZFN, TALEN and CRISPR/Cas9 these efforts have among others led to establishment of novel methodologies that both improve the targeting and indel detection efficacy of precise gene targeting. The methods and principles developed show great translational potential into the biotech and therapeutic genome targeting space.

Day One

Thursday 13th December 2018

11:10 am | Fast and Accurate Quantification of On and Off-target CRISPR Genome Editing Outcomes

Garrett Rettig
Director of Molecular Genetics Product Development
IDT

Garrett has been in the Molecular Genetics Research department at Integrated DNA Technologies for the past 10 years. During that time, he has been a lead scientist on various RNA knockdown projects – particularly high-throughput screening of siRNAs and chemical modifications thereof. Recently, he has focused on improving methods and developing new technologies for CRISPR-mediated genome editing.

Day One

Thursday 13th December 2018

2:10 pm | Development of Novel Cas9 and Cas12a Mutants: Defining Editing Efficiency and Specificity

Guru Channabasavaiah
Associate Professor- Genetics, Director- Mouse Genome Engineering Core Facility
University of Nebraska Medical Center

Guru (CB Gurumurthy) is an Associate Professor of Developmental Neuroscience, Munroe Meyer Institute for Genetics and Rehabilitation at the University of Nebraska Medical Center. He is also the Director of the UNMC’s Mouse Genome Engineering Core Facility. His research interests are improving and development of novel genome engineering technologies. He is a co-developer of popular technologies such as Easi (Efficient additions with ssDNA-inserts)-CRISPR, CRISPR-first: PITT-next and GONAD (Genome Editing via Oviductal Nucleic Acids Delivery). Some of these technologies have already been adapted in many labs worldwide.

Day Two

Thursday 13th December 2018

3:00 pm | CRISPRing Made Easier to Create Animal Models for Basic and Drug Discovery Research

Horizon Representative

Day Two

Thursday 13th December 2018

9:10 am | CRISPR Screening on Primary T cells

Jesper Gromada
CSO
Exonics Therapeutics

Jesper Gromada joined Exonics Therapeutics as Chief Scientific Officer in 2018. He brings to the company more than 20 years of experience working in various roles at Novo Nordisk, Eli Lilly, Novartis and most recently at Regeneron Pharmaceuticals, where he served as Vice President. A major focus of Jesper’s work has been the development of therapeutics for patients with metabolic and skeletal muscle diseases. He has published more than 140 peer-reviewed manuscripts, including in high impact journals such as the New England Journal of Medicine, Cell, Science and Cell Metabolism, and has given numerous invited lectures nationally and internationally. Jesper holds a Ph.D. from University of Copenhagen, Denmark.

Day One

Thursday 13th December 2018

9:10 am | How Gene Editing Therapies Can Treat Genetic Neuromuscular Diseases

Jonathan Gootenberg
PhD McGovern Fellow
MIT

Jonathan Gootenberg draws from fundamental microbiology to engineer new molecular tools, which he applies to the study of aging. These tools, including the popular genome editing system CRISPR, allow for unprecedented manipulation and profiling of cellular states in the body, and have multiple applications in basic science, diagnostics, and therapeutics. Along with McGovern Fellow Omar Abudayyeh, Gootenberg uses gene editing, gene delivery, and cellular profiling methods to understand the changes that occur in the brain and other organs during aging, with the goal of generating new therapies for degenerative disease.

Day One

Thursday 13th December 2018

3:50 pm | Harnessing Novel CRISPR Systems for Genome Engineering and Human Health

Lucas Harrington
Co-founder, Chief Discovery Officer
Mammoth Biosceinces

Lucas co-founded Mammoth Biosciences while completing his PhD at UC Berkeley where he worked with Jennifer Doudna to explore the untapped diversity of CRISPR systems found in metagenomic data. Mammoth Biosciences is focused on creating a CRISPR application platform that embraces uses of CRISPR beyond simply gene editing. Mammoth is first focused on transforming disease detection, harnessing the precision and programmability of CRISPR. Prior to his PhD, Lucas attended Washington University in St. Louis.

Day Two

Thursday 13th December 2018

11:55 am | Discovering and developing next-generation CRISPR platforms for diagnostics

Megan van Overbeek
Associate Director of Functional Genomics
Caribou Biosciences, Inc.

Day One

Thursday 13th December 2018

9:40 am | Next-generation Gene Editing Technology for Allogeneic T Cell Therapeutics

Namjin Chung
Head of Functional Genomics
AbbVie

Namjin Chung is the founding head of Functional Genomics platform at AbbVie and a thought leader in the field of drug discovery and translational research empowered by CRISPR and other genomics technologies. He leads development of AbbVie’s CRISPR and functional genomics strategy, its implementation through building internal capabilities and leveraging external collaborations, and cross-functional projects across therapeutic areas including immunology, immuno-oncology, oncology and neuroscience. His team is responsible for developing and applying diverse CRISPR-based methods and reagents, sustained delivery of novel proprietary drug targets into R&D pipeline, functional validation of human genetic variants, and mechanistic and translational research for marketed and clinical-stage assets. Prior to AbbVie, Namjin worked at Merck and BMS with increasing level of organizational responsibilities. Namjin earned his PhD in Molecular Cancer Biology from Duke and completed his postdoctoral training at MIT.  

Day Two

Thursday 13th December 2018

5:20 pm | Chairs Closing Remarks

10:55 am | Synergy between CRISPR Functional Genomics and Human Genetics to Enhance Biopharmaceutical R&D Pipeline Success

8:30 am | Chair’s Opening Remarks

Neville Sanjana
Core Faculty Member and Assistant Professor
New York Genome Center and Departments of Biology and Neuroscience

Neville Sanjana, PhD, is a Core Faculty Member at the New York Genome Center and Assistant Professor in the Departments of Biology and of Neuroscience and Physiology at New York University. As a bioengineer, Dr. Sanjana creates new tools to understand the impact of genetic changes on the nervous system and cancer evolution. His lab has harnessed high-throughput gene editing to pinpoint which regions of the genome are involved in diverse diseases. He is a recipient of the NIH’s New Innovator Award and Pathway to Independence Award, the AAAS Wachtel Prize for Cancer Research, the DARPA Young Faculty Award, the Sidney Kimmel Scholar Award, the Melanoma Research Alliance Young Investigator Award, and the Brain and Behavior Research Foundation Leichtung Family Investigator Award. Dr. Sanjana holds a PhD in Brain and Cognitive Sciences from MIT, a BS in Symbolic Systems, and a BA in English from Stanford University.

Day Two

Thursday 13th December 2018

9:40 am | New Frontiers for Pooled Screens: Finding Regulatory Elements in the Noncoding Genome and Capturing Multi-Cell Interactions

Niren Murthy
Professor
UC Berkeley

Dr. Niren Murthy is a professor in the Department of Bioengineering at the University of California at Berkeley. Dr. Murthy received his Ph.D. from the University of Washington in Seattle in Bioengineering in 2001, and then did Postdoctoral research at U.C. Berkeley in Chemistry from 2001-2003. He started his academic career at Georgia Tech in 2003 and in 2012 moved back to U.C. Berkeley. Dr. Murthy’s laboratory is an interdisciplinary laboratory that focuses on the development of new materials for drug delivery and molecular imaging. Murthy laboratory has developed several new biomaterials and imaging agents, such as the maltodextrin based imaging agents, which are focused on improving the treatment and diagnosis of infectious diseases. In addition, Murthy laboratory has developed numerous reagents for detecting radical oxidants, such as the hydrocyanines.

Day Two

Thursday 13th December 2018

2:30 pm | Exploring Innovative Methods for Non-Viral In-Vivo Delivery

Wojtek Auerbach
ISTT Presdent, Senior Director Emeritus, Embryonic Stem Cells Technologies
Regeneron Pharmaceuticals Inc.

Dr. Wojtek Auerbach is a Senior Director Emeritus at Regeneron Pharmaceuticals responsible for Embryonic Stem Cell technologies. Since October 2017 he hold a position of president of ISTT.  He earned his MSc. Degree in the Department of Genetics at Warsaw University and PhD. At Institute of Biochemistry and Biophysics, Polish Academy of Science. Before joining Regeneron in 2001 he worked at Hospital for Sick Children Research Institute in Toronto, Canada, Skirball Institute of Biomolecular Medicine at New York University School of Medicine and Howard Hughes Medical Institute (HHMI) generating animal models of human genetic diseases such as Cystic Fibrosis, Fanconi Anemia or Huntington’s Disease.

Day Two

Thursday 13th December 2018

3:30 pm | Generation of humanized mouse models

Omar Abudayyeh
Independent MIT McGovern Fellow
McGovern Institute for Brain Research at MIT

I am currently an independent MIT McGovern Fellow at the McGovern Institute for Brain Research at MIT. Previously, I was an MD/PhD student at Harvard Medical School/Harvard-MIT Health, Sciences, and Technology program and completed my PhD in Feng Zhang’s lab at the Broad Institute of MIT and Harvard in 2018. I have had multiple fellowship positions, including Friends of the McGovern Institute Fellowship, Paul and Daisy Soros Fellowship, National Science Foundation Graduate Fellowship, National Defense Science and Engineering Graduate Fellowship, and the NIH F30 National Research Service Award. I am currently on leave from Harvard Medical School while pursuing my independent research activities. In 2012, I graduated with a S.B. degree in Mechanical Engineering and Biological Engineering from the Massachusetts Institute of Technology as a Henry Ford II Scholar and Barry Goldwater Scholar.

Day One

Thursday 13th December 2018

3:50 pm | Harnessing Novel CRISPR Systems for Genome Engineering and Human Health

PJ Brooks
Program Coordinator
NIH Common Fund Program on Somatic Cell Genome Editing

Philip John (P.J.) Brooks is a program director in the Office of Rare Diseases Research at NCATS.  He earned his Ph.D. in neurobiology from the University of North Carolina at Chapel Hill, followed by a postdoctoral fellowship at the Rockefeller University.  Prior to NCATS, he was an Intramural investigator at the NIH, studying rare neurologic diseases resulting from defective DNA repair.  He represents NCATS on the Trans-NIH Microbiome Working Group and Gene Therapy Working Group,  and is the coordinator of the NIH Common Fund Programs on Somatic Cell Genome Editing.  In addition to his other duties, he manages a portfolio of small business grants supporting platform approaches to the delivery of nucleic acid therapeutics.  Dr. Brooks interested in platform approaches and novel clinical trial designs to address multiple rare diseases in parallel, including gene therapy, genome editing, and therapeutics targeting molecular etiologies underlying multiple rare diseases.

Day One

Thursday 13th December 2018

4:20 pm | The NIH Common Fund Somatic Cell Genome Editing (SCGE) Program: Tools for Translation into the Clinic

Samantha Maragh
Leader, Genome Editing Program
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 Two

Thursday 13th December 2018

4:30 pm | Panel Discussion: Standardization and Reproducibility of CRISPR Protocols Across the Industry

Stuart Chambers
Senior Scientist
Amgen

Stuart Chambers is a Senior Scientist at Amgen managing the human pluripotent stem cell (hPSC) and bioassay technology team within the Genome Analysis Unit (GAU) located in South San Francisco. He uses cells derived from hPSCs across all three germ layers to conduct genome-wide screens for the purposes of biomarker analysis, target identification, and drug discovery. The GAU serves as a point of entry for technology development and adoption at Amgen. He oversees projects including in vitro assay development, human disease modeling, CRISPR editing of hPSCs, high-content and live cell imaging, 3D organoid culture, and the development of automation for iPSC culturing. As a Postdoctoral Associate in Lorenz Studer’s lab at Sloan-Kettering Institute in New York City, Stuart made a crucial discovery of how to optimally coax neural tissue from hPSCs

Day Two

Thursday 13th December 2018

4:00 pm | Industrializing iPSC CRISPR Editing and Disease Modeling

TJ Cradick
Head of Genome Editing
CRISPR Therapeutics

TJ Cradick, PhD has developed genome editing and gene therapy reagents and therapeutics for more than 15 years. Since 2012-13, CRISPR/Cas nucleases have been the focus (see publication list). Previously studied and published on TAL Effector Nucleases (TALENs), and Zinc Finger Nucleases (ZFNs).  Many of these studies in academia and industry included measuring and improving specificity.  TJ co-authored manuscripts detailing bioinformatics web tools: ZFN-Site, PROGNOS, SAPTA and COSMID (CRISPR off-target). TJ was Director of the Protein Engineering Facility at the Georgia Institute of Technology where the research included developing assays for CRISPR specificity and decreasing off-target events. TJ’s undergraduate degree is from MIT, Masters from UCSF and PhD from the University of Iowa.  

Day One

Thursday 13th December 2018

8:40 am | CRISPR Therapeutics Provides Clinical Insights into CTX001 for the Treatment of Sickle Cell Disease

Ultan McDermott
MB PhD Chief Scientist and Former Group Leader, Wellcome Sanger Institute
AstraZeneca, Honorary Faculty

Ultan McDermott became Chief Scientist at AstraZeneca in 2017 with a focus on drug resistance in cancer, after eight years at the Wellcome Sanger Institute (seven of those as group leader). Ultan continues to maintain strong working relationships with colleagues in the Cancer, Ageing and Somatic Mutation programme at the Sanger as a member of Honorary Faculty. He also holds an Honorary Consultant post in the Oncology department at Addenbrooke’s Hospital in Cambridge, where he treats colorectal cancer patients. Ultan joined the Sanger Institute in 2009 as a clinical research fellow and was appointed to the faculty as a Group Leader in 2010 with the award of a Cancer Research UK Clinician Scientist Fellowship. Previously he worked as a postdoctoral fellow with Jeff Settleman at Massachusetts General Hospital Cancer Center, Boston on high-throughput cancer cell line drug screens and resistance mechanisms. He is a Fellow of the Royal College of Physicians.

Day Two

Thursday 13th December 2018

11:25 am | How to Build a Streamlined Approach to CRISPR Screening