The following topics were discussed:
- Details of carrying out CRISPR knockout, interference and activation screens
- Arrayed CRISPR screening
- Maximizing data analysis of CRISPR screens
- Applications in drug discovery
- Latest developments together with future directions in the field of CRISPR screening
Event participants were able to:
- Attend presentations by Horizon experts and KOLs
- Participate in Panel/Roundtable discussions on latest topics
- Have the opportunity to network with other participants
- Meet for 1:1 discussion with Horizon CRISPR screening experts
Presenters at the Boston, Cambridge Masterclass:
Professor Amit Choudhary
Assistant Professor of Medicine at Harvard Medical School and an Associate Biologist at Brigham and Women’s Hospital
Amit Choudhary is an Assistant Professor of Medicine at Harvard Medical School and an Associate Biologist at Brigham and Women’s Hospital. He performed his pre-doctoral studies in Chemistry at Indian Institute of Science‒Bangalore and doctoral studies in Biophysics with Prof. Ronald Raines at the University of Wisconsin‒Madison, where he elucidated a new force that is akin to the hydrogen bond in its quantum mechanical origin and widespread prevalence. In 2011, he was appointed a Junior Fellow of the Harvard Society of Fellows and was hosted by Prof. Stuart Schreiber at the Broad Institute. There, he shifted his research focus to beta-cell chemical biology and was named a Career Awardee at the Scientific Interface by the Burroughs Wellcome Fund. His laboratory now develops chemical technologies and studies exceptional organisms that survive conditions considered pathological to humans. The efforts of Choudhary laboratory have been recognized by Juvenile Diabetes Research Foundation’s Innovation Award, Burroughs Wellcome Fund’s Career Award at the Scientific Interface, NIH Director’s Transformative Research Award, and DARPA’s SAFE GENES award.
Dr. Benedict Cross
R&D Manager at Horizon Discovery
As a Geneticist from the UK, Ben completed his PhD at the University of Manchester and trained as a post-doctoral scientist at the University of Cambridge studying reverse chemical genetic screening in the unfolded protein response. His research identified and defined the mechanism of a new tool compound against a key proteostasis kinase. Ben joined Horizon in 2013 to expand and advance Horizon’s functional genomic screening capability and to lead a major research alliance in synthetic lethal target discovery. Ben led the establishment of pooled CRISPR-Cas9 screening at Horizon and developed the Dual Screening methodology with CRISPRi and CRISPRa to target complex mechanism of action analysis. Ben now heads up the teams applying these functional genomic screening platforms in target discovery, target validation and other research programmes.
Dr. James Goldmeyer
Product Manager at Horizon Discovery
James has been with Dharmacon, a Horizon Discovery Group Company for over nine years and currently serves as the Product Manager for the Horizon vector-based reagents portfolios, including all shRNA, sgRNA, and gene expression platforms. James works closely with an expert group of R and D scientists on innovative tools and services for gene modulation and engineering. He has previously been in roles serving as a technical expert and educator around the complete product range of RNAi, gene-expression, and Gene editing portfolios. As such, he is intimately familiar with the technical concepts and experimental considerations necessary for successful application of a wide range of products.
Dr. Carlos Le Sage
Senior Scientist II, Functional Genomic Screening Platform at Horizon Discovery
Le Sage’s research focusses mainly on discovering novel resistance and sensitivity mechanisms of cancer cells exposed to newly developed compounds. He has many years of experience in the development and use of functional genomic techniques, initially with RNAi and more recently with CRISPR gene modifying and modulating technologies for genome-wide loss- and gain-of-function screening.
Since joining Horizon Discovery, Le Sage has engaged in a large number of client-based projects centered on functional genomics. He received the Trailblazer Award for his involvement in developing and providing CRISPR activation and interference genome-wide screen technologies as off-the-shelf services in 2017.
Prior to joining Horizon Discovery, Le Sage completed postdoctoral research with Steve Jackson at the Gurdon Institute in Cambridge. He holds a Ph.D. in biology from the Netherlands Cancer Institute through Leiden University.
Dr Eric Lightcap
Scientific Fellow at Takeda
I lead Oncology Molecular Genomics (OMG), a data-driven and translationally focused team seeking to reduce the time it takes for valid concepts to achieve clinical utility. We are dedicated to high quality generation and interpretation of comprehensive data experiments ranging from genome-wide to single-cell to enable drug discovery and development in immuno-oncology and immunology from target identification through early clinical proof of concept.
The OMG Group works collaboratively with the Oncology Drug Discovery Unit, Immunology Unit and Translational Research and Early Clinical team to inform target identification, indication selection, target validation, compound mechanism of action, combination strategy, and discovery of pharmacodynamic and predictive biomarkers.
Professor Ophir Shalem
Assistant Professor at the Department of Genetics at the University of Pennsylvania
During his postdoc in Prof. Feng Zhang’s lab, Ophir was one of the pioneers in developing genome-wide CRISPR screens. His lab combines functional genomics, technology development and mechanistic studies to study protein quality control pathways and cellular neurodegeneration.
Dr. Martin Kampmann
Assistant Professor in the UCSF Department of Biochemistry and Biophysics and the Institute for Neurodegenerative Diseases
Dr. Kampmann is an Assistant Professor in the UCSF Department of Biochemistry and Biophysics and the Institute for Neurodegenerative Diseases, and an Investigator at the Chan Zuckerberg Biohub. He received his BA and MA in Natural Sciences (Biochemistry) from Cambridge University and his PhD from the Rockefeller University, where he used biophysical approaches to characterize the architecture and dynamics of the nuclear pore complex with Dr. Günter Blobel. As a postdoctoral fellow in Dr. Jonathan Weissman’s group at UCSF, Dr. Kampmann spearheaded the development of a functional genomics platform that makes it possible to robustly identify human genes relevant to a cellular process of interest, and to elucidate cellular pathways and networks using systematic genetic interaction maps. One important application of the screening platform is the identification of genes that control the sensitivity of cells to a given drug. This approach can identify the relevant cellular targets of new compounds, predict drug-resistance mechanisms, and guide the rational development of combination therapies. The platform incorporates CRISPR interference and CRISPR activation screening to enable genome-wide gain- and loss-of-function screens. In his independent group, Dr. Kampmann implemented this CRISPRi/a platform in human iPSC-derived cell types (including neurons and other cell types). This approach enables the dissection of cellular and molecular mechanisms of human diseases, with a focus on neurodegenerative diseases. Genetic modifier screens in patient iPSC-derived cells and CRISPR-corrected isogenic controls enable the elucidation of the mechanisms of disease-associated genes, and the identification of therapeutic strategies. An important focus is on protein homeostasis in the context of disease, including the prion-like propagation of protein aggregation in neurodegenerative diseases such as Alzheimer’s Disease and Parkinson’s Disease. Dr. Kampmann has received several highly prestigious awards, including the NIH Director’s New Innovator Award (2015), the Paul G. Allen Family Foundation’s Allen Distinguished Investigator Award (2015), and the NIH Pathway to Independence Award (2014). The Kampmann lab is affiliated with the Innovative Genomics Initiative and the Paul F. Glenn Center for Aging Research at UC Berkeley and UCSF.
Dr. Traver Hart
Assistant Professor in the Department of Bioinformatics and Computational Biology at the University of Texas MD Anderson Cancer Center
Dr. Hart earned his Ph.D. in Cell and Molecular Biology in 2008 in the lab of Dr. Edward Marcotte at the University of Texas at Austin. He later joined the laboratory of Dr. Jason Moffat at the Donnelly Centre of the University of Toronto as a postdoctoral fellow to study functional genomics and genetic interactions in cancer, where he helped refine CRISPR/Cas9 technology for screening cancer cells and identifying genotype-specific therapeutic targets. In his own lab, he is extending these approaches to understand the tissue- and genotype-driven plasticity of genetic interaction networks in human cells.
Senior Product Manager at Horizon Discovery
Louise Baskin is a Senior Product Manager at Horizon Discovery with responsibility for Dharmacon synthetic RNA products. She joined Dharmacon RNA Technologies in 2005 and has since led the development and commercial launch of multiple genome-wide siRNA, microRNA, and CRISPR-Cas9 product lines, including specificity-enhanced siRNA, self-delivering siRNA, and the first genome-wide synthetic CRISPR RNA collection. She works closely with a respected team of R&D scientists to develop and expand Dharmacon tools and services for gene modulation. Louise studied biology at Creighton University and received her Master of Science degree in Molecular Biology and Genetics from Northwestern University.
CEO of ERS Genomics
Eric Rhodes recently joined ERS from Horizon Discovery where he held the role of Senior Vice President of R&D and Chief Technology Officer and was responsible for commercializing Horizon’s genome editing technologies as well as overseeing research and development. Over the course of his career he has held various positions focused on gene editing technologies including the positions of Director Business Development at Sigma-Aldrich and Vice President Business Development & Alliance Management at Sangamo Biosciences where he was responsible for commercializing gene-editing technologies and executed licensing deals with more than 25 major biotechnology and pharmaceutical companies. Eric holds a degree in microbiology and immunology from the University of California, Berkeley.
Dr. Benedict Cross (Horizon Discovery) also presented at the San Diego Masterclass.