Discovery on Target
Discovery on Target Mobile Header

Archived content

Day 1  |  Day 2 |  Short Courses |  Download Brochure 



Recommended Short Course:

3:30 pm - 6:30 pm Cardiovascular Safety in Drug Development – From Preclinical to Phase 1 (SC6)*

*Separate Registration Required



7:00 am Registration and Morning Coffee

Exploring Cellular Signaling Pathways and Mode-of-Action

8:30 Chairperson’s Opening Remarks

8:40 Targeting Histone Deacetylases in Non-Small Cell Lung Cancer

Pran Datta, Ph.D., Associate Professor, Surgery and Cancer Biology, Vanderbilt University Medical Center

Histone deacetylation is a crucial step in the loss of specific homeostatic molecular signals in the transition from normal lung epithelium to in situ carcinoma, and finally to invasive and metastatic cancers. We have determined that TGF-ß-induced tumor suppressor function is restored in TGF-ß resistant lung cancer cell lines by the treatment of HDAC inhibitors (HDI). These results suggest that restoration of TGF-ß signaling and the expression of tumor suppressor genes by HDIs, anti-cancer drugs currently in clinical trials, may be a potential alternative for therapeutic intervention of lung cancers.

9:10 New Insights into the Mechanisms of HDAC Inhibitors Anti-Cancer Activity

Roberto Rosato, Ph.D., Assistant Professor, Internal Medicine, Massey Cancer Center, Virginia Commonwealth University

During the last couple of years we have seen important advances in the understanding of the molecular mechanisms involved in HDAC inhibitors anti-cancer activity and in the signals triggered by cancer cells in response to these compounds in order to counteract cell death induction. I will present and discuss important new discoveries in terms of molecular signaling linking critical events that have been identified separately over the last years; this includes the identification of associations between pro-apoptotic signals such as generation of ROS and DNA damage to pro-survival pathways such as NF-kB.

9:40 Total Synthesis and Biological Mode of Action of Macrocyclic Histone Deacetylase Inhibitors

Albert Bowers, Ph.D., Research Fellow, Biological Chemistry & Molecular Pharmacology, Harvard Medical School

The synthesis, biochemical activity and biological activity of numerous macrocyclic depsipeptide histone deacetylase inhibitors will be presented. We have devised total syntheses of FK228 (romidepsin) and the natural product largazole. These agents are sub-nanomolar inhibitors of I HDACs and are being evaluated for therapeutic applications in cancer and erythropoiesis. This is a collaborative effort between our laboratory, Professor James Bradner’s laboratory at the Dana-Farber Cancer Institute, Professor Stuart Schreiber’s laboratory at the Broad Institute and Professor Olaf Wiest at the University of Notre Dame.

10:10 Grand Opening Coffee Break in the Exhibit Hall

10:40 Chemical Phylogenetics of Human Histone Deacetylases

James E. Bradner, M.D., M.Sc., Instructor in Medicine, Harvard Medical School and Dana-Farber Cancer Institute

The broad study of histone deacetylases in chemistry, biology and medicine relies on tool compounds to derive mechanistic insights. A phylogenetic analysis of I and II HDACs as targets of a comprehensive, structurally diverse panel of inhibitors revealed unexpected isoform selectivity even among compounds widely perceived as nonselective. These studies have served as inspiration for the design of compounds with high potency and selectivity amongst individual HDAC isoforms.

11:10 Anti-inflammatory Effects of HDACi Through Modulation of Foxp3+ Treg Functions

Wayne Hancock, M.D., Ph.D., Professor, Pathology, Children’s Hospital of Philadelphia and University of Pennsylvania

Certain HDACi enhance human and murine Treg functions by promoting Foxp3+ T regulatory (Treg) cell production and function. They act in part by promoting Foxp3 acetylation at key lysines. This presentation will consider the roles of HDACi as therapies for autoimmune diseases and transplantation.

11:40 Utility of Predictive Biomarkers in the Clinical Development of the HDAC Inhibitor PCI-24781

Sriram Balasubramanian, Ph.D., Director, Translational Research, Pharmacyclics, Inc.

PCI-24781 is an oral HDAC inhibitor active in lymphoma with a good safety profile, for which we are pursuing a biomarker-driven approach to clinical testing. For example, in colorectal cancer, clinical benefit has been observed which correlates with preclinical activity of PCI-24781 in colorectal tumor cell lines, xenograft models, and primary tumors using the EDR chemoresistance assay. We utilized both mRNA and miRNA profiling approaches in the primary tumors and identified predictive markers of resistance, some of which are shown to be functionally relevant to PCI-24781 activity.

12:10 Keynote Presentation:
Recent Advances in HDAC Biology

Eric M. Verdin, M.D., Professor of Medicine, University of California, San Francisco

Histone deacetylases have crucial roles in numerous biological processes, largely through their repressive influence on transcription. The expression of many HDAC isoforms in eukaryotic cells raises questions about their possible specificity or redundancy, and whether they control global or specific programs of gene expression. I will review recent analyses of HDAC function in development and disease.

12:40 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own


2:20 Chairperson’s Remarks

Joel M. Gottesfeld, Ph.D., Professor, Department of Molecular Biology, The Scripps Research Institute

2:25 Therapeutic Applications of Histone Acetylation-Independent Mechanisms of HDAC Inhibitors in Cancer Treatment

Ching-Shih Chen, Ph.D., Professor of Medicinal Chemistry, Internal Medicine and Urology, The Ohio State University

Accumulating evidence indicates that HDAC inhibitors mediate anti-tumor effects, in part, through histone actylation-independent mechanisms by disrupting the interaction with HDAC’s binding partners or deactivating the function of signaling proteins. Many of these non-histone targets, including Akt, Ku70, Hsp90, and Nur77, could be therapeutically exploited in combination therapy with other targeted agents.

2:55 Design and Development of a Novel Series of HDAC Inhibitors for the Treatment of Cancer and Inflammation

Stephen Shuttleworth, Ph.D., Chief Scientific Officer, Karus Therapeutics Ltd.

We have focused on the development of two distinct classes of HDAC inhibitors - with unique subtype selectivities - for the treatment of cancer and inflammatory diseases. These compounds display high in vitro biochemical and functional cellular activities, and exert potent and sustained pharmacodynamic effects. We have nominated a novel oncology development candidate and will initiate clinical studies in patients with solid tumors in early 2010. Additionally, we have designed potent, advanced in vivo lead compounds for the treatment of psoriasis and rheumatoid arthritis and data from these programs will be presented.

3:25 Development of Novel HDAC Inhibitors for CNS Indications

Holger Patzke, Ph.D., Principal Scientist, Neuropharmacology, EnVivo Pharmaceuticals, Inc.

Over the past years we have developed a set of novel HDACi that are suitable for use in neurodegenerative diseases based on their improved safety, brain penetration, potency and selectivity. We have concluded pre-clinical development of our lead compound, EVP-0334. EVP-0334 has excellent drug-like properties, is well-tolerated in mice and dogs, and enhances memory in rodents. EVP-0334 is a first-in-class agent that will enable investigation of the therapeutic potential of HDAC inhibition for neurodegenerative diseases.

3:55 Networking Refreshment Break in the Exhibit Hall

4:30 Histone Deacetylase Inhibitors as Therapeutics for Neurodegenerative Diseases

Joel M. Gottesfeld, Ph.D., Professor, Department of Molecular Biology, The Scripps Research Institute

Our laboratory recently identified a novel of HDAC inhibitors that reverse heterochromatin-mediated silencing of the frataxin gene in cell-based and animal models for the neurodegenerative disease Friedreich’s ataxia. These compounds exhibit no acute or chronic toxicity at potential therapeutic doses in mice, and also show therapeutic benefit in a mouse model for Huntington’s disease.

Gloucester Pharma5:00 Clinical Development of a Novel Pan-HDAC Inhibitor, Romidepsin, from Phase I Through the New Drug Application (NDA) 
Elizabeth Faust, Ph.D., Senior Director, Medical Affairs, Gloucester Pharmaceuticals
Romidepsin is a unique cyclic peptide that has been identified as a novel, potent, pan-HDAC inhibitor. More than 850 patients with cancer have received romidepsin as a single agent or in combination with other targeted and cytotoxic agents in clinical studies conducted worldwide. The FDA has accepted and is reviewing a New Drug Application (NDA) for the use of romidepsin in cutaneous T-cell lymphoma (CTCL). Gloucester Pharmaceuticals is also enrolling patients in a registration trial for peripheral T-cell lymphoma (PTCL) and is evaluating romidepsin in combination with other agents in various additional indications including multiple myeloma.

5:15 Discovery and Pre-clinical Profile of Novel Potent HDAC Inhibitors

Shridhar Narayanan, Ph.D., Senior Vice President, Biology, Orchid Research Laboratories Limited

We have discovered and developed a series of potent pan and isoform selective HDAC inhibitors for the treatment of cancer. The prototype molecule, OCIDxxxx is a potent subtype selective HDAC1 inhibitor. OCIDxxxx demonstrates high in vitro anti-proliferative effects in a variety of tumor cell lines. OCIDxxxx has good metabolic stability and oral bioavailability and is active in a diverse range of tumor xenograft models. GLP toxicology studies have been initiated for this compound and clinical studies are planned for 2010.

5:45 Happy Hour in the Exhibit Hall

7:00 Close of Day

Day 1  |  Day 2 |  Short Courses 


Suggested Event Package (November 1 – 4):

November 2 - 3


HDAC Inhibitors: Developing Molecules for Targeting Oncology and More

November 3 - 4


Kinase Inhibitors: Moving from Discovery to Development 

Japan-Flag Korea-Flag China-Simplified-Flag China-Traditional-Flag  

Register Today!

Final Agenda Now Available









The exhibit hall was sold out in 2015, so please contact us early to reserve your place. To customize your sponsorship or exhibit package for 2016, contact:

Jon Stroup
Sr. Business Development Manager






Next-Generation Histone Deacetylase Inhibitors

Strategies for Tackling Rare Genetic Diseases

Understanding CRISPR: Mechanisms and Applications

Autoimmunity – Small Molecule Approaches

NK Cell-Based Cancer Immunotherapy

Medical Dermatology Therapeutic R&D and Technical Innovation



Targeting Histone Methyltransferases and Demethylases

Targeting the Ubiquitin Proteasome System

Targeting the Microbiome
– Part 1

GPCR-Based Drug Discovery - Part 1

Advances in Gene Editing and Gene Silencing – Part 1

Gene Therapy Breakthroughs

Antibodies Against Membrane Protein Targets – Part 1

Targeting Cardio-Metabolic Diseases

Targeting Ocular Disorders


Targeting Epigenetic Readers and Chromatin Remodelers

Kinase Inhibitor Discovery

Targeting the Microbiome
– Part 2

GPCR-Based Drug Discovery - Part 2

Advances in Gene Editing and Gene Silencing – Part 2

Translating Cancer Genomics

Antibodies Against Membrane Protein Targets – Part 2

Metabolomics in Drug Discovery

TRAINING SEMINAR: Data Visualization


Monday, September 19
8:00 - 11:00 am

(SC1) Immunology Basics for Chemists

(SC2) Designing Peptide Therapeutics for Specific PPIs

(SC3) Phenotypic Screening and Chemical Probe Development

(SC4) Medical Dermatology Therapeutic R&D and Technical Innovation - Part 1

Monday, September 19
2:00 - 3:00 pm

(SC5) GPCR Structure-Based Drug Discovery

(SC6) RNA as a Small Molecule Drug Target

(SC7) Using IP Landscape Studies to Improve Your Confidence

(SC8) Medical Dermatology Therapeutic R&D and Technical Innovation - Part 2

Monday, September 19
3:30 - 6:30 pm

(SC9) Targeting of GPCRs with Monoclonal Antibodies

(SC10) Introduction to Targeted Covalent Inhibitors

(SC11) Contact Lens Drug Delivery Systems

(SC12) Introduction to Gene Editing

Monday, September 19
7:00 - 9:30 pm

(SC13) Convergence of Immunotherapy and Epigenetics for Cancer Treatment

Wednesday, September 21
7:00 - 9:30 pm

(SC14) Cancer Metabolism: Pathways, Targets and Clinical Updates

(SC15) Introduction to Allosteric Modulators and Biased Ligands of GPCRs

(SC16) Functional Screening Strategies Using CRISPR and RNAi

(SC17) Challenges and Opportunities in DNA Methyl Transferase (DNMT) Inhibitors as Therapeutics