Dr. LeeVincent H.L. Lee, Ph.D., The Chinese University of Hong Kong, Hong Kong

Research breakthroughs in pharmaceutical sciences

Dr. Lee is a world-renowned leader in drug delivery. His research was funded by the National Institutes of Health in the United States for more than 20 years. He has published more than 200 papers in three inter-related areas. They are: (a) defining the biochemical barriers to peptide and protein drug delivery; (b) elucidating the structure-function of drug transporters; and (c) characterizing the   pharmacokinetic barriers to drug delivery in treating retinal degenerative diseases. Over 30 graduate students, postdoctoral fellows, and visiting scientists from North America, Asia, and Europe have been trained by Dr. Lee. Several international honors and awards, including an Honorary Doctor of Science degree from the University of London, United Kingdom. Dr. Lee was elected President of the American Association of Pharmaceutical Scientists in 1996 and of the Controlled Release Society in 1993. A member of several editorial advisory boards and scientific advisory boards, Professor Lee was editor-in-chief of Pharmaceutical Research and Advanced Drug Delivery Reviews and was the founding co-editor of the Journal of Drug Targeting.

Dr. TakaduraYoshinobu Takakura, Ph.D., Kyoto University, Japan

Exosome-based nucleic acid drug delivery for cancer immunotherapy

Dr. Takakura has been a Professor at Graduate School of Pharmaceutical Sciences, Kyoto University since 1997 and was Dean of the Graduate School from April, 2014 to March, 2016. He received his Ph.D. in Pharmaceutical Sciences, from Kyoto University in 1986. He was a postdoctoral at Department of Pharmaceutical Chemistry, University of Kansas (Professor Ronald T. Borchardt’s Lab). He has conducted a series of studies in the field of macromolecular drug delivery in the field of biopharmaceutics. Macromolecular drug involves drug conjugates with macromolecular carrier, protein drugs, and nucleic acid drugs such as antisense oligonucleotides and plasmid DNA expressing therapeutic proteins and short interfering RNA (siRNA). His major recent research interest involves nucleic acid drug delivery for optimization of gene therapy, DNA vaccination, RNAi-based therapies. Very recently, he started the project on the development of exosome-based nucleic acid delivery system. He has published 295 peer reviewed original research articles, 42 review articles, and 48 book chapters. His honors include Young Investigator’s Award from the Japanese Society for Xenobiotic Metabolism and Disposition in 1995, Young Investigator’s Award from The Pharmaceutical Society of Japan, in1996, AAPS Fellow in 2009, and Takeru and Aya Higuchi Memorial Prize from the Academy of Pharmaceutical Science and Technology (APSTJ), Japan, in 2011. He is the Chair of the Board of Directors and Councilor, Japanese Society of Drug Delivery System (JSDDS), Councilor of APSTJ, and the Board of Director, Globalization of Pharmaceutics Education Network (GPEN).

Dr. KhaliliKamel Khalili, Ph.D., Temple University School of Medicine, USA

Elimination of HIV-1 DNA by CRISPR Technology in Living Animals

Dr. Khalili is the Laura H. Carnell Professor and Chair of Neuroscience, Director of the Center for Neurovirology and Director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine at Temple University.
Dr. Khalili has been actively investigating the molecular biology of HIV-1 and the pathogenesis of AIDS for more than 30 years. By concentrating on the mechanism involved in regulation of HIV-1 gene transcription and replication in several cell models, he has made several important discoveries related to LTR interaction with host factors, development of small inhibitory molecules for suppressing viral replication, the interaction of HIV-1 with other pathogens (most notably JCV) in brain, and the mechanism for reactivation of this neurotropic virus by HIV-1 and the development of PML in brain of AIDS patients. He has published more than 170 (out of 427 published papers) papers on HIV-1 etc. Most recently, his laboratory, for the first time, has developed a gene editing strategy that eliminates the entire HIV-1 genome in latently infected human culture and has shown significant efficacy in ex vivo as well as in vivo models. He has trained more than sixty graduate (Ph.D. and M.D./Ph.D) and postdoctoral fellows who are actively engaged in research related to neurovirology and neuroscience of HIV-1

Dr. DuJianzhong Du, Ph.D., Tongji University, China

Design of novel biomaterials for drug delivery

Jianzhong Du received his PhD in chemistry from Institute of Chemistry, Chinese Academy of Sciences (CAS) in 2004 under the supervision of Prof. Yongming Chen. His PhD thesis was awarded ‘The Top 50 PhD Dissertations in CAS’ in 2005 and ‘The Nominated National Top 100 PhD Dissertations in China’ in 2006. He was also an Alexander von Humboldt Fellow in Germany in 2006. He immediately moved to Prof. Steven Armes group at the University of Sheffield as a postdoctoral fellow in 2004. Then in 2008 he moved to Rachel O’Reilly’s group at the University of Cambridge. He was appointed an ‘Eastern Scholar’ professor in 2009 and a ‘Shanghai 1000 Plan’ professor in 2011 at Tongji University. Currently he is head of department of polymeric materials, Tongji University. He has published 80 peer-refereed journal papers in J. Am. Chem. Soc., Angew. Chem., ACS Nano, Chem. Soc. Rev., Porg. Polym. Sci., etc. His research interests include polymer vesicles for DNA/RNA/protein delivery, antibacterial, antitumor and theranostic applications. He received the National Award for the Progress in Science and Technology in 2016.

Dr. TsaiShengdar Tsai, Ph.D., St. Jude Children's Research Hospital, USA

Defining Genome-wide Activity of CRISPR-Cas Nucleases for Therapeutics

Dr. Shengdar Q. Tsai obtained his B.S. in Biochemistry and Chemistry from the University of Michigan, his M.S. in Bioinformatics and Ph.D. in Functional Genomics from North Carolina State University.  He was a postdoctoral research fellow and instructor at Massachusetts General Hospital and Harvard Medical School in the lab of Dr. J. Keith Joung, a pioneer of genome editing technologies. He is currently an Assistant Member in the Department of Hematology at St. Jude Children’s Research Hospital. Dr. Tsai has focused on developing methods for high-throughput genome editing with TALENs, and defining and improving the genome-wide specificity of CRISPR-Cas nucleases.  His long-term research goals are to develop safe and highly specific targeted genome-editing strategies for treating human genetic diseases.

Dr. SiegwartDaniel Siegwart, Ph.D., University of Texas Southwestern Medical Center, USA

Non-viral CRISPR/Cas gene editing enabled by co-delivery of mRNA and sgRNA inside of synthetic lipid nanoparticles

Dr. Daniel J. Siegwart is currently an Assistant Professor in the Simmons Comprehensive Cancer Center and Department of Biochemistry at the University of Texas Southwestern Medical Center. He received a B.S. in Biochemistry from Lehigh University in 2003, and a Ph.D. in Chemistry from Carnegie Mellon University (CMU) in 2008 under the supervision of University Professor Krzysztof Matyjaszewski. During his graduate studies, he received the Joseph A. Solomon Memorial Fellowship in Chemistry at CMU and was a National Science Foundation East Asia and Pacific Summer Institutes Fellow at The University of Tokyo in 2006 with Professor Kazunori Kataoka. He then completed a National Institutes of Health NRSA-sponsored Postdoctoral Fellowship at Massachusetts Institute of Technology with Institute Professor Robert Langer (2008-2012). The Siegwart Lab aims to discover and define the critical physical and chemical properties of synthetic carriers required for therapeutic delivery of small (e.g. ~22 base pair miRNA) to large (e.g. ~5,000 nucleotide mRNA) RNAs. Their research is grounded in chemical design and takes advantage of the unique opportunities for collaborative research at UT Southwestern. They ultimately aspire to utilize chemistry and engineering to make a beneficial impact on human health.

Dr. GurumurthyChannabasavaiah Gurumurthy, Ph.D., University of Nebraska Medical Center, USA

The CRISPR/Cas9 System and the Latest Trends in Animal Genome Engineering

CB Gurumurthy is Assistant Professor of the Department Developmental Neuroscience, Munroe Meyer Institute for Genetics and Rehabilitation, University of Nebraska Medical Center and he serves as the Director of UNMC Mouse Genome Engineering Core Facility. His research interests are genome engineering and technology development. With his experience both in traditional and advanced genetic engineering techniques, he is actively developing newer technologies for generation of genetically engineered animal models and developing widely-usable animal models for basic and drug discovery research. He received his BVSC (DVM) from Bangalore Veterinary College, India and MVSC & PHD in Veterinary Virology from Indian Veterinary Research Institute with a Gold Medal distinction in MVSC. He also received his Executive MBA from University of Nebraska at Omaha. Previously, he served as a Scientist in Molecular Biology, Drug Discovery Research at Dr. Reddy’s Laboratories, Hyderabad, India, for 2 years and a short post doctoral research project in Dr. Rakesh Mishra’s lab at Center for Cellular and Molecular Biology, Hyderabad, before he moved to Northwestern University, Chicago for a Post-Doctoral work in Cancer Biology and Mouse Molecular Genetics.

Dr. YangLili Yang, Ph.D., University of California, USA

Stem cell-engineered invariant natural killer T-cells for cancer therapy

Lili received her B.S. degree in Biology from the University of Science & Technology of China (USTC) in 1997, her M.S. degree in Biomedical Sciences from the University of California, Riverside (UCR) in 1999, and her Ph.D. degree in Biology from the California Institute of Technology (Caltech) in 2004. She obtained her Ph.D. training at the Laboratory of David Baltimore. Post-graduation, she stayed at Caltech and led a multi-institutional Engineering Immunity Program from 2004 to 2012, developing gene- and cell-based immunotherapies for cancer and HIV/AIDS. Her work resulted in over 24 peer-reviewed publications, 8 patents and 2 clinical trials. She joined the University of California, Los Angeles (UCLA) as an Assistant Professor in January of 2013. Her laboratory at UCLA studies tumor immunology and cancer immunotherapy, with a special focus on stem cell-engineered immunotherapy for cancer. In recognition of her research achievements, Lili has received multiple awards including the American Society of Gene & Cell Therapy (ASGCT) Outstanding New Investigator Award, the American Association of Immunologists (AAI) Young Investigator Award, the NIH Director’s New Innovator Award, the CIRM Partnering Opportunity for Translational Research Award, the STOP CANCER Research Career Development Award, the CHARVI/HVTN Early Career Investigator Award, the Forbeck Scholar Award, and the TR35 Award.

Dr. KimWon Jong Kim, Ph.D., Pohang University of Science and Technology, South Korea

Stimuli-responsive nanomedicine for anticancer therapy

Prof. Won Jong Kim received his BSc from Hanyang University in 1998, and M.S. and Ph. D. in Biomolecular Engineering in 2004 at Tokyo Institute of Technology. During his graduate studies with Profs T. Akaike and A. Maruyama, he developed a polymer-mediated DNA detection system. From 2004 to 2007, he was a postdoctoral fellow at the University of Utah under the supervision of Prof. Sung Wan Kim. Currently, he is a tenured associate professor at the Department of Chemistry, POSTECH and a group leader of Center for Self-assembly and Complexity, Institute for Basic Science (IBS). His contributions during the past 9 years as POSTCH faculty have been acknowledged by the publication of more than 80 peer-reviewed articles in the most important journals in his field, such as Nature Comm, Angew Chem, ACS Nano, Nano Lett, Small, and Biomaterials. Dr. Kim has also given more than 70 invited talks and lectures in national and international conferences and universities. He received KCS-Wiley Young Chemist Award (2011), Wiley-PSK Journal of Polymer Science Young Scientist Award (2012), KCS-Award for the Advancement of Science (2014), and PSK-Mid-career Researcher Academy Award (2015). He is an associate editor of newly launched journal “Nanotheranostics”, and editorial member of “Materials Today Chemistry”.

Rolf TurkRolf Turk, Ph.D., Integrated DNA Technologies, USA

Efficient homology-directed repair using long single-stranded DNA templates

Rolf Turk is a Staff Scientist at Integrated DNA Technologies in Coralville, Iowa. Rolf is originally from the Netherlands where he obtained a master’s degree in science at the University of Amsterdam. He continued his training as a graduate student at the Center for Human and Clinical Genetics at the Leiden University Medical Center. His research focused on the molecular mechanisms behind different forms of muscular dystrophy using gene expression profiling. After obtaining his Ph.D., he continued his research on muscular dystrophy as a post-doctoral researcher in the laboratory of Kevin P. Campbell, a HHMI Investigator at the University of Iowa. Since his employment at IDT in the fall of 2105, Rolf has been part of the Molecular Genetics Department where he focusses on the further development of RNA-guided endonucleases and optimizing delivery strategies.

Carol Szczepaniak

Carol Szczepaniak, Nebraska Coalition for Ethical Research (NCER) President

The Ethical Issues of CRISPR/Cas9 Technology: Aspiring Towards Perfection or Annihilation?

Carol received her Bachelor of Science degree in Biology from Benedictine University. After 36 years of combined scientific pursuits including chemical research, teaching and pharmaceutical consulting, she received her Master of HealthCare Ethics from Creighton University. Additionally, Carol has held the office of President of Nebraska Coalition for Ethical Research (NCER) since 2013. In that capacity, she has co-authored position statements on the ethics of Stem Cell Research, Mitochondrial Replacement Therapy, Therapeutic Cloning and CRISPR/Cas9. NCER publishes monthly bioinformatics on the ethics of current medical research, reaching subscribers in 56 countries.

Dr. PawelczakKatherine Pawelczak, Ph.D., VP of Research, NERx Biosciences

Chemical enhancement of CRISPR/Cas9 mediated site-specific genome engineering using novel inhibitors of the Ku-DNA interaction

Dr. Pawelczak has extensive research experience with precision genome editing technologies from her time working in Discovery at Dow AgroSciences, where she was responsible for work on the development and deployment of proprietary zinc finger nuclease genome engineering technologies. This work included developing novel molecular analytical techniques for determining efficiency of ZFN mediated genome engineering. She was also responsible for directing projects to further our understanding of basic mechanisms of ZFN technology, genome engineering and the relationship with DNA repair with the goal of improving product applications. Dr. Pawelczak’s graduate and post-doctoral experience focused on understanding molecular mechanisms of DNA repair proteins, with a particular emphasis on non-homologous end joining mediated repair of DNA double strand breaks. She has extensive experience in nucleic acid enzymology, protein-DNA interactions, biochemistry and molecular analysis. Her industry experience in precision genome engineering combined with her expertise in DNA repair mechanisms provide enormous support in the research

Dr. GodwinAndrew Godwin, Ph.D., University of Kansas Medical Center, USA

Exosomes in Precision Medicine: Cancer Detection and Monitoring using Liquid Biopsy Blood Testing

Andrew K. Godwin, Ph.D., is a leader in the field of translational research and personalized cancer medicine. He is an NCI funded investigator and a highly published (>380 manuscripts and scholarly review articles) and cited (>33,000 citations) scientist. Dr. Godwin is internationally recognized for his molecular biology/genetic studies of sarcoma (gastrointestinal stromal tumors and Ewing sarcoma), breast and ovarian cancer, and his efforts to help bridge the gap between basic and clinical science in order to improve patient care. His research focuses on the concept of obtaining a molecular definition of a tumor to define its treatment-sensitive elements, complementing his long-standing interest in the fields of cancer genetics, molecular targeted therapeutics, predictive biomarkers, early detection, and biobanking. Dr. Godwin obtained his PhD in Molecular Biology from the University of Pennsylvania while carrying out his thesis research at Fox Chase Cancer Center (FCCC) in Philadelphia. Dr. Godwin was recruited to KUMC as the Associate Director for Translational Research for the Cancer Center in 2010 from FCCC following 26 productive years. He was appointed Deputy Director of the Cancer Center in 2013 and serves as professor of Pathology and Laboratory Medicine and director of Molecular Oncology at the Medical Center. He was named a Kansas Bioscience Authority Eminent Scholar in 2010, the University of Kansas School of Medicine Chancellor’s Distinguished Chair in Biomedical Sciences Endowed Professor in 2012. He leads the personalized medicine initiative across the KU Health System and KUMC.

Dr. DixonMichael Dixon, Ph.D., President and CEO, UNeMed Corporation, University of Nebraska Medical Center, USA

Role of Intellectual Property in Drug Discovery and Development

Michael Dixon, Ph.D., is president and CEO of UNeMed, the University of Nebraska Medical Center’s technology transfer office. UNeMed helps faculty, students and staff commercialize their innovative ideas to improve public health for Nebraska residents and beyond. Dr. Dixon and the UNeMed staff work to match industry, entrepreneurs and investors with university researchers to foster partnerships for the commercial development of a new technology. Dr. Dixon’s tenure at UNMC began in 1998 when he joined the Eppley Institute for Research in Cancer and Allied Diseases. Dr. Dixon’s research interests at UNMC were focused in the areas of molecular biology, genetics and biochemistry. In 2003 he transitioned from the bench to the business side of science when Dr. Dixon joined the UNMC technology transfer office, and began working with researchers to protect and develop new technology. As president and CEO of UNeMed, Dr. Dixon is responsible for setting UNeMed’s strategic path and directing UNeMed’s activities, including efforts to protect, market and license new technology. Dr. Dixon is a graduate of Leadership Omaha Class 32 and a 2011 recipient of the Midlands Business Journal “40 under 40” award. As an active member of the community, Dr. Dixon serves on several Boards, including Bio Nebraska, a nonprofit trade association dedicated to the development and growth of Nebraska’s bioscience industry.

Dr. Timothy MillerTimothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics Inc ($ABEO)

Developing next generation gene therapy products for rare disease treatments

Timothy J. Miller, PhD, became our President and Chief Executive Officer and Director on May 15, 2015. Dr. Miller was President & CEO of Abeona Therapeutics LLC from 2013 to 2015. He has 16 years of scientific research, product development, regulatory and clinical operations expertise, with a focus on transitioning novel biotherapeutics through pre-clinical phases and into Phase 1 and 2 human clinical trials. Dr. Miller was President & CEO of Red5 Pharmaceuticals from 2013 until 2015 and was Vice President, Business Development of BioEnterprise Inc in 2015. He was Senior Director of Product Development at SironRX Therapeutics from 2010 to 2013. Between 1996 and 2010 Dr. Miller held various positions at several companies focusing on gene therapy and regenerative medicine. Dr. Miller earned his PhD in Pharmacology with a focus on Gene therapy/Cystic Fibrosis from Case Western University. He also holds a B.S. in Biology and M.S. in Molecular Biology from John Carroll University (Cleveland, OH).