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Nanomedicines: Addressing the Scientific and Regulatory Gap

Nanomedicines: Addressing the Scientific and Regulatory Gap

Thursday, November 21, 2013

The New York Academy of Sciences

Presented By

Presented by the New York Academy of Sciences


Nanomedicine is the application of nanotechnology for the treatment, diagnosis, monitoring, and control of biological systems. Nanomedicines hold great promise for addressing some of the most challenging problems in nearly every medical specialty, and this nascent field has the potential to generate many new opportunities for improving human health. However, there are also concerns that the fundamentally different properties of nanoparticles compared with bulk materials may pose significant safety issues, and therefore require additional regulatory scrutiny.

As such, this conference will focus on the following topics:

  • Current US and international regulatory frameworks for nanomedicines, and the future needs ahead;
  • Additional safety and toxicity research needed to determine unknown properties of nanomaterials; and
  • Lessons learned from featured drug development case studies of nanomedicines.

The conference will conclude with a panel discussion to bring together both scientific and regulatory perspectives to help guide future communication and action.

Early Bird registration has been extended until October 31.

Registration Pricing

By 10/31/2013After 10/31/2013Onsite
Student/Postdoc Member$40$50$60
Nonmember (Academia)$125$150$175
Nonmember (Corporate)$220$260$295
Nonmember (Non-profit)$125$150$175
Nonmember (Student / Postdoc / Fellow)$40$50$60

Presented by

  • The New York Academy of Sciences

Platinum Sponsor


* Presentation titles and times are subject to change.

November 21, 2013

8:00 AM

Breakfast and Registration

9:00 AM

Opening Remarks

9:15 AM

Keynote Address: The Evolving Role of Government and Scientist in Filling the Scientific and Regulatory Gaps in Nanomedicine
Sally Tinkle, PhD, IDA/Science and Technology Policy Institute

Session 1: Characterization of Nanomedicines: Techniques, Tools and Assessments

Chair: Raj Bawa, MS, PhD, Bawa Biotech LLC and Rensselaer Polytechnic Institute

10:00 AM

Characterization and Safety of Nanomedicines: Lessons Learned from the NCL
Scott E. McNeil, PhD, Nanotechnology Characterization Laboratory — Frederick National Laboratory for Cancer Research

10:30 AM

"Nano-similars" and Follow-On Nano-Sized Therapeutics
Stefan Mühlebach, PhD, Vifor Pharma Ltd. and University of Basel

11:00 AM

Networking Coffee Break

Session 2: Regulation of Nanomedicines

Chair: Stefan Mühlebach, PhD, Vifor Pharma Ltd. and University of Basel

11:30 AM

FDA's Approach to Regulation of Nanotechnology Products
Ritu Nalubola, PhD, US Food and Drug Administration

12:00 PM

Nanomedicines: EMA Guidance
Rogério Gaspar, PhD, University of Lisbon

12:30 PM

Nanodrugs in the Post-Blockbuster World — Commercialization and Critical Patent Issues
Raj Bawa, MS, PhD, Bawa Biotech LLC and Rensselaer Polytechnic Institute

1:00 PM

Networking Lunch

2:15 PM

Panel Discussion
Global Regulation of Nanomedicines: Can We Harmonize the Guidelines?

Moderator: Stefan Mühlebach, PhD, Vifor Pharma Ltd. and University of Basel

Raj Bawa, MS, PhD, Bawa Biotech LLC and Rensselaer Polytechnic Institute
Gerrit Borchard, PhD, University of Geneva, University of Lausanne
Ricardo Carvajal, JD, Hyman, Phelps & McNamara P.C.
Rogério Gaspar, PhD, University of Lisbon
Ritu Nalubola, PhD, US Food and Drug Administration
Sally Tinkle, PhD, IDA/Science and Technology Policy Institute

Session 3: Nanomedicines from Research to Clinic: Lessons from Case Studies

Chair: Scott E. McNeil, PhD, Nanotechnology Characterization Laboratory — Frederick National Laboratory for Cancer Research

2:45 PM

Doxil, The First FDA Approved Nano-Drug: Lessons Learned
Yechezkel (Chezy) Barenholz, PhD, Hebrew University-Hadassah Medical School

3:15 PM

TNF-PEGylated Gold Nanoparticles: A Platform for a Family of Nanomedicines
Lawrence Tamarkin, PhD, Cytimmune

3:45 PM

Networking Coffee Break

4:15 PM

Albumin-bound Nanoparticles: What Did We Learn?
Neil Desai, PhD, Abraxis Bioscience, A wholly owned subsidiary of Celgene Corporation

4:45 PM

Combination Nanotechnology: Drugs or Medical Devices?
Laurent Levy, PhD, Nanobiotix

5:15 PM

Panel Discussion
How to Accelerate the Development of Nanomedicines

Moderator: Scott E. McNeil, PhD, Nanotechnology Characterization Laboratory — Frederick National Laboratory for Cancer Research

Yechezkel (Chezy) Barenholz, PhD, Hebrew University-Hadassah Medical School
Neil Desai, PhD, Abraxis Bioscience, A wholly owned subsidiary of Celgene Corporation
Patrick Hunziker, MD, University Hospital Basel
Laurent Levy, PhD, Nanobiotix
Lawrence Tamarkin, PhD, Cytimmune

5:45 PM

Closing Remarks

6:00 PM




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


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


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.


Keynote: The Evolving Role of Government and Scientist in Filling the Scientific and Regulatory Gaps in Nanomedicine
Sally Tinkle, PhD, IDA/Science and Technology Policy Institute, Washington, District of Columbia

Medical research encompasses biology and bioengineering, research tools and methods, therapeutics and diagnostics. Nanoscience and nanotechnology focus on the understanding and control of matter at the nanoscale, including application-specific top-down / bottom-up design and engineering of nanoscale materials, systems, and devices. Nanomedicine lies at the intersection of these disciplines, bringing the power of advanced materials with novel size-dependent properties to the maintenance, diagnosis, and restoration of human health. The inherent multidisciplinary nature of nanoscale science and technology and the breadth of the fundamental research needed to realize the promise of these advanced materials has challenged the federal R&D enterprise and the scientific communities to reshape their relationships and business models. Within the context of an increasingly complex nanomedicine product pipeline, scientists, engineers, and regulators grapple with the application of existing regulations to nanoscale materials and devices with complex physical and chemical properties that challenge regulatory definitions. Federal efforts to organize nanotechnology under the National Nanotechnology Initiative have proven useful where they align with existing agency missions and operating procedures, and in certain areas and under certain conditions, provided momentum and rationale for change. It is against this backdrop that the scientific and regulatory gaps in nanomedicine can be identified and strategies and responsibilities for bridging gaps created.

Characterization and Safety of Nanomedicines: Lessons Learned from the NCL
Scott E. McNeil, PhD, Nanotechnology Characterization Laboratory (NCL) - Frederick National Laboratory for Cancer Research, Frederick, Maryland

The Nanotechnology Characterization Laboratory (NCL) at the US Frederick National Lab for Cancer Research (FNL) conducts preclinical efficacy and toxicity testing of nanoparticles intended for cancer therapeutics and diagnostics. The NCL is a partnership among the National Cancer Institute (NCI), the US Food and Drug Administration (FDA) and the National Institute of Standards and Technology (NIST). As part of its assay cascade, NCL characterizes nanoparticles' physical attributes, their in vitro biological properties, and their in vivo compatibility using animal models. The NCL also looks at trends across nanoparticle platforms, parameters that are critical to nanoparticle biocompatibility, and develops assays for preclinical characterization of nanoparticles. The NCL has developed more than 40 protocols that rigorously characterize nanoparticle physicochemical properties, as well as in vitro immunological and cytotoxic characteristics and ADME/Tox profiles in nonhuman animal models. These assays have undergone extensive in-house validation and are subjected to regular revision to ensure applicability to a variety of nanomaterials. The NCL has assessed more nearly 300 nanoparticles, including liposomes, dendrimers and other polymers, quantum dots, gold colloids, metal oxides, and fullerenes. This presentation will provide an overview of the NCL, discuss observed trends, and illustrate how physical parameters influence nanoparticle biocompatibility and toxicity.
Funded by NCI Contract # HHSN261200800001E.

“Nano-similars” and Follow-On Nano-Sized Therapeutics
Stefan Mühlebach, PhD, Vifor Pharma Ltd, Glattbrugg, Switzerland; Basel University, Basel, Switzerland

The generic paradigm and the biosimilar approach have been defined for the follow-on products of fully characterized small molecule drugs and the biologicals, respectively. A new class of non-biological complex drugs (NBCDs), which also includes nanomedicines, has emerged. NBCDs are medicinal products, not being a biological medicine, where the active substance is not a homo-molecular structure, but consists of different (closely related and often nanoparticulate) structures that can’t be isolated and fully quantitated, characterized, and/or described by physicochemical analytical means.
Therapeutic equivalence of NBCD follow-on products, including nanosimilars, can’t be assessed using the standard generic or the biosimilar approach. Generic drugs with low molecular weight have to show pharmaceutical identity and bioequivalence, i.e., comparable drug concentration vs. time curves in the plasma, representing the central compartment. Biosimilars have to be biologics. Intravenous nanocolloidals, like iron carbohydrates, liposomes, or glatiramoids have been used therapeutically for many years; long before the term ‘nanomedicines’ was coined. To obtain market authorization after patent expiry, intended follow-on copies have to be assessed by regulators for comparability. For nanomedicines resulting from a proprietary, laborious, and difficult to control manufacturing process, even minor differences can be clinically meaningful, even if not known or assessed by up-to-date physicochemical testing as shown for NBCDs. Such similar but not the same therapeutics need a stepwise regulatory approach requiring quality, non-clinical, and clinical data. Only this totality of evidence will finally guarantee therapeutic equivalence, a prerequisite for interchangeability and substitutability between a follow-on and the referenced (nanomedicine) therapeutic. The regulatory challenge is reflected by the proposals for discussion issued by EMA and FDA. Filling the gaps in our scientific insight is key to a science-based harmonized regulatory authorization procedure for NBCDs/ nanosimilars and will also impact their post-marketing surveillance policies.

FDA’s Approach to Regulation of Nanotechnology Products
Ritu Nalubola, PhD, US Food and Drug Administration, Silver Spring, Maryland,

The US Food and Drug Administration (FDA) regulates a broad range of products under the Federal Food, Drug, and Cosmetic Act (FFDCA) and other statutes. FDA is generally responsible for overseeing the safety and, as applicable, effectiveness of a wide range of products, including drugs, devices, foods, veterinary products, and cosmetics. Nanotechnology allows scientists to create, explore, and manipulate materials measured in nanometers (equal to one-billionth of a meter). It is an emerging technology that has the potential to be used in a broad array of FDA-regulated products, including medical products. Materials at the nanoscale can have different chemical, physical, or biological properties compared to their conventionally-scaled counterparts. FDA continues to regulate nanotechnology products under its existing statutory authorities, in accordance with the specific legal standards applicable to each type of product under its jurisdiction. FDA does not make a categorical judgment that nanotechnology is intrinsically safe or harmful. Rather, for nanotechnology-derived and conventionally-manufactured products alike, FDA considers the characteristics of the finished product and, as applicable, its safety, effectiveness, or other product attributes. Scientific considerations and broad policy issues relevant to FDA’s regulation of nanotechnology products, including FDA’s draft guidance to industry, will be discussed.

Nanomedicines: EMA Guidance
Rogério Gaspar, PhD, University of Lisbon, Lisbon, Portugal

Current developments in the research landscape of nanomedicines have brought attention to the fact that, as an already well-established area of clinical practice, nanomedicines now face some questions previously addressed by new chemical entities and biologicals. Advances brought by nanomedicines in oncology and infectious diseases are expanding within these clinical areas, as well as in other less targeted clinical situations. The advances in clinical practice associated with nanomedicines in the last 30 years will allow for significant improvements in the next phase, sustained by both solid basic research and an increased amount of clinical data compiled across different technologies and therapeutic areas. New nanomedicine developments in both personalized medicine and combination therapy are drivers for innovation in clinical practice. The innovation in materials science must meet clinical standards established for previously approved medicinal products that went through the challenge of regulatory approval for both clinical experiences (under clinical trials), and also for marketing authorization and routine clinical use. Meanwhile need for adequate standardization and characterization has moved the US to a specific platform initiative (NCL at NCI) and Europe is still looking for a more integrated way to address it.
New challenges in Regulatory Science will be met by better integration of materials science, by solving translational issues like validation of adequate models (e.g. preclinical human cells and tissues in appropriate setting to foster clinical translation and better outcomes within clinical phase), and by targeting adequate disease stage and disease evolution conditions within the current setting of trying to address appropriate personalized medicine questions. Nanomedicine is bringing converging sciences to an adequate platform of technologies that will provide better health care and enable the design and clinical use of innovative solutions for unmet clinical needs.
Regulatory framework in Europe and elsewhere is currently adjusting to new realities and incorporating the best scientific standards in anticipation of the regulatory needs for follow-on products, combination products and integrative platforms bringing together therapy and diagnostics. We envisage a near future where bridging diagnostics and therapeutics through nanotechnology-based tools brings the promise of personalized medicine as an attainable goal at an adequate cost for both society and patients.

Nanodrugs in the Post-Blockbuster World – Commercialization and Critical Patent Issues
Raj Bawa, MS, PhD, Bawa Biotech LLC, Ashburn, Virginia; Rensselaer Polytechnic Institute, Troy, New York

In today’s global economy, drug companies are under enormous pressure to maintain profitability in light of numerous challenges ranging from revenue losses due to patent expirations on blockbusters to enhanced regulatory oversight to an ever-increasing challenge from generic manufacturers. These market forces and drivers are rapidly dictating a change in pharma’s quest for discovering, developing, and delivering novel therapeutics. Due to this altered pharma landscape, new ground rules and competitive business strategies are needed in the post-blockbuster world. As a result, drug companies are turning to miniaturization and nanotechnology to enhance or supplement drug target discovery and drug development. Nanomedicine is part of this high risk, high payoff global phenomenon. For example, nanodrugs can increase half-life of conventional drugs by reducing immunogenicity and diminishing drug metabolism, advantages that could potentially extend the economic life of proprietary drugs and create additional revenue streams.
With this backdrop, my presentation will highlight, via pictures and animations:

  • size-driven innovations in pharma;
  • US Patent Office and critical issues, strategies and challenges relating to patenting nanodrugs – a primer for physicians and scientists;
  • issues resulting from the current “patent land grab” by “patent prospectors” in nanomedicine and the impact of emerging “patent thickets” on commercialization efforts;
  • overcoming “obviousness” when patenting nanomedicine inventions – The issue of size; and
  • impact and interplay of federal bodies (US FDA, US Patent Office, US Congress) on commercialization efforts.

Doxil, the First FDA Approved Nano-Drug: Lessons Learned
Yechezkel (Chezy) Barenholz, PhD, Hebrew University-Hadassah Medical School, Jerusalem, Israel

Doxil®, the first FDA-approved nano-drug (1995), is based on three unrelated principles: (i) prolonged drug circulation time and avoidance of the RES due to the use of PEGylated nano-liposomes; (ii) high and stable remote loading of doxorubicin driven by a transmembrane ammonium sulfate gradient, which also allows for drug release at the tumor; and (iii) having the liposome lipid bilayer in a “liquid ordered” phase composed of the high-Tm (53 ºC) phosphatidylcholine, and cholesterol. The latter helps to maintain the gradient and also enable to achieve a zero order slow drug release at the tumor site. Due to the enhanced permeability and retention (EPR) effect, Doxil is “passively targeted” to tumors and its doxorubicin is released and becomes available to tumor cells by as yet unknown means. Doxil success demonstrates the obligatory need for applying an understanding of the cross talk between physicochemical, nano-technological, and biological principles. However, in spite of the large reward, >3 years after Doxil-related patents expired there is only one FDA-approved generic “Doxil” available, the LipoDox. The reason for the lack of Doxil-like approved generics will be discussed in short.
In retrospective, it is obvious that in spite of Doxil’s success story it has few side effects (especially Foot and Hand syndrome) that, if overcome or reduced, will be highly beneficial and may improve Doxil performance. My short presentation will focus on novel ways to overcome the above side effects in order to improve the performance of pegylated liposomal doxorubicin (PLD).

TNF-PEGylated Gold Nanoparticles: A Platform for a Family of Nanomedicines
Lawrence Tamarkin, PhD and Giulio F. Paciotti, PhD, CytImmune, Rockville, Maryland

The use of nano-sized drug delivery systems to target potent, but toxic anticancer therapeutics to solid tumors is best accomplished by avoiding the drug’s uptake by the immune system and by limiting its biodistribution. Binding recombinant human tumor necrosis factor alpha (TNF) to the surface of 27 nm PEGylated colloidal gold particles (CYT-6091) meets these objectives. Each component serves a specific function. The gold nanoparticles limit biodistribution, while PEGylation prevents immune detection. TNF serves to actively localize nanoparticles to tumor, causing vascular disruption of the tumor blood supply. This action is why systemically administered TNF is clinically ineffective, since it causes vascular leak of healthy blood vessels. However, using the surgical procedure, isolated limb perfusion (ILP), infusion of native TNF followed by chemotherapy is dramatically effective in reducing tumors on arms and legs. CYT-6091 seeks to mimic the ILP clinical experience. In a Phase I clinical trial, systemically administered CYT-6091 was safe at potentially therapeutic levels of TNF. Core biopsies of tumor and healthy tissue revealed that CYT-6091 trafficked to tumors, not healthy organs. Mimicking the ILP protocol, CYT-6091 will be combined with standard of care chemotherapy in a Phase II clinical trial. However, the ideal nanomedicine delivers both TNF and chemotherapy to tumors. This design has been accomplished by binding an analog of paclitaxel to TNF-PEGylated gold nanoparticles (CYT-20000). While TNF on CYT-20000 enables rapid tumor targeting, paclitaxel is slowly released at the tumor site. These data highlight a tunable and scalable manufacturing process that is the basis for a family of nanomedicines.

Albumin-Bound Nanoparticles: What Did We Learn?
Neil Desai, PhD, Abraxis Bioscience, A wholly owned subsidiary of Celgene Corporation, Los Angeles, California

Nanotechnology is being increasingly applied to the area of drug development. Nanoparticle-based therapeutics can confer the ability to overcome biological barriers, effectively deliver hydrophobic drugs and biologics, and preferentially target sites of disease. However, despite these potential advantages, only a relatively small number of nanoparticle-based medicines have been approved for clinical use to date, with numerous challenges and hurdles at different stages of development. Albumin-bound nanoparticles (Abraxane®) have been successfully developed and commercialized and have proven their therapeutic advantage in diseases such as metastatic breast cancer and advanced pancreatic cancer. The presentation will cover aspects of the science, clinical results and development strategies with a view to identifying the important lessons learned during the development of Abraxane.

Combination Nanotechnology: Drugs or Medical Devices?
Laurent Levy, PhD, Nanobiotix, Paris, France; European Technology Platform of Nanomedicine, Berlin, Germany

The first Nanobiotix nanomedicine product has been classified as a drug in the US and as Medical Device in the EU. Is the status of a product, medical device or drug, key in the development of such nanomedicine? In fact no, status is “just” a regulatory environment for the development of a product, the nature and the targeted application of the nanomedicine will drive the assessment of benefit risk during development. Here a NanoXray specific case and lessons learned will be presented. First-in-class, the technology called NanoXray, is at the forefront of a new era of nanomedicine, where nanoparticles are not just a vehicle for targeted drug delivery, but have become the principal active element. The NanoXray technology is based on the physical properties of hafnium-oxide nanoparticles and is used to enhance the efficacy of radiotherapy treatment for a variety of cancer indications.
These nanoparticles are designed to enter tumor cells and, upon activation by a standard dose of radiation, they emit large amounts of electrons resulting in the generation of free radicals that destroy cancer cells (the same mode of action than radiotherapy but largely amplified). Nanoparticle-enhanced radiotherapy therefore amplifies the lethal dose of energy locally within the tumor without changing the effect of the dose passing through surrounding healthy tissues. The first product is currently under clinical investigation in Soft Tissue Sarcomas and Head and Neck cancer patients.

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