Destination Drug Discovery

A Satellite Symposium at the Lake Nona (FL) facility of the Sanford Burnham Medical Research Institute
Sunday, January 13
1:00 - 5:00 pm EST

SLAS, in close concert with the Sanford Burnham Medical Research Institute (SMBRI), with support from DiscoveRx and HighResBiosolutions, presents Destination Drug Discovery, a free satellite symposium for SLAS2013 participants. The program includes a tour of Sanford-Burnham's ultra-high throughput screening facility, presentations by top researchers form the Institute, and a reception with informal networking. The event is geared towards a broad array of scientific disciplines; both new and veteran investigators from chemical biology and drug discovery are encouraged to attend.

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Program Details

Destination Drug Discovery: Where is the next generation of medicines going to come from?

The source of tomorrow's medicines is of great concern to policy makers, scientists, physicians, and patients. The high rate of attrition and enormous costs requires a smarter approach to drug discovery, especially in the early stages of target and lead identification, validation, and optimization. This program will feature multiple internationally renowned scientists from SBMRI presenting their cutting-edge research, application of novel technologies, and approaches to discovering new medicines faster, cheaper, and better.

1:00 - 2:00 pm Arrivals, Welcome and Facility Tour
2:00 - 2:45 pm Keynote Presentation: Folding, Trafficking and Functional Selectivity of G Protein-Coupled Receptors: New Targets for Drug Discovery?
Presenter: Michel Bouvier, Biochemistry Professor and CEOIRICoR, Institute for Research In Immunologyand Cancer
During this talk, we will discuss the potential implications of two emerging concepts in GPCR biology for drugdiscovery. It is now clear that each GPCR can engage multiple signaling pathways and that distinct ligands can favor the activation of some of these pathways while inhibiting others. This concept known as functional selectivity or ligand biased signaling has obvious implications for our approaches toward drug discovery. We will review the concept of functional selectivity and describe the development of new tools that allow monitoring such selectivity in living cell and promise the development of drugs with better specificity profiles. An increasing number of genetic diseases have been linked to miss-folding and miss-targeting of GPCRs. We will describe how small molecules that target the miss-folded receptors can act as pharmacological chaperones and rescue folding, trafficking and function of the receptors. Example of the development of pharmacological chaperones for two diseases, nephrogenic diabetes insipidus (resulting from mutations in the V2 vasopressin receptor) and early onset severe obesity (resulting from mutations in the MC4 melanocortin receptor) will be presented.
2:45 - 3:10 pm The Use of Label-Free Technology to Monitor Receptor Activation and Desensitization
Presenter: Patricia McDonald, Associate Scientific Director, Translational Research Institute, The Scripps Research Institute
Activation of intracellular second messenger cascades, resulting from GPCR-ligand interactions, induce cytoskeletal rearrangement leading to changes in cell morphology. These rapid whole-cell responses can be measured using a label-free technology that measures changes in cellular impedance. Moreover, owing to the non-invasive nature of such technologies, in addition to detecting receptor activation; receptor 'desensitization', the process by which GPCR signaling is terminated, can also be detected and quantified. The use of emerging label-free technologies for the purpose of determining the propensity of various GPCR ligands to induce desensitization will be discussed.
3:10 - 3:35 pm Bromodomains: a Family of Druggable Epigenetic Effector Domains
Presenter: Stefan Knapp, Professor of Structural Biology and Group Head, Structural Genomics Consortium, University of Oxford
Bromodomains (BRDs) are acetyl lysine binding modules that have emerged recently as interesting targetsfor drug discovery. The main effort in this target area has been focused on the family of transcriptional regulators of the BET family of BRD proteins. In this presentation if will describe recent progress made identifying new inhibitors for this family as well as strategies inhibitor development that selectively target these protein interactions.
3:35 - 4:00 pm High Throughput Human Biology for Deconvolution of Drug Mechanisms of Action
Presenter: Ellen Berg, Chief Scientific Officer and General Manager, BioSeek (a Division of DiscoveRx)
The high rate of attrition and escalating costs in drug discovery requires innovative approaches. Assaysystems that provide a more physiologically relevant assessment of drug action can improve our ability to identify safe and effective therapeutics earlier in the process. Biologically Multiplexed Activity Profiling (BioMAP® profiling) uses human cell systems to assess the activities of drugs across a panel of complex primary human cell-based assays to provide a better understanding of drug mechanisms relevant to humandisease biology. We have generated a large database of drug activity profiles in these assays and have developed analytical tools for the rapid deconvolution of compound profiles to their target and pathway mechanisms. Here we will discuss how these data arebeing used to develop predictive models for drug safety and efficacy.
4:00 - 4:25 pm Phenotypic Screening at SBMRI: From Assay Development to uHTS, Identifying Inhibitors of Triacylglycerol Accumulation in Muscle
Presenter: Siobhan Malany, Chemical Biology Team LeaderSanford-Burnham Institute for Medical Research
We have developed and validated an assay in a 1536 well plate format to measure intracellular TAGaccumulation in differentiating H9c2 myoblasts. These cells are derived from rat heart, but exhibit skeletal muscle characteristics including the expression of skeletal muscle-specific genes. Cells were treated with oleic acid and stained for lipid using Nile Red. Florescence intensity was either determined by Envision plate reader or by High Content imaging and data sets compared for hits identified from screening the NIH's Molecular Libraries compound collection. In summary, we have identified potent inhibitors of lipid droplet accumulation in muscle cell line as probes to better understand the molecular pathways involved in lipid metabolism that leads to lipotoxicity.
4:25 - 4:35 pm Closing Remarks
5:00 pm Return to Gaylord Palms Convention Center

Transportation and Logistics

Transportation for SLAS2013 participants will be provided to and from the Gaylord Palms Resort and Convention Center. Buses will depart from the Gaylord Palms Transportation Lobby at 1:00 pm EST. Buses will return attendees to the Gaylord lobby by 5:00 p.m. so that they may enjoy the opening SLAS2013 reception in the exhibit area.


Participation in this symposium is free-of-charge to registered SLAS2013 attendees, however, space is very limited and allocated on a first-come, first-served basis. You must be 21 years of age or over in order to attend. Complete contact information is required to complete the registration process.


Any questions related to this event can be directed to or by phone at +1 (510) 979-1415.