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CRISPR/Cas9 in drug discovery: Applications in target discovery, validation, and hit screening

This webinar is brought to you by the Science/AAAS Custom Publishing Office

CRISPR/Cas9 in drug discovery: Applications in target discovery, validation, and hit screening

Recorded 11 May 2016



The CRISPR/Cas9 system allows for unprecedented ease and control when editing the genome. Its potential impact on drug discovery is vast, including enabling gene and cell replacement therapies, identifying novel drug targets through functional genomic screens, and simplifying the production of disease models using permanent knockouts for validating therapy targets and testing drug efficacy. But in practical terms, how is CRISPR/Cas9 currently being applied, and where might the future challenges and pitfalls be? Furthermore, how do assays based on the new CRISPR/Cas9 technology compare with current screening methodologies, particularly those using small interfering RNA (siRNA)?

During this webinar, our expert panel will address:

  • Areas of the drug discovery process where gene editing will have the most immediate and long term impact
  • The current and future role of CRISPR/Cas9 in target discovery, hit identification, and lead optimization (compared with siRNA screening)
  • The challenges that CRISPR/Cas9 brings to screening and assay development, particularly with respect to cell analysis
  • The importance of carrying out binding studies following gene editing.

The presentations will be followed by a live Q&A session with the online audience.

The webinar will last approximately 60 minutes and will be followed by a live webchat (see below).

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Speaker bios

Lorenz Mayr, Ph.D.

Cambridge, U.K.

Dr. Mayr is vice president, Reagents & Assay Development (RAD), with global responsibility for generation of biological reagents and assay development activities at AstraZeneca Pharmaceuticals in Cambridge, United Kingdom. His responsibilities include the generation of proteins and cell lines for hit-finding, hit-to-lead, and lead optimization activities including structure and biophysics activities across all therapeutic areas, as well as the generation of tool antibodies, transgenic animals, stem cells, and primary cells as tools for target validation studies and lead optimization programs. His department in the United Kingdom (Cambridge and Alderley Park) and Sweden (Gothenburg) is responsible for assay development activities for biochemical, cell-based, and phenotypic assays for all therapeutic areas at AstraZeneca. The current research interests of the RAD Department concern precise-genome editing (PGE), transcriptomics, secretomics, high-content biology, novel antibody scaffolds and affinity reagents, next-generation sequencing (NGS), and the use of these technologies in complex 3D cellular models/organoids for target validation and lead optimization. Dr. Mayr completed his Ph.D. in biochemistry and biophysics at the University of Bayreuth in Germany and his postdoctoral training in the same subject area at the Massachusetts Institute of Technology in Cambridge, Massachusetts. He worked for Bayer Pharma AG in Leverkusen and Wuppertal, Germany and for Novartis Pharma AG in Basel, Switzerland before joining AstraZeneca.

Ralph Garippa, Ph.D.

Memorial Sloan Kettering Cancer Center
New York, NY

Dr. Garippa is the Director of the RNAi and Gene Editing (CRISPR-Cas9) Core Facility at the Memorial Sloan Kettering Cancer Center. His group develops protocols in the design and execution of loss-of-function screens and provides consultation in the areas of short hairpin RNA (shRNA) and single guide RNA (sgRNA) library design, vector construction, CRISPR-Cas9 knockout protocols, and data analysis. They also design and run biochemical and cell-based small molecule screens using a 350,000 compound library, and the core is fully equipped to perform high-speed, automated fluorescent microscopy for high-content screening. Dr. Garippa holds a Ph.D. in pharmacology from Columbia University in New York City (where he studied with Drs. Fred Maxfield and Tim McGraw) and a B.A. degree in biology from Fairleigh Dickinson University in New Jersey.

Sean Sanders, Ph.D.

Washington, DC

Dr. Sanders did his undergraduate training at the University of Cape Town, South Africa, and his Ph.D. at the University of Cambridge, UK, supported by the Wellcome Trust. Following postdoctoral training at the National Institutes of Health and Georgetown University, Dr. Sanders joined TranXenoGen, a startup biotechnology company in Massachusetts working on avian transgenics. Pursuing his parallel passion for writing and editing, Dr. Sanders joined BioTechniques as an editor, before joining Science/AAAS in 2006. Currently, Dr. Sanders is the Director and Senior Editor for Custom Publishing for the journal Science and Program Director for Outreach.

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