Gene editing partnerships to enhance drug pipeline productivity
In this pharma webinar we discuss how the decision was taken on a knock out approach to generate iPSC disease models to more efficiently and quickly validate two potential lung cancer targets. We also present considerations for undertaking successful cell engineering projects for high-content screening assay development and target validation.
Optimising target-based assays and streamlining hit-to-lead processes are key for a productive R&D drug pipeline. Researchers in both the pharmaceutical and biotech industries, as well as in academic environments, are increasingly searching for novel technologies and related services that enable them to both innovate and speed up their research pipeline. The creation of disease models for target validation and reporter cell lines for high-content screening assays are good examples of where one needs high-technology knowledge and cell engineering expertise to be able to efficiently create the ideal cell model in a reasonable time frame. Trying to have a grip on novel gene editing knowledge and hands-on experience is a time and cost consuming effort. For this reason, researchers are increasingly deciding to externalize that part of their work, in order to focus on target-specific research and other aspects of drug development.
Ronald Van Brempt – Staff physician, Pediatric Intensive Care Unit, Leiden University Medical Center (LUMC)
The need for knocking out two lung-regeneration genes in iPSC’s – Ronald addresses in this pharma webinar the need for “pure KO” primary cell lines in the exploration of the function of novel genes or the mechanism of action of novel proteins. The limitations of certain techniques (shRNA technology, immortalized cell lines) will be demonstrated using practical examples.
Ronald’s research focus at LUMC is Novel genes and proteins involved in lung regeneration. Ronald has previously trained at the University Hospitals of Leuven, Belgium, the Johns Hopkins Hospital Baltimore, and the Children’s Hospital Boston in the USA.
Mark Gerber, Ph.D. – Principal R&D Scientist, Advanced Genetics and Cellular Technologies Supervisor, Cell Design Studio Sigma Aldrich
Considerations for a successful cell engineering project – in this pharma webinar Mark presents considerations for undertaking successful cell engineering projects using examples for high-content screening assay development and target validation.
Mark joined Sigma-Aldrich in 2006, and has worked in the areas of biotherapeutic production, stem cell applications and gene regulation. In 2014, Mark was recruited to lead the Cell Design Studio team in the engineering of custom cell lines utilizing ZFN, CRISPR and shRNA technologies. Mark obtained his Ph.D. in Biochemistry and Molecular Biology from Saint Louis University School of Medicine where he used RNAi in Drosophila models to elucidate developmental and biochemical roles for RNA polymerase II-associated transcription factors. Following graduate school, Mark served as a Postdoctoral Fellow at Washington University in St. Louis where he investigated signalling pathways involved in the development of human meningioma.
*Cell Design Studio is a trademark of Sigma-Aldrich Co. LLC.
Supported by Sigma Aldrich
In late 2015, Sigma-Aldrich joined forces with Merck Millipore. Their combined organisation, which operates as MilliporeSigma in the United States and Canada, is the life science business of Merck – and a global leader in the $125 billion life science industry. Their shared purpose is to solve the toughest problems in life science by collaborating with the global scientific community. Together, they offer a broad portfolio of more than 300,000 products, including many of the most highly respected brands in the industry.
Find out more: www.sigmaaldrich.com