Sylviane Muller is a Distinguished class research director in the French Centre National de la Recherche Scientifique (CNRS) and Professor at the Institute of Advanced Studies of the Strasbourg University, in charge of the chair Therapeutic immunology. She is deputy director of the CNRS Molecular and cellular biology Institute (IBMC) in Strasbourg (France), Chair and Director of the CNRS Unit entitled Immunopathology and therapeutic chemistry, and Head and coordinator of the Drug discovery Center for cancer and inflammation Medalis awarded 'Laboratory of Excellence’. She defended a doctoral degree in Molecular Biology in 1978 and a thesis in Science in 1984 in Strasbourg. She was a post-doctoral fellow at the Max-Planck Institute for Immunobiology in Freiburg (Germany). She is the co-author of 249 publications in peer-reviewed journals and 97 review articles and chapters. Her scientific activity has led to ~30 patents (most are licenced). She is the co-founder of two companies, namely Neosystem (1986) and ImmuPharma (2002). She is a member of the editorial board of several scientific journals and of international scientific Societies. She gave numerous lectures in Europe and the US, and participated to many international meetings as an invited speaker. She co-organized ten international congresses in the field of autoimmunity and lupus. She received the CNRS Silver Medal (2009). She is Chevalier de l’Ordre de la Légion d’Honneur (2010).
Research field:
In 2001, S. Muller started her own CNRS laboratory at IBMC, where she concentrates her activity on systemic lupus erythematosus, which represents a prototype of autoimmune rheumatic disease. Pr. Muller’s team is interested in understanding the molecular and cellular pathways involved in autoreactive lymphocytes activation and the events leading to cell death/living phenomena (apoptosis, autophagy) that are central in lupus. Combining fundamental knowledge of lupus with a long-lasting experience in peptide immunochemistry, S. Muller and her team were able to develop a very novel strategy, based on synthetic peptides, designed to modulate the aberrant immune response and restore immune system functions in lupus mice and patients. The results of a peptide-based Phase IIb clinical trial including in ~150 lupus patients gave extremely promising results and a phase III clinical trial has started in 2015.

JD received his PhD degree in Organic Chemistry in 1996 under the supervision of Prof Meslin at the UFR des Sciences et Techniques in Nantes; he has dedicated this time to the discovery of hetero Diels-Alder reactions to reach novel approaches to a range of heterocycles . In 1997, he joined Professor Young’s group at the University of Sussex as a post-doctoral Fellow, where in worked on the synthesis of unnatural aminoacids containing fluorine and their incorporation into proteins. He joined Vertex in 1998 where he is currently working. JD has been involved in a number of research programmes and has developed leadership responsabilities for kinase projects, mostly in the oncology field. In the past few years, his expertise led him to be appointed as Chemistry Head for the ATR programme, which resulted into the discovery of two clinical candidates, one of which, VX-970, is the topic of today’s presentation.

Tjeerd Barf obtained his Ph.D. in Medicinal Chemistry 1996 from the University of Groningen, The Netherlands. He subsequently joined Pharmacia & Upjohn in Uppsala, Sweden, in 1997 and continued his career with the spin-off company Biovitrum (2000). He has led medicinal chemistry programs concerning metabolic diseases and assumed overall program leadership later on. In 2005, he moved to Organon (Oss, The Netherlands), and as a Group Leader, his responsibilities encompassed heading immunology lead finding projects. After the acquisition by Schering-Plough and the merger with Merck, he led a global cross-site lead optimization program in the therapeutic area of autoimmune diseases. The last decade, he has been intimately involved in drug discovery programs that utilize the covalent binding technology, mainly with the focus on covalent kinase inhibitors in the oncology and autoimmune disease space. As of 2011, expansion of these drug discovery activities took place in privately owned biotech companies. He is one of the founders of Acerta Pharma and presently holds the position of EVP of Chemistry.

Patrice Courvalin, M.D., is Professor de Classe Exceptionnelle at the Institut Pasteur where he is the Head of the Antibacterial Agents Unit since 1983. He and his collaborators are experts in the genetics and biochemistry of antibiotic resistance. In particular, he first described and then elucidated vancomycin resistance in Enterococcus. His research has led to a revision of the dogma describing natural dissemination of antibiotic resistance genes. He and his colleagues demonstrated that a wide variety of pathogenic bacteria can promiscuously exchange the genetic material conferring antibiotic resistance, proved that conjugation could account for dissemination of resistance determinants between phylogenetically remote bacterial genera, elucidated the transposition mechanism of conjugative transposons from Gram-positive cocci, and more recently, has obtained direct gene and protein transfer from bacteria to mammalian cells. His work is reported in more than 290 publications in international scientific journals.

Current position: Prin. Scientist, Biology-Discovery, Virology & Vaccine Late Discovery Research field: Infectious disease therapeutics Education: Doctor of Philosophy, Cell & Molecular Biology, Pennsylvania State University Candidate for Masters in Pharmaceutical Chemistry University of Florida Bachelor of Science, Biochemistry; Minor in Biology University of Delaware Professional experience: 2007-2014: Idenix Pharmaceuticals, Inc., Director of Biology, HCV Project Team Leader Established and utilized models to investigate the inhibition, resistance and toxicity of small molecules against hepatitis C, among other side projects and responsibilities, including a drug discovery project leader for multiple programs and new target initiatives. 2003-2007: Postdoctoral fellow: Microbiology & Molecular Genetics Harvard Medical School Focused on a genetic system for generation of a recombinant gammaherpesvirus for antiviral assays, vaccine vector candidates for SIV, posttranslational regulation, and microRNA identification. 2002-2003: Postdoctoral fellow: Penn State University Generation and utilization of recombinant insect viruses for gene transfer of mammalian expression cassettes to primary hepatocytes.

Motonari Uesugi is a Professor of The Institute for Integrated Cell-Material Sciences and Institute for Chemical Research, Kyoto University. After completing postdoctoral training in Harvard Chemistry Department, Dr. Uesugi started his independent career in Baylor College of Medicine, Houston, where he has established an interdisciplinary laboratory in the area of chemical biology. He was tenured in Baylor in 2005, and moved to Kyoto University as a full professor in 2005. He is a recipient of Tokyo Techno Forum 21 Gold Medal Award (2006) and German Innovation Award (2011). Dr. Uesugi and his co-workers aim to gain a fundamental understanding of biological events through the study of small molecules.

Michael J. Sofia, Ph.D. is currently CSO of Tekmira/OnCore Inc. a company focused on the discovery and development of therapies to treat HBV. Previously he was CSO and Co-founder of OnCore Biopharma which merged with Tekmira in March 2015. Prior to co-founding OnCore BioPharma, Inc., Mike most recently was Senior Vice President of Chemistry, Princeton Site Head and Senior Advisor at Gilead Sciences. Previously he was Sr. Vice President of Chemistry at Pharmasset, Inc. He joined Pharmasset in 2005 and had responsibility for research strategy, medicinal chemistry, process research, computational chemistry and analytical chemistry functions and was a clinical development project leader. He is an inventor of Sovaldi (sofosbuvir), a treatment of hepatitis C infection currently approved and marketed by Gilead Sciences, Inc. Prior to Pharmasset, he held positions of Group Director, New Leads Chemistry at Bristol-Myers Squibb, Vice President of Research at Intercardia Research Labs (formerly Transcell Technologies) and was Research Investigator and Project Leader at Lilly Research Labs, Eli Lilly & Co. He did his postdoctoral training in synthetic organic chemistry, as an NIH fellow, at Columbia University and earned his Ph.D. in organic chemistry from the University of Illinois, Urbana-Champaign. He received his B.A. degree in chemistry from Cornell University. He has authored 100 publications, 9 book chapters and numerous abstracts and is an inventor on over 30 patents. He also holds a professorship position at the Baruch S. Blumberg Institute and is on the editorial board of several scientific journals. Mike is the recipient of the PA BIO 2014 Scientific Achievement Award for the discovery of Sofosbuvir.

John Wai obtained his BSc in chemistry from the University of Hong Kong and his PhD from the University of British Columbia, Canada with late Professor Edward Piers. After finishing his post-doctoral training with Professor K. Barry Sharpless, John joined Merck Research Laboratories, Department of Medicinal Chemistry (West Point, PA) in 1989. At Merck, he contributed to the discovery efforts of a number of programs, from target validation & lead identification to lead optimization & early development. This includes HIV-1 non-nucleoside reverse transcriptase inhibitors, ras-farnesyl protein transferase inhibitors, fibrinogen receptor antagonists, HIV integrase strand transfer inhibitors, HIV RNase H inhibitors, gamma secretase inhibitors, etc. Through the years, John rose through the ranks and was promoted to be Director of Medicinal Chemistry in 2005. In 2007, he received the Distinguished Scientific Award from the inaugural Merck WP Basic Research Reward and Recognition Forum for his work on HIV integrase inhibitors. In 2013, John received the Heroes of Chemistry Award from the American Chemical Society for his contribution to the discovery and development of Isentress, the first HIV integrase inhibitors approved for treatment of AIDS (2007). In 2014, he joined WuXi AppTec (Shanghai) as Vice President of Medicinal Chemistry, and has recently been appointed as Adjunct Professor of Fudan University, College of Pharmacy, Department of Medicinal Chemistry.

Dr. Rodriguez was born October 27, 1978 and raised in the south of France by his parents Jean-Paul and Françoise Rodriguez. After receiving his undergraduate degree at the University of Avignon in June 2002, he performed graduate studies at Marseille and Oxford under the supervision of M. Santelli and Sir J. E. Baldwin. During his Ph.D. studies, he completed the total synthesis of several natural products, providing novel chemical ways to create complex molecular frameworks. Following completion of his doctoral studies, he joined the University of Cambridge in November 2005 as a Cancer Research UK postdoctoral fellow under the mentorship of S. Balasubramanian and was promoted to Senior Research Associate in 2009. His research encompassed the design and synthesis of small molecules to study the existence and biological consequence(s) of G-quadruplex nucleic acids in the human genome. In 2012, Raphaël joined the CNRS (ICSN, Gif-sur-Yvette, France) as a group leader and is now based at the Institut Curie Research Centre (Paris). Current research interests include chromatin and cancer stem cell biology.

I received an MPhil. in 1999 and a Ph.D. in 2002 both in organic chemistry at the Manchester University, in the UK. During my Ph.D., I was author of 8 publications. I then started my industrial carrier at EvotecOAI, where I developed my expertise in parallel synthesis. Quickly, I joined Faust pharmaceuticals for a new position in 2004, where I did my initiation to medicinal chemistry and neurodegerative diseases, looking for active drugs for mGluRs. I then moved to new challenges at Galapagos. My carrier at Galapagos was paved with successes including the discovery of 7 active small molecules, which moved to preclinical studies. 3 compounds were tested in phase I clinical trial with a positive proof of mechanism in auto-immune diseases. From these 3, 2 are currently in phase II, GLPG0778 and GLPG0634. The two compounds already showed promising results, Psoriasis for GLPG0778 and RA for GLPG0634. GLPG0634 was the first selective JAK1 inhibitor known in clinical trials in inflammatory and auto-immune conditions. I am today leading a group of 25 researchers and I am inventor of more than 16 patents.