February 7-11, 2026
Thomas Michael Menino Convention & Exhibition Center (MCEC)
Boston, MA, USA
February 7-11, 2026
Thomas Michael Menino Convention & Exhibition Center (MCEC)
Boston, MA, USA
Drug attrition in clinical trials due to inaccurate preclinical target validation is a huge problem facing drug discovery. Drug candidates with adequate pharmacokinetics and safety margins still commonly fail, due to lack of efficacy. This suggests that often the ‘wrong’ target has been selected as the focus of the therapeutic program. Successful target validation will increasingly rely on the use of novel genetic and non-genetic tools including CRISPR and targeted protein degradation to disease relevant models. Genome editing tools such as CRISPR-Cas9 are reshaping what is possible within biological sciences. This is especially true for target validation and animal model generation. With genome editing tools, discovery scientists can knock out genes, add tags and mutate genes, all at the endogenous level. Furthermore, CRISPR enables bench-level access to a universe of targeted and genome-wide functional screens to understand gene function, unlock new drug targets, and deconvolute the mechanism of action of drug-like small molecules.
This course will introduce the state-of-the-art in genome editing, the current limitations and the step-by-step methodologies of how to apply these technologies to improve the drug discovery process, with an emphasis the on the selection of novel targets for drug development. We will also dive into the methods of conducting pooled CRISPR screens and the considerations for designing them. Finally, the course will discuss the practical approaches to target identification and validation through a modern toolbox of techniques that will increase the chances of success.
Scott T. Younger, PhD
Children’s Mercy Research Institute, Children’s Mercy Kansas City
Scott Younger is the Director of Disease Gene Engineering within the Genomic Medicine Center at Children's Mercy Kansas City. His laboratory is focused on dissecting the molecular mechanisms through which rare genetic variants identified in patients at Children's Mercy lead to disease. Younger joined Children's Mercy from the Broad Institute of MIT and Harvard where his group worked on the development of new methodologies to expand the utility of CRISPR-based genetic screens. Prior to working at the Broad Institute he completed his postdoctoral studies at Harvard University as an American Cancer Society Fellow. He holds a PhD in cell and molecular biology from UT Southwestern Medical Center. He also received an MS in biotechnology from the University of Texas at San Antonio and a BSI in bioinformatics from Baylor University.
Samuel A. Hasson, PhD
Voyager Therapeutics
Sam Hasson is currently an Associate Director at Voyager Therapeutics (Cambridge, Massachusetts). A major aim of his work is to develop novel applications of AAV-based gene therapy in the CNS to transform the treatment of unmet medical needs. Prior to joining Voyager in 2020, Hasson led groups within Amgen Neuroscience and Pfizer Neuroscience with a focus on the deconvolution of human disease genetics for drug target selection, employing genome editing technologies to enable this process. As a postdoc, he trained with Richard Youle and Jim Inglese at the National Institutes of Health.
Davide Gianni, PhD
AstraZeneca
Davide Gianni is Senior Director of Functional Genomics at AstraZeneca’s BioPharmaceuticals R&D, where he leads a team of scientists advancing new therapeutic opportunities across key disease areas.
Gianni joined AstraZeneca in 2015 from Boehringer Ingelheim, where he led oncology target discovery efforts. Earlier, he completed postdoctoral research at The Scripps Research Institute, investigating the role of reactive oxygen species in diseases including cancer, neurodegeneration and cardiovascular conditions.
He has authored over 30 publications and co-edited SLAS Discovery’s first Special Issue on Functional Genomics. He plays a key role in AstraZeneca’s external collaborations, including with the Joint MRC-MTI-AZ Functional Genomics Screening Laboratory, the Joint AZ/CRH Functional Genomics Centre and the MRC funded UK Functional Genomics Initiative.
His work has earned multiple honors, including the Alzheimer’s Association Young Scholar Award, the Boehringer Ingelheim Golden Award, the Discovery Sciences Science Award and the SLAS Discovery Excellence Award.