Nanomedicines: Addressing the Scientific and Regulatory Gap

Speakers

Organizers

Raj Bawa, MS, PhD

Bawa Biotech LLC and Rensselaer Polytechnic Institute

Dr. Raj Bawa is president of Bawa Biotech LLC, a biotech/pharma consultancy and patent law firm he founded in 2002. Trained as a biochemist and microbiologist, he is an inventor, entrepreneur and a registered patent agent licensed to practice before the US Patent & Trademark Office. He has extensive expertise in pharmaceutical sciences, biotechnology, nanomedicine, drug delivery, medical devices and biodefense-related scientific, FDA regulatory and patent law issues. Since 1999, he has held various adjunct faculty appointments at Rensselaer Polytechnic Institute in Troy, NY where he currently is an adjunct professor of biology. Since 2004, he has been an adjunct associate professor of natural and applied sciences at the Extended Learning Institute of NVCC in Annandale, VA. Since 2012, he has been a scientific advisor to Teva Pharmaceutical Industries, Ltd., Israel. He previously served as patent legal advisor at Sequoia Pharmaceuticals, Gaithersburg, MD and as senior scientist at SynerGene Therapeutics, Inc., Potomac, MD. He recently served as principle investigator of two National Cancer Institute/SBIR contracts titled "Targeted nanocomplexed iron oxide for early detection with concurrent hyperthermia treatment of cancer" and "A targeted nanocomplex for early detection of lung cancer." In the 1990s, Dr. Bawa held various positions at the US Patent and Trademark Office, including primary examiner (6 years) and instructor at the US Patent Academy. He is a life member of Sigma Xi, founding director of the American Society for Nanomedicine, co-chair of the Nanotech Committee of the American Bar Association and Global Advisory Council Member of the World Future Society. He has authored over 100 publications, co-edited three texts, presented or chaired at over 200 conferences worldwide and serves on the editorial boards of 12 peer-reviewed journals.

Melanie Brickman Stynes, PhD, MSc

The New York Academy of Sciences

Scott E. McNeil, PhD

Nanotechnology Characterization Laboratory – Frederick National Laboratory for Cancer Research

Melinda Miller, PhD

The New York Academy of Sciences

Stefan Mühlebach, PhD

Vifor Pharma Ltd. and University of Basel

Keynote Speaker

Sally Tinkle, PhD

IDA/Science and Technology Policy Institute

Dr. Sally Tinkle joined the Science and Technology Policy Institute (STPI) in Washington, DC in April 2013 in order to bring her expertise in human health research, policy, and administration, especially as it relates to emerging technologies and environmental exposures, to the policy arena. Before joining STPI, she served as the Deputy Director of the National Nanotechnology Coordination Office of the National Science and Technology Council where she led strategic planning and implementation of the National Nanotechnology Initiative, especially for environmental, health, and safety research. As a Senior Science Advisor in the Office of the Deputy Director, National Institute of Environmental Health Sciences (NIEHS), National Institutes of Health (NIH), Dr. Tinkle worked on health issues related to biofuels and the bioeconomy, application of global earth observations to human health monitoring, environmentally-induced pulmonary health conditions, as well as nanotechnology. Dr. Tinkle also served as a research laboratory Team Leader at the National Institute of Occupational Safety and Health, focusing on the relationship of skin exposure to the development of occupational lung disease. Dr. Tinkle received her PhD from the Department of Physiology at the University Of Colorado School Of Medicine and was a postdoctoral fellow at the National Jewish Center for Immunology and Respiratory Medicine, Department of Occupational and Environmental Health Science, Denver, Colorado.

Featured Speakers

Raj Bawa, MS, PhD

Bawa Biotech LLC and Rensselaer Polytechnic Institute

Yechezkel (Chezy) Barenholz, PhD

Hebrew University-Hadassah Medical School

Professor Barenholz (Daniel G. Miller Professor in Cancer Research) received his PhD at the Hebrew University-Hadassah Medical School, Jerusalem in 1971. He has been on the faculty of the Hebrew University since 1968 and was promoted to a Professor in 1981. He was a Visiting Professor it the Department of Biochemistry, University of Virginia School of Medicine, Charlottesville VA, USA from 1973 to 2005. He has been a Visiting Professor at the following universities: University of Utrecht, The Netherlands, 1992; the University of Kyoto, Japan, 1998; La Sapeinza University, Rome, 2006; Jaiotung University, Shanghai, China, 2006; Kings College, University of London, UK, 2006; and, the Danish Technical University DTU, Copenhagen, 2010. The basic research of Professor Barenholz focuses on the biophysics of lipid assemblies such as liposomes and micelles, and on the composition-structure-function relationships of biological membranes (with special focus and contributions related to sphingolipids).

His applied research centers around the development of drug delivery systems (DDS) and drugs based on such DDS including low molecular weight anti-cancer, anti-inflammatory, and local anesthetic drugs, as well as delivery systems for peptides, proteins, nucleic acids, and vaccines. This is exemplified by Doxil®, which was based on his invention and was developed to an FDA- and world-wide-approved anti-cancer drug by Professor Barenholz together with the oncologist Professor Alberto Gabizon, and SEQUUS Pharmaceuticals, Menlo Park CA, USA. Doxil® (Caelyx® in Europe) is the first FDA-approved nano drug and the first FDA-approved liposomal drug (1995). It is distributed today all over the world by Johnson and Johnson. Doxil sales exceeds half a billion dollars a year. Professor Barenholz, with the help of others, based on his inventions, founded the following start-up companies: 1. NasVax Ltd (now a public company on the Israeli stock market), a vaccine developing company, based on, among others VaxiSomeTM, a Barenholz-invented polycationic sphingolipid adjuvant; 2. Moebius Medical, which develops a liposome-based medical device for treatment of osteoarthritis. Moebius finished successfully her first clinical trial and are now preparing for a large pivotal clinical trials; LipoCure Ltd for the development of liposomal nano drugs based on Professor Barenholz' inventions for treatment of cancer and inflammatory diseases [rheumatoid arthritis (RA) and multiple sclerosis (MS)], as well as for special liposomes remote loaded with local anesthetics for prolonging analgesia duration. Two of the liposomal drugs under development in LipoCure are in final preparation for clinical trials.

Professor Barenholz is a coauthor of more than 360 scientific publications having altogether more than 10,000 citations. He is a co-inventor in more than 30 approved patent families. He was an executive editor of Progress in Lipid Research, an editor of 4 Special Issues, and is on the editorial board of 5 scientific journals.

Professor Barenholz was awarded the following prizes and awards: the Donders Chair Professor at the Faculty of Pharmacy, University of Utrecht, The Netherlands {1992); the Kaye award for innovation, twice (1995 & 1997) at the Hebrew University, Jerusalem, Israel; the international Alec D. Bangham (the founder of the Liposome field) award (1998); the Teva Founders Prize (2001), Israel; an Honorary Doctor degree for "outstanding contributions to lipid membrane research and highly innovative achievements in nanomedicine" from the Technical University of Denmark (DTU) in 2012, (Copenhagen, Denmark); and the international Controlled Release Society's (CRS) most prestigious CRS Founders Award for 2012. In 2003 Professor Barenholz founded (from Doxil royalties) the "Barenholz Prizes" for Israeli PhD students to encourage excellence and innovation in applied science. Professor Barenholz is married to Dr. Hanna Barenholz together they share 4 daughters and 12 grandchildren.

Gerrit Borchard, PharmD, PhD

University of Geneva, University of Lausanne

Gerrit Borchard is a licensed pharmacist and obtained his PhD in pharmaceutical technology from the University of Frankfurt (Germany) for his thesis on the interaction of colloidal drug carrier systems with the immune system. After holding several academic posts, including a lecturer position at Saarland University (Germany) and Assistant and Associate Professorships at Leiden University (The Netherlands), he joined Enzon Pharmaceuticals, Inc. (USA) as Vice President Research. In 2005, he was appointed Full Professor of Biopharmaceutics at the University of Geneva (Switzerland), and Scientific Director of the Centre Pharmapeptides in Archamps (France), an international center for biopharmaceutical research and training.

In the past, Prof. Borchard has served as Scientific Advisor for the Controlled Release Society (CRS), as Scientific Secretary of the European Association of Pharmaceutical Biotechnology (EAPB), and has headed the Academic Section of the International Association for Pharmaceutical Technology (APV). Since 2008, he served as Vice President of the School of Pharmaceutical Sciences Geneva-Lausanne (EPGL) and since 2013 as president. In 2012 Prof. Borchard joined the Non Biological Complex Drugs (NBCD) working group hosted at Top Institute Pharma (TIP, Leiden, The Netherlands) and was nominated Chair of the NBCD working party at the European Directorate for the Quality of Medicines & Health Care (EDQM) by Swissmedic.

In 2010, Prof. Borchard became a member of the Swiss Academy of Pharmaceutical Sciences (SAPhS). He was nominated Fellow of the Swiss Society of Pharmaceutical Sciences (SSPhS) in 2010, and President of SSPhS in 2012. In 2013, he was elected Vice President to the Executive Committee of the European Federation of Pharmaceutical Sciences (EUFEPS).

Due to his working in both academia and industry, and living in four countries, Prof. Borchard has acquired extensive experience in diverse working and cultural environments, and is fluent in the German, English, Dutch and French languages. Time allowing, he loves to roam the trails and by-roads of the Jura mountains on foot and bike.

Ricardo Carvajal, JD

Hyman, Phelps & McNamara, P.C.

Mr. Carvajal provides FDA and FTC regulatory counseling and litigation support to manufacturers and marketers of foods (including dietary supplements and medical foods), cosmetics, and OTC drugs. He has substantial experience with all food and dietary supplement issues, including recalls, Reportable Food Registry issues, GMP and HACCP compliance issues, and implementation of the Food Safety Modernization Act. He also has substantial experience with labeling and advertising issues, including those that arise from the use of health, nutrient content, structure/function, and disease claims. Mr. Carvajal counsels product developers on regulatory strategy, including requirements pertaining to self-determinations of GRAS status and determinations of new dietary ingredient status. From 2002 to 2007, Mr. Carvajal served as Associate Chief Counsel in FDA's Office of Chief Counsel, where he counseled the agency on regulatory issues arising in the context of foods produced through biotechology and nanotechnology. He currently serves on the Editorial Advisory Board for the Food and Drug Law Institute Monographs, and served as Chair of the Public Policy Outreach and Implementation Task Force of the Institute of Food Technologists.

Neil Desai, PhD

Abraxis Bioscience, A wholly owned subsidiary of Celgene Corporation

Neil Desai is currently Vice President of Strategic Platforms at Celgene Corp. Prior to its acquisition by Celgene in Oct 2010, he was Sr. Vice President of Global Research and Development at Abraxis Bioscience, in Los Angeles, California, USA, where he led the development of Abraxane®, the company's flagship product and considered to be the first true nanotherapeutic. Dr. Desai is an inventor of Abraxis' nanoparticle-albumin bound (nab®) drug delivery platform and was responsible for company's product pipeline and the development its intellectual property portfolio. This platform has been clinically proven with global approvals for Abraxane® in metastatic breast cancer and most recently for pancreatic cancer. Prior to his positions at Abraxis, Dr. Desai was Senior Director of Biopolymer Research at VivoRx, Inc and VivoRx Pharmaceuticals, Inc. (predecessor companies of Abraxis), where he worked on the early discovery and development of Abraxane, developed novel encapsulation systems for living cells and was part of the team that performed the world's first successful encapsulated islet cell transplant in a diabetic patient. Dr. Desai has more than 25 years of experience in the research and development of novel therapeutic delivery systems with over 100 issued patents and peer-reviewed publications. He is a reviewer for several scientific journals in the area of cancer therapeutics and drug delivery. He is an active participant in FDA and EU Nanotechnology initiatives and a member of the Steering Committee for the NCI Alliance for Nanotechnology in Cancer. Dr. Desai holds an MS and PhD in Chemical Engineering from the University of Texas at Austin, USA, and a BS in Chemical Engineering from the University Institute of Chemical Technology in Mumbai, India.

Rogério Gaspar, PhD

University of Lisbon

Rogério Gaspar (born 1961), Currently a Full Professor and Vice-Rector at the University of Lisbon. Since 2007 served as Head of its Pharmaceutical Technology Department and of the research group Nanomedicine & Drug Delivery Systems group at the Research Institute for Medicines and Pharmaceutical Sciences (iMed.UL, co-founded in 2007). In 2012/2013 served also as Vice-Dean at the FFUL. Rogério Gaspar has (2009-2013) served as a member of the Executive Committee of the European Federation for Pharmaceutical Sciences (EUFEPS), Vice-President (Science Policy & Regulatory Science) in 2011-2012. He was involved at different levels on the European Regulatory System for Medicinal Products. From 1995 as an expert and member of the national evaluation board and of the CPMP (now CHMP, at European Medicines Agency, EMA); between January 2000 to July 2002 he was Vice-chairman of the Management Board of INFARMED (Portuguese medicines regulator) and member of the Management Board of the European Medicines Agency (EMEA) - also member of committees and expert groups at the European Council of Ministers and European Commission. From 2002 to 2008 was a external consultant within a pharmaceutical company and consultant to ASEAN countries (2005-2006 in a EU cooperation programme). From 2008 to 2011 he returned to regulation at the Portuguese national regulatory authority in medicines (INFARMED), and in 2009/11 as invited member of the newly formed Ad-hoc expert group in Nanotechnology of the European Medicines Agency. Also involved in the several Nanomedicine coordination activities of the European Science Foundation (ESF) as expert or group member (2003-2011).  Current research interests of his group regard the development of new therapeutic strategies using liposomes, polymeric biodegradable nanoparticles, and polymer therapeutics. His main focus in research is currently oriented towards cytosolic delivery of nucleic acids and use of targeted delivery systems for combination therapy in cancer. Is research group is currently focused in aspects related to intracellular trafficking modulation for advanced drug delivery.

Patrick Hunziker, MD

University Hospital Basel

Patrick Hunziker has studied Medicine the University of Zurich, Switzerland. He received a doctoral decree based on thesis work in experimental immunology from the University of Zurich and did further research in experimental haematology at University Hospital in Zurich, Switzerland. He earned specialist degrees in Internal Medicine, Cardiology and Intensive Care Medicine. As a fellow the Massachusetts General Hospital, Harvard Medical School, worked on cardiac imaging in a joint project with the Massachusetts Institute of Technology, Cambridge. His professional activities in Europe, the U.S., Africa and China gave him a broad insight into the needs for the medicine of the future in a variety of settings.

Hunziker became involved in medical applications of Nanoscience in the late nineties and has been the pioneer physician in Nanomedicine in Switzerland since then. With improved prevention, diagnosis and cure of cardiovascular disease as his main research topic, he worked in the nanoscience fields of atomic force microscopy, nanoptics, micro/nanofluidics, nanomechanical sensors and polymer nanocarriers for targeting. He is since 2007 the founding president of the European Society of Nanomedicine, cofounder of the European Foundation for Clinical Nanomedicine and co-initiator of the European Conference for Clinical Nanomedicine and is clinically active as deputy head of the Clinic for Intensive Care Medicine at the University Hospital Basel, Switzerland. In November 2008 Patrick Hunziker became professor for Cardiology and Intensive Care Medicine at the University of Basel. 2011 he became President of the International Society for Nanomedicine.

Laurent Levy, PhD

Nanobiotix

Dr. Laurent Levy has over 20 years' experience in nanomedicine, supported by a solid understanding of physics, chemistry and biology. Currently, he is the VP of the ETP Nanomedicine. He obtained his doctorate in nanotechnology from the Pierre & Marie Curie University and the CEA, France, before pursuing a post-doctoral fellowship at the Institute for Lasers, Photonics and Biophotonics at the State University of New York, USA.

Dr. Levy's cutting-edge biotechnology and nanotechnology research culminated in the development of a nanomedicine platform (NanoXray) to address the biggest drawbacks of radiotherapy caused by the high radiation doses necessary to destroy tumor targets. In 2003, he co-founded Nanobiotix, a nanomedicine company pioneering novel approaches for the local treatment of cancer. Prior to Nanobiotix, Dr. Levy was a consultant in the development of nanotechnology applications for pharma companies (Guerbet, Rhodia) in addition to biotechnology start-ups.

Scott E. McNeil, PhD

Nanotechnology Characterization Laboratory – Frederick National Laboratory for Cancer Research

Dr. McNeil serves as the Director of the Nanotechnology Characterization Laboratory (NCL) for SAIC-Frederick and Frederick National Laboratory for Cancer Research, where he coordinates preclinical characterization of nanotech cancer therapeutics and diagnostics. At the NCL, Dr. McNeil leads a team of scientists responsible for testing candidate nanotech drugs and diagnostics, evaluating safety and efficacy, and assisting with product development -- from discovery-level, through scale-up and into clinical trials. NCL has assisted in characterization and evaluation of nearly 300 nanotechnology products, several of which are now in human clinical trials. Dr. McNeil is a member of several working groups on nanomedicine, environmental health and safety, and other nanotechnology issues. He is an invited speaker to numerous nanotechnology-related conferences and has several patents pending related to nanotechnology and biotechnology. He is also a Vice President of SAIC-Frederick.

Prior to establishing the NCL, he served as a Senior Scientist in the Nanotech Initiatives Division at SAIC where he transitioned basic nanotechnology research to government and commercial markets. He advises industry and State and US Governments on the development of nanotechnology and is a member of several governmental and industrial working groups related to nanotechnology policy, standardization and commercialization. Dr. McNeil's professional career includes tenure as an Army Officer, with tours as Chief of Biochemistry at Tripler Army Medical Center, and as a Combat Arms officer during the Gulf War. He received his bachelor's degree in chemistry from Portland State University and his doctorate in cell biology from Oregon Health Sciences University.

Stefan Mühlebach, PhD

Vifor Pharma Ltd. and University of Basel

Stefan Mühlebach, is Scientific Director (Global Regulatory Affairs) at Vifor Pharma Ltd in Switzerland, and Chair of the Non-Biological Drugs Working Party in Leiden, the Netherlands. He obtained a diploma in pharmacy (MSc Pharm) at the University of Bern in Switzerland in 1975 and specialised in hospital pharmacy. He has a PhD in pharmacology and toxicology (1979) and became a lecturer (venia docendi) in pharmacology at the Medical Faculty of the University of Bern in 1993 and since 2000 at the Medical Faculty of the University of Basel.

He was appointed Professor of Pharmacology and Hospital Pharmacy at the University of Basel in 2004. He is a group member of the Clinical Pharmacy & Epidemiology Unit in the Department of Pharmaceutical Sciences. Stefan Mühlebach's professional career has spanned over 30 years, heading Hospital Pharmacies in Biel and Aarau, and then working for Swissmedic, the Swiss Agency for Therapeutic Products (2005-2008) and Vifor Pharma Ltd (since 2008) as Head of Pharmacopoeia and Scientific Officer, respectively. He was appointed Chair of the NBCD working group at TIPharma (The Netherland) in 2010.He continued university teaching and research in addition to his professional functions and has produced over 50 peer-reviewed publications.

Ritu Nalubola, PhD

US Food and Drug Administration

Ritu Nalubola, PhD is a Senior Policy Advisor in FDA’s Office of Policy, Office of the Commissioner. She advises senior leadership at FDA on complex and cross-cutting policy issues, including nanotechnology, genetic engineering, food safety, and nutrition. Her areas of expertise include emerging technologies, novel science-policy issues, food regulation and policy, and international consensus-based standards. In her current role, she leads the development and coordination of the Agency’s regulatory policy activities relevant to the application of nanotechnology in FDA-regulated products. Dr. Nalubola also represents FDA in nanotechnology dialogue at various domestic and international policy forums. Prior to joining the Office of the Commissioner, Dr. Nalubola worked at FDA’s Center for Food Safety and Applied Nutrition on a diverse range of food policy issues related to nutrition, labeling, food safety, and CODEX guidelines. She also worked with the US Agency for International Development on international public health issues before starting her career at FDA in 2000.

Lawrence Tamarkin, PhD

Cytimmune

Dr. Tamarkin has lead CytImmune from its founding in 1988 as a diagnostic company to its current focus on cancer therapeutics. Dr. Tamarkin is the co-inventor of the gold nanoparticle-based, tumor-targeted platform technology, which is covered in 49 allowed and 42 pending patents both domestically and internationally. The Company's first cancer nanomedicine, CYT-6091 (Aurimune), which targets and destroys cancer blood vessels, has been successfully tested in a Phase I advanced-stage cancer patient study, and Phase II testing in combination with approved chemotherapies is planned. Recognizing that cancer isn't a single disease, under Dr. Tamarkin's leadership a pipeline of nanotherapeutics is being developed, and the second-in-a-family of cancer nanomedicines, CYT-20000 (AuriTol) adds an analog of Taxol to the CYT-6091 platform. Dr. Tamarkin succeeded in leading CytImmune to enter into an agreement with AstraZeneca to rescue an AstraZeneca proprietary cancer drug using the CYT-6091 platform and the chemistries used for CYT-20000. Dr. Tamarkin was graduated from SUNY at Stony Brook receiving a BS degree and received his PhD degree from the University of Connecticut.