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Developing Academic Research into Clinical Therapies

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Byers Auditorium, Genentech Hall, UCSF Mission Bay

600 16th Street

San Francisco, CA 94158

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This event is open to academic registrants only (faculty, staff, postdocs, students). Please use an .edu email address when registering.

For scientists developing therapeutics—whether small molecules, biologics, or cell-based—a university lab has unique advantages as well as disadvantages compared to a research setting at a large company. Academic scientists enjoy intellectual freedom but don’t have access to the same resources as their industry peers.

The Bay Area, however, is fertile ground with leaders in big pharma and a supportive ecosystem for entrepreneurs. UC scientists are well-positioned to commercialize their research whether they choose to partner with industry, license their technology, or launch a startup.

At UCSF we are lucky to have some of the world’s leading researchers developing therapies for unmet needs. Join us on the afternoon of Tuesday, December 6, for “Developing Academic Research into Clinical Therapies,” a half-day symposium featuring UCSF professors Michelle Arkin, Wendell Lim, Dean Sheppard, Brian Shoichet, and Kevan Shokat.

Sponsored by MilliporeSigma.

Hosted by QB3, UCSF, and the Academic Drug Discovery Consortium. Reception to follow.

Agenda

1:00-1:15 pm Registration

1:15-1:20 pm Welcome: Reg Kelly, QB3

1:20-1:35 pm Introduction: Heather Hargett, MilliporeSigma

1:35-2:20 pm Research Talk: Kevan Shokat, UCSF/Berkeley

2:20-3:05 pm Research Talk: Dean Sheppard, UCSF

3:05-3:20 pm Break

3:20-3:30 pm Overview of ADDC: Michelle Arkin, UCSF

3:30-4:15 pm Research Talk: Brian Shoichet, UCSF

4:15-5:00 pm Research Talk: Wendell Lim, UCSF

5:00-6:00 pm Reception

Speakers

Michelle Arkin, PhD
Associate Professor of Pharmaceutical Chemistry
Director of Biology, Small Molecule Discovery Center
Michelle Arkin is an Associate Professor of Pharmaceutical Chemistry and the Director of Biology at the Small Molecule Discovery Center at UCSF. The Small Molecule Discovery Center is a research ‘colaboratory’ that works with academic and biotech investigators to develop small-molecule probes and drug leads. Michelle’s research is focused on structure/function and chemical biology of allosterically regulated enzymes and protein-protein interactions. Her lab also has a strong interest in developing probes and drug leads to address mechanisms of neurodegeneration, cancer, and parasitic disease. Michelle is involved in academic drug discovery as an investigator in the NCI’s Chemical Biology Consortium and the Tau Consortium, an editor of the NIH’s Assay Guidance Manual, and incoming board president of the Academic Drug Discovery Consortium.

Regis Kelly, PhD, OBE
Executive Director, QB3
Byers Family Distinguished Professor, UCSF
Dr. Regis B. Kelly is the Executive Director of QB3, one of the four Governor Gray Davis Institutes for Science and Innovation. Scientists on the three QB3 campuses (UCB, UCSC & UCSF) working at the interface of the physical and biological sciences. The QB3 innovation team converts discoveries into practical benefits for society. As Director, Dr. Kelly helped launch two QB3-associated companies, QB3@953, a life sciences startup incubator, and Mission Bay Capital, a venture fund. As a result of his experiences, Dr. Kelly was appointed as Senior Advisor on Innovation and Entrepreneurship to the President of the University, Janet Napolitano.

Prior to joining QB3 in 2004, Regis Kelly served as executive vice-chancellor at UCSF and chairman of the Department of Biochemistry and Biophysics. He holds the inaugural Byers Family Distinguished Professorship. His academic research was in the field of molecular and cellular neurobiology. His training was at Harvard, Stanford, Caltech and the University of Edinburgh. He has served on many national and international boards. In 2014 he was appointed an officer of the Order of the British Empire (OBE) for services to science, innovation, and global health

Wendell Lim, PhD
Professor & Chair, Department of Cellular and Molecular Pharmacology
Investigator, Howard Hughes Medical Institute
Director, UCSF Center for Systems & Synthetic Biology
Wendell Lim is interested in understanding how living cells use molecular networks to process information and make decisions. His laboratory uses biochemical, structural, genetic, computational, and engineering approaches to elucidate the mechanism of signaling proteins and networks. His lab is also interested in understanding how signaling modules can be used to generate new cellular functions, such as therapeutic immune cells programmed to recognize and treat cancer or other diseases. This work has led to the formation of the company Cell Design Labs.

Cell Design Labs is a biotherapeutics company pioneering breakthrough science to develop disruptive cell-based therapies for cancer and other devastating diseases. Cell Design Labs leverages the power of the body’s immune system to develop smart, living therapies with the capability to treat our most challenging diseases with unprecedented power, precision, safety and durability.

Dean Sheppard, MD
Professor, UCSF School of Medicine
Dean Sheppard’s research focuses on the molecular mechanisms underlying pulmonary (and other organ) fibrosis, asthma and acute lung injury. One aim of the research is to identify new therapeutic targets to ultimately improve the treatment of each of these common diseases. The work begins with basic investigation of how cells use members of the integrin family to detect, modify and respond to spatially restricted extracellular clues and how these responses contribute to the development of common lung diseases. Utilizing mice with global or conditional knockouts of five integrins, the epithelial-restricted integrin, avβ6, and the widely expressed integrins a9β1, avβ5, avβ3 and avβ8, the lab has identified important roles for these integrins in models of each common lung disease and key steps upstream and downstream of the integrins that provide potential therapeutic targets. We have also used the knockout mice to develop potent blocking monoclonal antibodies against 4 of these which are in various stages of clinical development.

Brian Shoichet, PhD
Professor, UCSF School of Pharmacy
The Shoichet lab seeks to discover chemical reagents that can illuminate biological problems. A longstanding effort to do so is by exploiting protein structures to predict new reagents and therapeutic leads (structure-based ligand discovery). Two ongoing projects are developing new computational methods for ligand discovery and applying these to G-Protein Coupled Receptors (GPCRs), which are the single largest family of signaling receptors in human cells.

Allied with this effort is an experimental research program that tests the new methods in well-controlled systems, determining x-ray crystal structures and measuring binding thermodynamics. The experimental program has led to unexpected discoveries, including the observation that many drugs and reagents can form colloidal aggregates in solution. This has led us to investigate how the physical organic chemistry of drugs affects their behavior in vitro and in vivo, influencing drug delivery and formulation.

A new effort turns the entire structural view on its head, developing computational methods to relate receptors by the similarity of their ligands, rather than by protein sequence or structure. This changes pharmacological relationships dramatically—targets that would normally be considered sequence neighbors are pushed far apart (because their ligands are dissimilar), whereas other targets that supposedly have nothing to do with one another become neighbors (because their ligands are very similar). Since the new relationships are articulated by ligands, they may be directly tested both on isolated receptors and, increasingly, against model whole organisms, such as zebra fish, C. elegans and mice. This project seeks to discover the integrated chemical circuits through which drugs and reagents affect whole organisms.

Kevan Shokat, PhD
Professor and Chair, Department of Cellular and Molecular Pharmacology, UCSF
Professor, Department of Chemistry, UC Berkeley
Investigator, Howard Hughes Medical Institute
Research in Dr. Shokat’s laboratory is focused on the discovery of new chemical based tools to decipher cellular signaling networks with an emphasis on protein kinases and more recently, GTPases. The analysis of signal transduction pathways has proven challenging using the traditional tools of biochemistry, genetics, and chemistry. Biochemical approaches are often limited in utility because signaling networks span from the cell surface to the control of transcription and translation, confounding reconstitution efforts from purified proteins. Genetic approaches allow specific perturbation of single components in an intact cell or organism, yet are often confounded by the emergent properties of signaling cascades. Chemical and pharmacological approaches enable rapid, reversible, and graded (dose-dependent) inactivation of single components in intact cells or organisms. Unfortunately, highly selective chemical probes (agonists, antagonists, traceable substrates, etc.) of protein kinases are difficult to develop because the 500 protein kinases share highly homologous ATP binding pockets. My laboratory has solved this fundamental problem for the largest family of enzymes in the human genome, protein kinases, by development of a strategy based on a combination of protein engineering and organic synthesis. We have termed this approach chemical genetics.

Sponsor

About MilliporeSigma

The life science business of Merck KGaA, Darmstadt, Germany, which operates as MilliporeSigma in the U.S. and Canada, has 19,000 employees and 72 manufacturing sites worldwide, with a portfolio of more than 300,000 products enabling scientific discovery. Merck KGaA, Darmstadt, Germany completed its $17 billion acquisition of Sigma-Aldrich in November 2015, creating a leader in the $130 billion global life science industry.

Merck KGaA, Darmstadt, Germany is a leading company for innovative and top-quality high-tech products in healthcare, life science and performance materials. The company has six businesses – Biopharmaceuticals, Consumer Health, Allergopharma, Biosimilars, Life Science and Performance Materials– and generated sales of €12.85 billion in 2015. Around 50,000 employees work in 66 countries to improve the quality of life for patients, to foster the success of customers and to help meet global challenges. Merck KGaA, Darmstadt, Germany is the world’s oldest pharmaceutical and chemical company – since 1668, the company has stood for innovation, business success and responsible entrepreneurship. Holding an approximately 70 percent interest, the founding family remains the majority owner of the company to this day. Merck KGaA, Darmstadt, Germany operates as EMD Serono, MilliporeSigma and EMD Performance Materials in the U.S. and Canada.

Hosts

QB3

QB3 is the University of California’s hub for innovation and entrepreneurship in the life sciences. The institute supports UC researchers and empowers Bay Area entrepreneurs to launch startup companies and partner with industry. QB3 helps bio-entrepreneurs create hundreds of high-value jobs and bring more than $750 million into the Bay Area each year. With campus divisions at Berkeley, San Francisco, and Santa Cruz, QB3 is also affiliated with the San Francisco incubator QB3@953 — home to more than 45 companies — and the seed-stage venture capital firm Mission Bay Capital. Visit qb3.org.

UC San Francisco

UC San Francisco (UCSF) is a leading university dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care. It includes top-ranked graduate schools of dentistry, medicine, nursing and pharmacy; a graduate division with nationally renowned programs in basic, biomedical, translational and population sciences; and a preeminent biomedical research enterprise. It also includes UCSF Health, which comprises top-ranked hospitals, UCSF Medical Center and UCSF Benioff Children’s Hospitals in San Francisco and Oakland – and other partner and affiliated hospitals and healthcare providers throughout the Bay Area.

Academic Drug Discovery Consortium

The goal of the Academic Drug Discovery Consortium (ADDC) is to build a collaborative network among the growing number of university-led drug discovery centers and programs. The ADDC aims to allow scientists to exchange technical expertise on drug discovery and development strategies as well as form partnerships with each other, biopharma companies, and drug discovery-focused contract service organizations and consultants.

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Byers Auditorium, Genentech Hall, UCSF Mission Bay

600 16th Street

San Francisco, CA 94158

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