High-throughput screening (HTS) is a fundamental, efficient, broadly used, and fast strategy to identify hits representing a key starting point for the development of novel drugs. The targets for HTS are selected for their disease relevance and can be specific molecular targets, but also intracellular pathways or altered phenotypic evidence. The choice of the most suitable assay format for a given target/pathway is mainly driven by the disease relevance of the assay. Apart from the disease relevance, assays suitable for HTS need to be sensitive, robust, and cost effective. The robustness of an assay can be assessed with quality parameters which include the Z’ score, pharmacological reproducibility and the signal stability over time.
In the last years, the targets identified for conducting HTS campaigns became more and more challenging, as in most cases they do not belong to the more classical druggable families, such as GPCRs, ion channels, or enzymes like kinases, but are more likely to come from a wide range of categories most often defined as undruggable.
In our webinar, we will present a collection of many different cell-based and cell-free assays we developed using different approaches and technologies, to run HTS on tough targets, including protein-protein and protein-RNA interactions, protein degradation, protein aggregation, RNA splicing modulation and inducible RNA silencing, phenotypic analysis, organellar ion channels and solute carrier proteins.
Key words: HTS-grade assays, multi-subunit enzyme, protein aggregation, protein interaction, protein degradation, RNA focused assays, splicing assays, lysosomal disease, autophagy, organellar patch clamp, phenotypic assays.
CEO & CSO
Director, Discovery Services
Stefan Lohmer is Axxam’s co-founder and Chief Executive Officer. Prior to founding Axxam, he was head of the assay development unit at the Bayer Research Centre in Milan and the Head of Genomics worldwide for Bayer AG. He was responsible for generating and managing Bayer’s external genomic alliances with Millennium and Lion Bioscience. Stefan Lohmer joined Bayer in 1992. He holds a degree in Molecular Biology and Biochemistry from the University of Cologne and completed his PhD at the Max Planck Institute for Plant Breeding in Cologne.
Lia Scarabottolo has a degree in Pharmaceutical Chemistry and Technology and a Master in Experimental Pharmacology (University of Milan). Since January 2007, she is Director of the Discovery Services Department at Axxam, with the responsibility of the units of: Cell Biology, Biochemistry, Screening Technologies, Electrophysiology, High Content Screening, Molecular Biology, Cell Factory and iPSC. She is program director for many of the collaborations in place with national and international companies, as well as scientific coordinator for projects granted by the Italian government, the European Commission, and international organisms.