AIMBE/NIH Summit

Validation and Qualification of New In Vitro Tools and Models for the Preclinical Drug‑Discovery Process

March 19, 2012

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Co-hosted by AIMBE, NIH's National Institute of Biomedical Imaging and Bioengineering (NIBIB), FDA and NIST to ensure cross-agency collaboration

Focused on developing reliable guidelines for validating emerging in vitro technologies, aiming to accelerate their acceptance in preclinical drug development and regulatory review.

• Provided a structured framework for rigorous validation and qualification of in vitro tools, shaping subsequent advisory guidance from NIH, FDA, and NIST.

• Established crucial cross-sector dialogue—involving academia, regulators, and industry—to align on standards and accelerate scientific innovation.

2nd AIMBE Workshop

Validation and Qualification of New In Vitro Tools and Models for The Pre-clinical Drug Discovery Process

September 17-18, 2012

View Workshop Program:  2nd AIMBE Workshop program 9-13-12

Welcome Letter:

This Workshop is being sponsored by the American Institute for Medical and Biological Engineering (AIMBE). The overall goal of this series of workshops is to develop guidelines for investigators developing new systems for the pre-clinical drug development process on how to validate these new technologies so that they become useful, meaningful tools. Specific emphasis will be on model systems that may augment or replace existing models, especially animal models, in the FDA drug approval process. The agenda and goals for the first meeting, which covered current perspectives on validation as well as latest technologies for pre-clinical regulatory science can be found on AIMBE’s website (www.aimbe.org).

On day one of this workshop, the first session will review current perspectives on validation and qualification of new in vitro technologies from the perspective of the National Toxicology Program, the European Medicines Agency, and the National Institute for Standards and Technology (NIST). In the second session, an overview of new technologies that could be validated and qualified in the near future for use in pre-clinical drug discovery will be presented including “organ on a chip” grants recently funded by the NIH, a high content screening platform, a drug industry perspective of validation, and a laboratory-developed immune system for vaccines, that will be one of the technical evaluations on the second day.

On day two of the Workshop, drafting of validation and qualification guidelines will begin with talks on the two remaining technologies chosen as examples for evaluation. Session three will consist of brief overviews of the two remaining example technologies followed by a perspective on validation and qualification guidelines for each of these technologies by teams led by AIMBE fellows. Each overview will be followed by general audience participation for input on drafting guidelines for each of the three chosen technologies. It is expected that there will emerge common guidelines for validation and qualification among the different technologies but also some uniqueness for each. The fourth session will present the results of the general discussion to get final feedback from the Workshop participants and begin planning for the follow-on workshops.

We are looking forward to an interesting and productive meeting!

Sincerely,

Chair:

James J Hickman, PhD

AIMBE Board of Directors

Professor of Nanoscience, Technology, Chemistry, Biomolecular Science and Electrical Engineering

University of Central Florida

 Program Agenda

September  17         Day 1

September 18          Day 2

Speaker Biographies

James J. Hickman, Ph.D.

James J. Hickman is the Founding Director of the NanoScience Technology Center and a Professor of Nanoscience Technology, Chemistry, Biomolecular Science, Physics and Electrical Engineering at the University of Central Florida.  Previously, he held the position of the Hunter Endowed Chair in the Bioengineering Department at Clemson University. Dr. Hickman has a Ph.D. from the Massachusetts Institute of Technology in Chemistry, as well as BS and MS from Penn State University in Chemistry. For the past twenty years, he has been studying the interaction of biological species with modified surfaces, first in industry and in the latter years in academia. While in industry he established one of the first bioelectronics labs in the country that focused on cell-based sensors and their integration with electronic devices. He has extensive experience in surface modification and surface analysis for biological and neuroscience applications, and the integration of these systems with MEMS devices and components.  He is also interested in creating hybrid systems for biosensor and biological computation applications and the creation of functional in vitro systems. He has worked at the NSF part-time as a special advisor to the Experimental and Integrative Activities Division in CISE in the area of biological computation and previously had worked with the Information Technology Office at DARPA to help develop new programs in Ultrascale computing.  He is also the founder and current CTO of a biotechnology company, Hesperos, that is focusing on cell-based systems for high-throughput functional genomics and toxicity. He has 85 publications and 17 book chapters, in addition to 20 patents.  Dr. Hickman was elected a Fellow of the American Institute of Medical and Biomedical Engineers in 2004 (AIMBE) and a fellow of the AVS in 2007 and is currently a member of the Board of Directors for AIMBE. He was a founding member of the Biomaterial Interfaces Technical Group for the American Vacuum Society, and Chair from 1995-2000.  He received the NSF Director’s Award for Collaborative Integration for contributions to integrating biology and information technology research in 2002. He received the SAIC Publication Award in 1993, 1994 and 1995; the Berman Award from the NRL in 1993 and 1995; the SAIC Technology Achievement Award in 1995 and the MIT Gold Award for community service in 1989.

Raphael Lee, Ph.D.

Dr. Lee, a Paul S. and Allene T. Russell Professor at the University of Chicago, holds appointments in Surgery (Plastic), Medicine (Dermatology), Molecular Medicine, Translational Medicine and Organismal Biology & Anatomy (Biomechanics). He directs the Laboratory for Molecular Regeneration at the University. He is also a founder and Chairman, Board of Directors of Avocet Polymer Technologies, Inc., Renacyte BioMolecular Technologies, Inc., Electrokiinetic Signal Research and of Maroon Biotech, Inc. all of Chicago, Illinois.  Dr. Lee is a surgeon and biomedical engineer. His professional research interests have focused on the effects of physical forces on tissue injury and healing processes, pharmaceutical control of scar formation, and in reconstructive surgery. He completed general surgery residency at the University of Chicago and plastic surgery residency at the Massachusetts General Hospital of Harvard University.  Dr. Lee’s research group is recognized for characterizing much of the molecular biophysics of cell injuries caused by electrical shock, ionizing irradiation and thermal trauma. In 1992 Dr. Lee’s laboratory developed a pharmaceutical method to restore structure and function to disrupted cell membranes and refold heat denatured proteins using biocompatible surfactants copolymers resulting in a now widely validated new class of trauma therapeutics that for first time off promise to restore viability damaged tissue following severe injury such as motor vehicle accidents and military trauma. Most recently, his polymer research has been extended to develop synthetic chaperones to restore function to denatured and aggregated proteins which has broad implications in tissue preservation and thermal trauma. In plastic surgery, Dr. Lee and his surgery residents were first to report obturator sensate fasciocutaneous flaps for congenital and acquired perineal deformities.

Dr. Lee’s research has resulted in new therapeutics which translated into establishment of four biotechnology companies. He directed an international WHO sponsored program to determine the health economic impact of Chernobyl on Belarus.  In 1999, he received the DSc degree from the University of South Carolina being cited for this contribution.

Regularly listed in “America’s Top Surgeons” by the Consumers’ Research Council of America and US News & World Report,  and earlier one of “America’s brightest Scientists under the age of 40” by Science Digest, Dr. Lee has received more than 40 professional awards including being named a Schering Scholar (1978), MacArthur Prize Fellow (1981), a Searle Scholar (1985). He has been elected to fellowship in the American Association of Plastic Surgeons, Institute for Electrical and Electronics Engineers, Biomedical Engineering Society, American Institute for Medical and Biological Engineering, and American Association for the Advancement of Science.   In 1981, he received the Coller Society Award for outstanding general surgery residents and in 1988, the James Barrett Brown Award from the American Association of Plastic Surgeons for "advancing knowledge in the field of Plastic Surgery"; In 1997 he was awarded the American Electrical Power Association Award for “Advancing Electrical Safety and Health".

Dr. Lee has also been named “distinguished alumnus” by three universities.  Dr. Lee has served as President, Society for Physical Regulation in Biology and Medicine, the Drexel 100, The Quadrangle Club, and the American Institute for Medical and Biological Engineering.  He is a Trustee of Drexel University and serves on the board of several companies. Several of Dr. Lee’s students and fellows have become international leaders in medical and bioengineering research. His laboratory, consistently funded for 30 years, attracted more than $25 million in research grants. He has authored and co-authored more than 200 publications, 4 books and many patents.

Christine A. Kelley, Ph.D.

Dr. Christine A. Kelley is the Director for the Division of Discovery Science and Technology (DDST) in the National Institute of Biomedical Imaging and Bioengineering (NIBIB) at the National Institutes of Health (NIH).   She received her B.S. in Animal Science from the University of Massachusetts, Amherst in 1978 and her Ph.D. degree in Cell and Molecular Biology from Boston University in 1988.  Her graduate research focused on the role of pericytes in the microvasculature.  From 1988-1996 Dr. Kelley conducted postdoctoral and then independent research on the function and regulation of smooth muscle and nonmuscle myosin isoforms in the Laboratory of Molecular Cardiology in the National Heart, Lung, and Blood Institute (NHLBI) at the NIH.  In 1996 Dr. Kelley became a Program Director for Vascular Biology within the Division of Heart and Vascular Diseases in the NHLBI, before moving in 1998 to a position as Program Director for Tissue Engineering and Biomaterials within the same Division.

Dr. Kelley joined the NIBIB in March, 2002 as a Program Director for Bioengineering and assumed her current position as Director of DDST in May, 2003.  The Division supports the development of innovative biomedical engineering technologies for the benefit of public health.  The Division portfolio is complex and far-ranging in scope.  It includes programs in biomaterials, bioinformatics, biomechanics, biosensors, drug and gene delivery systems, medical devices, surgical instruments, medical implant science, mathematical modeling, simulation and analysis, nanotechnology, rehabilitation engineering, telemedicine and tissue engineering.

William Stokes, DVM, DACL AM

 Dr. William S. Stokes is Director of the U.S. National Toxicology Program’s Interagency Center for the Evaluation of Alternative Toxicological Methods (NICEATM) at the National Institute of Environmental Health Sciences and the Executive Director for the Interagency Coordinating Committee on the Validation of Alternative Methods (ICCVAM). He directs the validation and interagency evaluation of new proposed safety testing methods and coordination of validation, regulatory acceptance, and national and international harmonization issues across ICCVAM’s 15 Federal agencies. NICEATM and ICCVAM work to translate innovative new science and technology from the bench into scientifically valid safety assessment methods that can be used to protect public health and prevent disease and injuries.

Dr. Stokes is a Rear Admiral and Assistant Surgeon General in the U. S. Public Health Service Commissioned Corps, where he served as the 8^th Chief Veterinary Officer. He received his B.S. with a major in biomedical engineering from the University of Louisville and his D.V.M. from the Ohio State University. He is a Board Certified Environmental Scientist and a board certified Diplomate of the American College of Laboratory Animal Medicine and the American College of Animal Welfare.  He has conducted collaborative biomedical research for over 33 years, beginning with assignments at the U.S. Army Medical Research Institute of Infectious Diseases and as Chief of Veterinary Services in the Department of Clinical Investigation at Tripler Army Medical Center.

Dr. Stokes transferred to the USPHS and NIH with assignment at the National Institute of Child Health and Human Development as Animal Program Director. At NIEHS, he has served as the Animal Program Director, Chief of the Comparative Medicine Branch, and Associate Director for Animal and Alternative Resources. He has served as a Council member for the Institute of Laboratory Animal Research at the National Academy of Sciences and is the author or coauthor of more than 275 peer-reviewed publications, book chapters, reports, and abstracts. He has received numerous awards, including the HHS Secretary’s Award for Distinguished Service, the Surgeon General’s Exemplary Service Medal, two NIH Director’s Awards, the Society of Toxicology’s Enhancement of Animal Welfare Award, the K.F. Meyer-James H. Steele Gold Head Cane Award, and the American Veterinary Medical Association’s Charles River Prize for outstanding contributions to laboratory animal medicine.

Sonja Beken, Ph.D.

Sonja Beken obtained her Master in Biological Sciences at the Vrije Universiteit Brussel (VUB), Belgium, holds a PhD in Pharmaceutical Sciences (VUB) and finalized a Master in Applied Toxicology at the University of Surrey, UK. From 1998 until 2000 she worked as Scientific Staff Member at Belgian Platform for Alternative Methods (BPAM). From 2003 to 2009 she acted as member of ECVAM’s scientific advisory committee (ESAC).

Today, Sonja Beken is the coordinator of the Unit of non-clinical evaluators within the Belgian Federal Agency for Medicines and Health Products (FAMHP).  This Unit is responsible for the evaluation of non-clinical data (pharmacology, pharmacokinetics and toxicology) submitted to support all phases of the life cycle of drug development (e.g. marketing authorization applications, clinical trial applications, EU and national scientific advice, paediatric investigation plans, etc).

She is a Member of the Safety Working Party (SWP) of the European Committee on Human Medicinal Products (CHMP) at the European Medicines Agency (EMA).  Since June 2011, Sonja Beken is the Chair of the CVMP/CHMP Joint Ad Hoc Expert Group on 3R’s (JEG 3Rs) at the EMA.

Her main areas of expertise relate to regulatory science, (in vitro) toxicology and metabolism as well as alternative models to animal experiments. 

Anne Plant, Ph.D.

Anne Plant received her PhD in Biochemistry from Baylor College of Medicine.  She is currently leader of the Cell Systems Science Group at NIST.  The focus of the group’s work is the study of quantitative metrics of complex biological responses, primarily through quantitative cell imaging and image analysis, and flow cytometry.  Of particular interest is how stochastic fluctuations influence individual cell responses and their significance in stem cell renewal and differentiation.  Another focus area is the response of cells to extracellular matrix, including the role of different receptors and mechanical forces to intracellular signaling, cell proliferation and cell phenotype.  

Rosemarie Hunziker, Ph.D.

Rosemarie Hunziker, Ph.D. is the Director of Tissue Engineering/ Regenerative Medicine, Biomaterials and Medical Devices at the National Institute of Biomedical Imaging and Bioengineering (NIBIB) within the National Institutes of Health (NIH) in the US Department of Health and Human Services (DHHS).  In that capacity she has programmatic oversight of discovery and applied research grants.  Dr. Hunziker brings a diverse background to this broad sphere of R&D.  She has education, training and experience in microbiology, immunogenetics and immunochemistry, developmental biology, animal husbandry, molecular biology, tissue engineering/regenerative medicine and stem cells, biomaterials, and technology transfer.  She is committed to nurturing discovery and realizing the practical benefits of the exciting developments at the forefront of cell-based tools and therapies.

Dan Tagle, Ph.D.

Associate Director for Special Initiatives

National Center for Advancing Translational Sciences (NCATS) at the NIH

Dr. Tagle recently joined NCATS as Associate Director for Special Initiatives where he will continue to lead and manage the NIH Microphysiological Systems program that started out under the NIH Common Fund.  Prior to joining NCATS, Dr. Tagle was a Program Director for Neurogenetics at the National Institute of Neurological Disorders and Stroke (NINDS) where he was involved in developing programs in genomics –based approaches for basic and translational research in inherited brain disorders. Dr. Tagle obtained his Ph.D. in Molecular Biology and Genetics from Wayne State University in 1990. He was an NIH NRSA postdoctoral fellow in Human Genetics at the laboratory of Dr. Francis S. Collins at the University of Michigan. Prior to joining NINDS in 2001, Dr. Tagle was an Investigator and Section Head of Molecular Neurogenetics at the National Human Genome Research Institute (NHGRI) since 1993 and has been involved in the positional cloning of genes for Huntington’s disease, ataxia-telangiectasia and Niemann-Pick type C disease.

Michael Jackson, Ph.D.

VP, Drug Discovery and Development

Prebys Center

Sanford Burnham Medical Research Institute

Dr. Jackson is V.P. of Drug Discovery and Development at Sanford-Burnham Medical Research Institute where he directs the operations of the Prebys Center, a fully enabled drug discovery enterprise, equipped with state of the art uHTS screening and downstream pharmacology and medicinal chemistry. The center has specialized capabilities to conduct image based high content screens (HCS) including patient derived “disease in a dish” phenotypic screens. Prior to joining the Sanford-Burnham in 2009, Dr. Jackson spent 15 years working within Johnson and Johnson’s pharmaceutical research and development organization. He was VP, and subsequently Senior VP of Drug Discovery at J&J PRD, responsible for small molecule drug discovery research at five geographic sites within the United States. In 2005, Dr. Jackson was appointed President of ALZA Corporation, a drug delivery company within J&J where he managed all aspects of research and development, successfully gaining regulatory approval for several drug delivery products.

Prior to his industry career, Dr. Jackson was a postdoctoral fellow and subsequently assistant Professor at The Scripps Research Institute, CA, where he published widely in the field of cell biology and immunology. He conducted his Ph.D. research in Dundee, Scotland in molecular toxicology.

Jack Reynolds, Ph.D.

Dr. Jack A. Reynolds retired from Pfizer in August 2006 where he was Senior Vice President of R&D and Worldwide Head of Safety Sciences and Comparative Medicine.  With over 30 years in the pharmaceutical industry, he is a seasoned leader within the pharmaceutical industry having made substantial contributions to numerous development programs and important drug approvals, including most recently; Lyrica, Chantix, Celebrex, Detrol, Exubera, VFend, Geodon, Macugen, Relpax, Tikosyn, Trovan, Avelox, Spiriva, Sutent, Taxol, Viagra, Zithromax, Zoloft, Selzentry, Videx, and Stavudine.  At Pfizer, he defined and implemented a new paradigm to elucidate, understand, manage and mitigate the inherent risks of medicines. This industry-leading and transformational paradigm shift in safety testing and decision making had a profound impact on Pfizer’s early stage portfolio in reducing attrition, improving drug development effectiveness and expanding their R&D Portfolio.  This scientifically driven and technology enabled early de-risking of drug development programs is only now being emulated by others.  Dr. Reynolds has acquired the reputation of an industry trailblazer.  His career has been characterized by extensive work and leadership in building partnerships with regulatory agencies through leadership roles in professional societies, industry executive groups and scientific organizations, which includes the current Systems Toxicology initiative with FDA and NCI.  Dr. Reynolds is continuing to lead paradigm changing initiatives in safety sciences and translational medical research as evidenced by the creation of the Center for Molecular Safety Sciences in collaboration with Duke University and the University of North Carolina.  Dr. Reynolds is active in numerous professional societies; he is the co-founder and past president of the Regulatory and Safety Evaluation Subsection of the Society of Toxicology, he is the founder and current Advisory Board Chair of the Drug Safety Executive Council which now includes a membership of over 2000 pharmaceutical industry professionals.  This council plays an important cross-industry role in collaboratively evaluating, validating and qualifying new technologies for safety testing and public health decision making.  He served on the Board of Directors for Cellumen and was a Strategic Advisor to Cellumen prior to their acquisition.  He also served as a strategic advisor to the recently launched Hepregen Corporation and as a strategic advisor to the Battelle Memorial Foundation, has provided leadership to the repositioning of their Multi-scale Toxicology Initiative.  He is a Founder and General Managing Partner of Pharma Capital Partners in San Francisco.  He is also a co-founder and CEO of AnaBios Corporation based in San Diego, CA, a service provider of human tissue based functional testing services.  He is board certified in both Veterinary Pathology and Laboratory Animal Medicine and is a Fellow of the International Academy of Toxicologic Pathologists.  He is currently an Adjunct Professor in the Division of Pharmacotherapy and Experimental Therapeutics at the School of Pharmacy, The University of North Carolina at Chapel Hill. In 2004, Dr Reynolds received a Washington State University Distinguished Alumnus Award for Research and Teaching.

William Warren, Ph.D.

William L. Warren is a Vice President and heads the VaxDesign campus of Sanofi Pasteur.  The VaxDesign campus specializes in biomimetic systems such as an in vitro human immune system (MIMIC® System) to accurately assess new drugs and vaccines in a more predictive and physiologic way.  Warren is a member of the Research and Development Management Committee, External Innovation Executive Committee, the New Vaccines Advancement Committee and the Global Leader Network at Sanofi Pasteur.

Prior to this, he was CEO and founder of VaxDesign Corporation before it was acquired by Sanofi Pasteur.  He was also a Managing Partner of Sciperio Inc., which is an innovative high-technology development company.  He directed a diverse portfolio of R&D programs as a program manager at DARPA in the Defense Sciences Office. Warren was a principal member of the technical staff at Sandia National Laboratories, and received his B.Sc. Honors and Ph.D. degrees in Engineering Science from The Pennsylvania State University.

He is a Fellow of the American Institute for Medical and Biological Engineering, has authored over 190 referred publications, the editor of three conference proceedings, has over 200 scientific presentations, and over two dozen patents or patent applications.  He has received three R&D 100 awards from non-volatile memories, to micro-dispensing systems, to a surrogate human immune system; the 2011 BioFlorida Company of the Year; the 2011 Florida Companies to Watch; the 2009 Outstanding Collaborator Award from the Defense Threat Reduction Agency; the 2009 Governor’s New Product Award; the Schwartz Business Innovation Award; the Medical Marker Award; the Industry Week Innovation Award; the Discover Magazine Award, and several outstanding paper awards. He is on the board of directors and scientific advisory board of several companies and organizations such as Florida’s Blood Centers.

Ping Zhao, Ph.D.

Ping Zhao is currently a senior clinical pharmacologist in Immediate Office of the Office of Clinical Pharmacology, Office of Translational Sciences, Food and Drug Administration (FDA).  He obtained his BS in Pharmacy from Beijing Medical University in China in 1994, and his PhD in Pharmaceutics from University of Washington, Seattle, WA in 2002.  Since then, Ping worked as DMPK scientist in several pharmaceutical companies.  In June 2008, Ping joined the Office of Clinical Pharmacology at FDA.  His primary responsibilities are to assess the effect of multiple patient factors (intrinsic and extrinsic) using mechanistic modeling and simulation and to evaluate the application of physiologically-based pharmacokinetic (PBPK) tools to support regulatory reviews.  He contributes the update of several “Guidance for industry” documents (e.g., drug-drug interaction, pediatrics, renal impairment, pregnancy).  Ping serves as an ad hoc clinical pharmacology reviewer for numerous INDs and NDAs, provides consultation to fellow reviewers on drug metabolism, drug-drug interaction and the application of PBPK, and organizes/provides training on the use of PBPK tools.  He manages several FDA research projects including Critical Path, and mentors several research fellows.

Michael L. Shuler , Ph.D.

Michael L. Shuler is the James and Marsha McCormick Chair of the Department of Biomedical Engineering as well as the Samuel B. Eckert Professor of Chemical Engineering in the School of Chemical and Biomolecular Engineering at Cornell University, Ithaca, New York.  He is currently the director for a NCI funded Physical Sciences-Oncology, the Center for the Microenvironment and Metastasis. Shuler received both of his degrees in chemical engineering (BS, University of Notre Dame, 1969 and Ph.D., University of Minnesota, 1973) and has been a faculty member at Cornell University since January 1974.  Shuler’s research is focused on biomolecular engineering and includes development of “Body-on-a-Chip” for testing pharmaceuticals and chemicals for toxicity, production systems for useful compounds, such as paclitaxel from plant cell cultures, and computer models of cells relating physiological function to genomic structure.  Shuler’s research has helped to lay the foundation for modern biochemical engineering and has led to commercial processes for production of the anticancer agent, Taxol, to tools to produce proteins from recombinant DNA (the “High Five” cell line), to software to support systems biology, and to devices for drug development (HuRel has licensed Body-on-Chip patents from Cornell. Shuler and F. Kangi have authored a popular textbook, “Bioprocess Engineering; Basic Concepts”.

He has received numerous national and international awards for his research as well as several teaching awards from Cornell. He received the Marvin Johnson Award from the American Chemical Society.  He has an honorary doctorate from the University of Notre Dame (2008).  He has received the Amgen Award in Biochemical Engineering, as well as the Professional Progress, Food, Pharmaceutical and Bioengineering Division Award, and the Warren K. Lewis Awards from the American Institute of Chemical Engineers. Also, he was the inaugural awardee for the J.E. Bailey Award from the Society for Biological Engineering.  Shuler has been elected to membership in the National Academy of Engineering and the American Academy of Arts and Science and is a fellow of numerous other professional societies.

Warren Grundfest, Ph.D.

Dr. Warren S. Grundfest is the former Chair of Biomedical Engineering at UCLA where he holds appointments as Professor of Bioengineering, Electrical Engineering and Surgery.  He is currently the Co-Chair of the AIMBE Advocacy Committee and served previously as the Chair of AIMBE’s Council of Societies.  He serves as the Senior West Coast Clinical Advisor and Portfolio Manager for Nanomedicine and Biomaterials for TATRC (the Telemedicine and Advanced Technology Research Center of the U.S. Army), and also serves as a member of the FDA Science Advisory Board on the Subcommittee for CDRH.  He serves as a Panel Member for several NIH Study Sections and DoD review committees.  He is one of the nation’s foremost experts on image-guided therapies and medical device development.  His research interests include minimally invasive surgery, biophotonics, optical diagnostics, medical robotics, and advanced medical imaging technologies.  Dr. Grundfest is past President of IBMISPS, a Fellow of the American College of Surgeons, the AIMBE, and SPIE.  He holds 15 patents, has 3 more pending, and has authored 200+ papers and 46 book chapters.  He has been involved with multiple corporate and venture technology development programs.

Warren S. Grundfest, MD, Professor of Bioengineering and Electrical Engineering, UCLA School of Engineering, Professor of Surgery, UCLA School of Medicine,  4121H Engr.V, Box 951600, University of California at Los Angeles, Los Angeles, CA 90095-1600

Phone: 310/794-5550; Fax: 310/794-5956; E-mail: warrenbe@seas.ucla.edu.

William Bentley, Ph.D.

William E. Bentley is the Robert E. Fischell Distinguished Professor of Engineering and founding Chair of the Fischell Department of Bioengineering. He is also appointed in the Department of Chemical and Biomolecular Engineering at the University of Maryland, College Park and the Institute for Bioscience and Biotechnology Research. Dr. Bentley received his undergraduate (BS, '82) and Master of Engineering degrees ('83) from Cornell University and his Ph.D. ('89) from the University of Colorado, Boulder, all in chemical engineering. At Maryland since 1989, Dr. Bentley has focused his research on the development of molecular tools that facilitate the expression of biologically active proteins, having authored over 200 related archival publications. Recent interests are on deciphering and manipulating signal transduction pathways, including those of bacterial communication networks, for altering cell phenotype. He has served on advisory committees and panels for the NIH, NSF, DOD, DOE, USDA, and several state agencies. He has mentored over 25 Ph.D.s, some of whom are academics at: Cornell (x2), University of Colorado, Clemson, University of Connecticut, Tufts, Postech (Korea), and Tsing-Hua (Taiwan). He co-founded a protein manufacturing company, Chesapeake PERL, based on insect larvae as mini bioreactors. Dr. Bentley is a Fellow of the AAAS and AIMBE and is an elected member of the American Academy of Microbiology.

Workshop #3

Validation and Qualification of New In Vitro Tools and Models for the Pre-Clinical Drug Discovery Process

March 14-15, 2013

Executive Summary
The third AIMBE/NIH workshop on validation and qualification of new in vitro tools and models for the pre-clinical drug discovery process was held on March 14th and 15th at the NIH Campus in Bethesda, MD. The overall goal of this series of workshops is to develop guidelines for investigators developing new pre-clinical drug development models on how to validate and qualify these new technologies so that they become useful, meaningful tools qualified by the FDA. The workshop was able to generate specifics for validation and qualification of new in vitro systems based upon the more broadly developed groundwork achieved in the first two meetings.

Read the full report here: Third Workshop Final Report

Event page:  https://aimbe.org/events/third-aimbe-nih-workshop

Fourth AIMBE/NIH Workshop on Validation and Qualification of New In Vitro Tools and Models for the Pre-clinical Drug Discovery Process

March 6-7, 2014

Bethesda, Maryland

NIH Campus, Lister Hill Auditorium 

The American Institute for Medical and Biological Engineering (AIMBE) and the National Institute of Biomedical Imaging and Bioengineering (NIBIB) will be holding the fourth meeting in a series of Workshops addressing the topic of Validation and Qualification of New In Vitro Tools and Models for the Pre-clinical Drug Discovery Process on March 6-7, 2013 in Bethesda, MD. The overall goal of this series of workshops is to develop guidelines for investigators developing new tools for the pre-clinical drug development process on how to validate these new technologies so that they become useful, meaningful tools.  

This workshop, which was rescheduled due to the government shutdown, will build upon the results and recommendations of the previous workshops and will begin to develop guidelines for validation and qualification of new model systems. Specific emphasis will be on model systems that may augment or replace existing models, especially animal models, in the Food and Drug Administration (FDA) drug approval process.

Day one of the workshop will review current perspectives on validation and qualification of new in vitro technologies from the perspective of the National Toxicology Program, Interagency Coordinating Committee on the Validation of Alternative Methods (ICCVAM), European Centre for the Validation of Alternative Methods (ECVAM), the European Medicines Agency, and the FDA.  On day two of the Workshop, discussion of validation and qualification guidelines will begin with talks on five technologies chosen as examples for evaluation followed by a perspective on validation and qualification guidelines for these technologies by teams led by AIMBE fellows. Breakout session will follow with general audience participation for input on what guidelines would be necessary for each of the three chosen technologies. The results of the breakout groups will be presented, followed by a discussion to get feedback from the remainder of the participants and begin planning for the follow-on workshops.

Event sponsor:

• American Institute for Medical and Biological Engineering
• National Institute of Biomedical Imaging and Bioengineering

Chairs:

James J Hickman, PhD
AIMBE Fellow
Professor of Nanoscience Technology, Chemistry, Biomolecular Science, and Electrical Engineering
University of Central Florida
jhickman@mail.ucf.edu

And

Christine A. Kelley, PhD
AIMBE Fellow
Director
Division of Discovery Science and Technology
National Institute of Biomedical Imaging and Bioengineering
NIH
kelleyc@mail.nih.gov

Agenda

Fourth Workshop on Validation and Qualification of New In Vitro Tools and Models for the Pre-Clinical Drug Discovery Process

March 6-7, 2014

Lister Hill Auditorium, NIH Campus, Bethesda, MD

March 6             Day 1

March 7             Day 2

Fifth AIMBE/NIH Workshop on Validation and Qualification of New In Vitro Tools and Models for the Pre clinical Drug Discovery Process

February 12, 2015

NIH Campus, Lister Hill Auditorium, Bethesda, MD
The American Institute for Medical and Biological Engineering (AIMBE), the National Institute of Biomedical Imaging and Bioengineering (NIBIB), and the National Center for Advancing Translational Sciences (NCATS) will be holding the fifth meeting in a series of Workshops addressing the topic of Validation and Qualification of New In Vitro Tools and Models for the Pre-clinical Drug Discovery Process on February 12, 2015 in Bethesda, MD. The overall goal of this series of workshops is to develop guidelines for investigators developing new tools for the pre-clinical, and possibly clinical, drug development process on how to validate and qualify these new technologies so that they become useful, meaningful tools. The focus of previous meetings was on drug toxicity. The focus of the meeting will be on drug efficacy.

Event sponsors:

• American Institute for Medical and Biological Engineering
• National Institute of Biomedical Imaging and Bioengineering
• National Center for Advancing Translational Sciences

Chairs:

James J Hickman, PhD

AIMBE Fellow

Professor of Nanoscience Technology, Chemistry, Biomolecular Science, and Electrical Engineering

University of Central Florida jhickman@mail.ucf.edu

Christine A. Kelley, PhD

AIMBE Fellow Director

Division of Discovery Science and Technology

National Institute of Biomedical Imaging and Bioengineering NIH

kelleyc@mail.nih.gov

Agenda -Thursday, February 12

6th Workshop intro 5-25-17 slides

Dear Workshop Participants:

Welcome and thank you for your interest in participating in the 6^th Workshop in the series!  This Workshop is being sponsored jointly by the American Institute for Medical and Biological Engineering (AIMBE), the National Institutes of Health, National Institute of Biomedical Imaging and Bioengineering (NIBIB) and the National Center for Advancing Translational Sciences (NCATS). We all believe this is a critical time to be discussing the validation and qualification processes that are going to be necessary to ensure that new in vitro tools and technologies for pre-clinical drug discovery become meaningful and useful tools for the drug development community. Emphasis will be on regulatory issues, rare diseases and orphan drugs.

This workshop will build upon the success of the previous five AIMBE/NIBIB Workshops (http://www.nibib.nih.gov/NewsEvents/MeetingsEvents/MeetingSummaries/AIMBE2012), with the most recent Workshop held at NIH on February 12-13, 2015.

The first session of the Workshop will address current perspectives on regulatory issues, orphan diseases and rare diseases from Industry and the US Food and Drug Administration. In the second session, there will be a series of talks on alternative methods, in vitro pre-clinical drug discovery tools and technologies from the perspective of several companies. The third session, on Day 2, will build upon the events of the first two sessions and will include presentations on Rare Diseases and Microphysiological “Organs-on a-Chip” Systems. This session will also include a panel discussions on guidelines and requirements for validation and qualification of Rare Disease.

By the conclusion of the Workshop we hope to have developed ideas for validation and qualification guidelines for new in vitro technologies for the pre-clinical drug discovery process for rare diseases that can be used by microphysiological system developers in critical discussions with the FDA and EMA.

We are looking forward to an interesting and productive meeting!

Sincerely,

Co-Chairs:

James J. Hickman, PhD
AIMBE Fellow
Professor of NanoScience, Technology, Chemistry, Biomolecular Science, and Electrical Engineering
University of Central Florida
jhickman@mail.ucf.edu

Dan Tagle, PhD, MS

Associate Director for Special Initiatives, Office of the Director

National Center for Advancing Translational Sciences/NIH

danilo.tagle@nih.gov

Christine A. Kelley, PhD
AIMBE Fellow

Acting Associate Director, Extramural Science Programs
National Institute of Biomedical Imaging and Bioengineering, NIH
kelleyc@mail.nih.gov

Šeila Selimović, PhD, MS

Program Director, Tissue Chips / Tissue Preservation Technologies / Biosensors

National Institute of Biomedical Imaging and Bioengineering NIH

seila.selimovic@nih.gov

Logistics

Meeting Location

Natcher Auditorium, Bldg 45 (basement level), NIH Main Campus

Posters are exhibited in the Natcher Atrium, Bldg 45 (ground level).

Agenda

Thursday, May 25

Friday, May 26

Workshop Organizers 

Christine Kelley, PhD – Acting Associate Director, Extramural Science Programs, NIBIB/NIH

Dr. Christine A. Kelley received her Ph.D. degree in Cell Biology from Boston University in 1988. Her graduate research focused on the role of pericytes in the microvasculature. From 1988-1996 Dr. Kelley conducted postdoctoral and independent research on the function and regulation of smooth muscle and nonmuscle myosin isoforms in the Laboratory of Molecular Cardiology in the National Heart, Lung, and Blood Institute (NHLBI). In 1996 Dr. Kelley became a Health Scientist Administrator in the Vascular Biology Research Group within the Division of Heart and Vascular Diseases in the NHLBI, before moving in 1998 to a position as a Health Scientist Administrator in the Bioengineering and Genomic Applications Research Group within the same Division. Dr. Kelley assumed her current position in NIBIB in March 2002.

James Hickman, PhD – Professor, University of Central Florida

James J. Hickman is the Founding Director of the NanoScience Technology Center and a Professor of Nanoscience Technology, Chemistry, Biomolecular Science, Material Science and Electrical Engineering at the University of Central Florida. Previously, he held the position of the Hunter Endowed Chair in the Bioengineering Department at Clemson University. Dr. Hickman has a Ph.D. from the Massachusetts Institute of Technology in Chemistry. For the past twenty-five years, he has been studying the interaction of biological species with modified surfaces, first in industry and in the latter years in academia. While in industry he established one of the first bioelectronics labs in the country that focused on cell-based sensors and their integration with electronic devices and MEMS devices. He is interested in creating hybrid systems for biosensor and biological computation applications and the creation of functional in vitro systems for human body-on-a-chip applications. He has worked at NSF and DARPA in the area of biological computation. He is also the founder and current Chief Scientist of a biotechnology company, Hesperos, that is focusing on cell-based systems for drug discovery and toxicity. He has 119 publications and 20 book chapters, in addition to 19 allowed patents out of 42 total patent applications.

Danilo Tagle, PhD – Associate Director for Special Initiatives, NCATS/NIH

Dan Tagle recently served as acting director of the NCATS Office of Grants Management and Scientific Review and currently serves as executive secretary to the NCATS Advisory Council and Cures Acceleration Network Review Board. Prior to joining NCATS, Tagle was a program director for neurogenetics at the National Institute of Neurological Disorders and Stroke (NINDS), where he was involved in developing programs concerning genomics-based approaches for basic and translational research in inherited brain disorders. Prior to joining NINDS in 2001, Tagle was an investigator and section head of molecular neurogenetics at the National Human Genome Research Institute and has been involved in the highly collaborative effort toward the positional cloning of genes for Huntington’s disease, ataxia-telangiectasia and Niemann-Pick disease type C. He has served on numerous committees and advisory boards, including the editorial boards of the journals Gene and the International Journal of Biotechnology. Tagle obtained his Ph.D. in molecular biology and genetics from Wayne State University School of Medicine in 1990. He was an NIH National Research Service Award postdoctoral fellow in human genetics in the laboratory of Francis

1. Collins, M.D., Ph.D., at the University of Michigan. Tagle has authored more than 150 scientific publications and has garnered numerous awards and patents.

Šeila Selimović, PhD – Director of Tissue Chips and Biosensor Programs, NIBIB/NIH

Dr. Selimović manages the Tissue Chips / Tissue Preservation Technologies and Biosensor Programs within NIBIB. In 2015, she was selected as one of the "50 Leaders of Tomorrow" from among hundreds of young biotech leaders in the Mid-Atlantic region. Prior to her current position, she was chosen by the American Association for the Advancement of Science to serve as a Science and Technology Policy Fellow at the U.S. Department of State. Previously, she was a Fellow at Harvard Medical School and Brigham & Women’s Hospital in Boston, Massachusetts. Dr. Selimović’s research has focused on the development of microfluidic platforms for applications in biophysics and biological engineering, and her research interests include the physics of microscale flows, protein crystallization, colloidal suspensions, as well as rheology and microrheology. Dr. Selimović earned her Ph.D. and M. Sc. degrees in Physics from Brandeis University, with National Science Foundation support, and her B.A. degree in Physics and German from Wellesley College. She is a member of Sigma Xi.

Participant Biographies

Gilda Barabino, PhD – President, AIMBE

Gilda A. Barabino is Berg Professor and Dean of The Grove School of Engineering at The City College of New York (CCNY). She holds appointments the in the Departments of Biomedical Engineering and Chemical Engineering and the CUNY School of Medicine. Prior to joining CCNY, she served as Associate Chair for Graduate Studies and Professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory. At Georgia Tech she also served as the inaugural Vice Provost for Academic Diversity. Prior to her appointments at Georgia Tech and Emory, she rose to the rank of Full Professor of Chemical Engineering and served as Vice Provost for Undergraduate Education at Northeastern University.  She is a noted investigator in the areas of sickle cell disease, cellular and tissue engineering, and race/ethnicity and gender in science and engineering. Dr. Barabino received her B.S. degree in Chemistry from Xavier University of Louisiana and her Ph.D. in Chemical Engineering from Rice University. She is a Fellow of the American Association for the Advancement of Science, the American Institute of Chemical Engineers, the American Institute for Medical and Biological Engineering (AIMBE) and the Biomedical Engineering Society (BMES). She was awarded an honorary doctorate by Xavier University of Louisiana in 2016. She is the President of AIMBE and a Past-President of BMES. Dr. Barabino is a member of the National Science Foundation’s (NSF) Advisory Committee for Engineering and has served on the National Institutes of Health’s (NIH) National Advisory Dental and Craniofacial Research Council. Dr. Barabino consults nationally and internationally on STEM education and research, diversity in higher education, public policy, workforce development and faculty development. She directs the NSF Minority Faculty Development Workshop and is the founder and Executive Director of the National Institute for Faculty Equity.

William Bentley, PhD – Professor, University of Maryland, College Park (MODERATOR)

William E. Bentley is the Robert E. Fischell Distinguished Chair of Engineering and the Inaugural Director of the Robert E. Fischell Institute for Biomedical Devices. He is also appointed to the Department of Chemical and Biomolecular Engineering at the University of Maryland, College Park and the Institute for Bioscience and Biotechnology Research. At Maryland since 1989, Dr. Bentley has focused his research on the development of molecular tools that facilitate the expression of biologically active proteins, having authored over 270 related archival publications. He is a fellow of AAAS, ACS, AIMBE, and the American Academy of Microbiology. He has served on advisory committees for the NIH, NSF, DOD, DOE, FDA, USDA, and several state agencies and has mentored more than 30 PhDs and 15 postdocs, many now in leadership roles within industry (18), federal agencies (4) and academia (22). He co-founded a protein manufacturing company, Chesapeake PERL, based on insect larvae as mini bioreactors.

Brian Berridge, DVM, PhD, DACVP – Senior GSK Fellow, GlaxoSmithKline

Brian Berridge is a Senior GSK Fellow and Head of Animal Research Strategy in the Office of Animal Welfare, Ethics and Strategy at GlaxoSmithKline.  In that position he leads efforts to advance the scientific impact of animal and non-animal modeling in support of pharmaceutical development.  He was previously a Principal Research Pathologist at Eli Lilly and a Director of Regulatory and Discovery Pathology at GlaxoSmithKline.  Brian is an Oklahoma State University-trained veterinarian with residency and PhD training from Texas A&M University. He is a Diplomate of the American College of Veterinary Pathologists and holds an adjunct Associate Professor position in the Department of Population Health and Pathobiology at North Carolina State University. He teaches cardiovascular toxicology at the University of North Carolina.  He is a member of the Executive Board and Board of Trustees for the ILSI Health and Environmental Sciences Institute where he also co-chairs the HESI Cardiac Safety Technical Committee and the Integrated CV Strategies Working Group.  Brian also chairs an international effort to harmonize cardiovascular nomenclature in regulatory toxicologic pathology and a CV Specialty Interest Group within the Society of Toxicologic Pathologists.  He is a member of the editorial boards for the journals Toxicology Pathology and Cardio-Oncology and chairs the IQ Microphysiological Systems Working Group collaborating with NIH NCATS to define more structured approaches to characterizing and validating organ chip technologies.  Brian is a member of the Scientific Advisory Committee for Alternative Toxicologic Methods to NICEATM and is the current Chair of the IQ 3Rs Leadership Group.

Khaled Bouri, PhD, MPH – Interdisciplinary Scientist, FDA (MODERATOR)

Dr. Bouri joined the FDA in 2009 as a Commissioner’s Fellow. Currently, he is an interdisciplinary scientist and serves as the Senior Science Advisor to the Director of the Office of Regulatory Science Innovation & Critical Path Initiatives. Prior to joining FDA, Dr. Bouri was a senior research fellow at the George Washington University School of Public Health. Dr. Bouri received his Ph.D. in Molecular and Cellular Pharmacology from the University Louis Pasteur, Strasbourg, France, and a Master in Public Health Communication and Marketing from the George Washington University. He has twenty years of experience in cutting-edge biomedical and public health research and practice, with a specific focus on integrating genomic and other emerging technologies into the health care system. His current work focuses on advancing regulatory science and policy at the FDA by supporting research programs internally and externally, and by conducting research that supports FDA’s core mission in protecting public health while promoting innovative medical technologies.

Paul Brown, PhD – CDER / FDA

Dr. Brown is an Associate Director for Pharmacology and Toxicology in the Center for Drug Evaluation and Research at the FDA. He has been at the FDA since 1996 when he joined the Division of Dermatology and Dental Drug Products as a Pharmacology/Toxicology reviewer. He was supervisor for Pharmacology / Toxicology in this Division from 2003 to 2008. Prior to coming to the FDA he was a Pharmacology Research and Training Fellow in the National Cancer Institute from 1991 to 1996. He received his Ph.D. in toxicology from the University of Maryland in 1991. Dr. Brown is involved in policy and guidance development in a number of areas for the pharmacology and toxicology discipline within CDER.

Warren Casey, PhD – Acting Director, NICEATM

Dr. Casey is the Director of National Toxicology Program Interagency Center for the Evaluation of Alternative Toxicological Methods (NICEATM) and Executive Director of the US Interagency Coordinating Committee on the Validation of Alternative Methods (ICCVAM).  These groups work together to facilitate the development, validation, regulatory acceptance, and industry adoption of non-animal test methods. Prior to assuming his current position, Dr. Casey worked at GlaxoSmithKline for 15 years in a variety of roles, including: Manager of Pharmaceutical Microbiology, Head of In Vitro Biomarker Development, and Manager of Discovery and Investigative Toxicology.  Dr. Casey received his undergraduate degree in biochemistry and his PhD in microbiology from North Carolina State University (NCSU), where he has been named a Distinguished Alumnus and also holds an adjunct professorship in the Department of Microbiology.  He has been a Diplomate of the American Board of Toxicology (DABT) since 2007, received the 2016 Society of Toxicology Animal Welfare Award, currently serves as the vice president of the SOT In Vitro and Alternative Methods Specialty Section and co-chairs the OECD Validation Management Group – Non Animal.

Jonathan Goldsmith, PhD – Director, Rare Disease Program, Office of New Drugs / FDA

Dr. Goldsmith is Associate Director of the Rare Diseases Program at CDER’s Office of New Drugs. He earned his medical degree from New York University, received his post-graduate training in Internal Medicine at Vanderbilt, and completed specialty training in hematology at the University of North Carolina. He has had an extensive career in academia as a tenured professor, in regulated industry focusing on clinical drug development, and with rare disease foundations.

Federico Goodsaid, PhD – Senior Vice President, Clinical and Regulatory Affairs, TOMA Biosciences

Federico Goodsaid has worked at TOMA Biosciences on the preclinical and clinical development and regulatory submission for a 130-gene targeted NGS onco panel. His previous work at Vertex was focused on therapeutic product submissions in cystic fibrosis and diagnostic and biomarker development and regulatory submissions in rare diseases. He was previously Associate Director for Operations in Genomics and Biomarker Qualification Coordinator at the Office of Clinical Pharmacology/Office of Translational Sciences/ Center for Drug Evaluation and Research/ U.S. FDA, working on the regulatory application and development of genomics and biomarkers at the FDA. His B.A. was in Biochemistry and Biophysics from the University of California at Berkeley and his Ph.D from Yale University in Molecular Biophysics and Biochemistry. He was a Postdoctoral Fellow at Cornell University and at Washington University in St. Louis. Before he joined the FDA, he was Senior Staff Scientist at Applied Biosystems and Lead for the Molecular Toxicology Group at the Schering-Plough Research Institute.

Thomas Hartung, MD, PhD – Professor, Johns Hopkins Bloomberg School of Public Health

The main goal of Dr. Hartung’s work is toward a paradigm shift in toxicity testing to improve public health. Due to his background as head of the European Center for the Validation of Alternative Methods of the European Commission (2002-2008), he is involved in the implementation of the 2007 NRC vision document “Toxicity Testing in the 21st Century – a vision and a strategy”. He has furthered the translation of concepts of evidence-based medicine to toxicology (evidence-based toxicology). This aims for systematic assessment of the quality of all tools for regulatory toxicology and the development of new approaches based on annotated pathways of toxicity (the Human Toxome). He has a broad background in clinical and experimental pharmacology and toxicology documented in more than 450 publications. Previous work centered on the immune recognition of bacteria, including pyrogen testing, and the induced inflammatory response. In experimental and clinical approaches, the pharmacological modulation of these responses was studied. He has relocated to the US in early 2009 and established beside the directorship for the Center for Alternatives to Animal Testing (CAAT) a laboratory for developmental neurotoxicity research based on genomics and metabolomics. The respective technologies were made available by a Thought-Leader Award from Agilent.

Reyk Horland, PhD – Vice President of Business Development, TissUse, GmbH

Since 2010 Reyk has been actively involved in the development of TissUse’s Multi-Organ-Chip platform for culture analysis of drug candidates, cosmetics, chemicals and consumer products. He currently holds the position of Head of Business Development at TissUse. Prior to TissUse Reyk studied Biotechnology at the Technische Universität Berlin and specialized in Medical Biotechnology. During his academic career, he was involved in various tissue-engineering programs, all with a focus on bringing the respective products to market.

Christopher Hughes, PhD – Professor, University of California, Irvine

Dr. Hughes is currently Chair of Molecular Biology & Biochemistry at UC Irvine, with a joint appointment in Biomedical Engineering. He is also Director of the Edwards Lifesciences Center for Advanced Cardiovascular Technology, and a AAAS Fellow. Dr. Hughes did his Ph.D. at Kings College London in neuropathology, before coming to the US in for post-doctoral training at Harvard Medical School and Yale. He has extensive experience in the field of vascular biology and the process of angiogenesis. In particular, his lab has studied the role of notch signaling in vascular development and was the first to demonstrate the role of notch in regulating tip cell function during angiogenic sprouting. He has recently published on the role of the transcription factor slug in angiogenesis, and is working on the role of wnt regulation of vascular assembly. Dr. Hughes’ lab developed the widely-used fibrin/bead assay of angiogenic sprouting. In collaboration with Drs. Steve George and Abe Lee he has also been developing novel drug-screening platforms, including “Vascularized Micro-Organ” microfluidic devices. Dr. Hughes also works extensively with the non-profit organization, cureHHT (formerly HHT Foundation International), which supports patients with this rare vascular disorder. He is currently Chair of their Global Research and Medical Advisory Board. Dr. Hughes’ work has been continuously NIH-supported since he joined UCI in 1996.

Dr. Daria Julkowska – Coordinator, Agence National de la Recherche

Daria Julkowska is a Scientific Coordinator at the French National Research Agency (ANR), where she is responsible for the management of several European and international funding programs. Among others, since 2010 she is involved in E-Rare, the ERA-Net for Research programs on rare diseases, where for the first two years she occupied the position of the project manager to finally (April 2013) take over the coordination of the program. As Coordinator, she developed and put into action a set of collaborations facilitating rare diseases research, including the partnerships with European Research Infrastructures and Patients’ Organizations. She has an extensive knowledge and understanding of European funding schemes and programs. Since February 2017 she serves as the chair of the Funders Constituent Committee of IRDiRC. Of Polish origin, Dr. Julkowska obtained her international PhD in molecular biology at the University of Paris XI, France and University of Gdansk, Poland in 2005. She pursued her scientific vocation by the post-doctoral experience in cellular biology, at the Institut Pasteur, Paris and extensive training in communication and European Union counselling. She also holds MSc in Management of Research from the University of Paris Dauphine (FR).

Krishna Kandarpa, MD, PhD – Director of Research Sciences and Strategic Directions, Office of the Director, NIBIB/NIH

Krishna Kandarpa is a cardiovascular and interventional radiologist. Most recently, he was Chief Medical Officer/Chief Scientific Officer/EVP-R&D at Delcath Systems, Inc., New York, NY, which markets a combination device/drug treatment for cancers in the liver. Prior to this, he was tenured Professor and Chair of the Department of Radiology at the University of Massachusetts Medical School and Radiologist-in-Chief at the UMass Memorial Medical Center. Earlier, he was at the Weill Medical College of Cornell University, where he was a Professor of Radiology and Chief of Service and Director of the Division of Cardiovascular & Interventional Radiology at The New York Presbyterian Hospital (Cornell), when he simultaneously held an adjunct professorship at the College of Physicians and Surgeons of Columbia University. He embarked on his academic career at Harvard Medical School and was an Associate Professor of Radiology, co-director of Cardiovascular & Interventional Radiology and the Vascular Center, at the Brigham and Women’s Hospital, Boston, MA. While there, he was also on the faculty of the Harvard-Massachusetts Institute of Technology Health Sciences and Technology Program.

Jens Kelm, PhD – Chief Technology Officer, Co-Founder and Member of the Management Team, Insphero

Jens is currently chief technology officer (CTO) and co-founder of InSphero AG, Zurich, Switzerland. He has 15 years' experience in 3D cell culture technology developing a wide variety of tissues focusing on the cellular self-assembly approach. Previously scientific director at the Center for Applied Biotechnology and Molecular Medicine at the University of Zurich CABMM. He co-founded the Swiss competence center for "Tissue Engineering for Drug Development” (TEDD) where he still active within the steering committee. On a European level, he is part of the organizing committee of the bi-annual 3D cell culture conference (DECHEMA) and is involved as work package leader in the EU flagship program EuToxRisk-21.

Laura Koontz, PhD – Personalized Medicine Staff, CDRH/FDA

Laura M. Koontz is a member of the Personalized Medicine Staff at the Food and Drug Administration in the Center for Devices and Radiological Health. Prior to joining the FDA, she was the Director of Policy for the Ovarian Cancer National Alliance, where she oversaw Congressional and regulatory policy. Prior to that, she was an ASHG-NHGRI Genetics and Public Policy Fellow from 2012-2013 and worked in the House of Representatives. Dr. Koontz holds a PhD in Biochemistry, Cellular and Molecular Biology from the Johns Hopkins University School of Medicine.

Daniel Levner, PhD – Chief Technology Officer, Emulate

Daniel Levner, PhD, is Chief Technology Officer at Emulate, Inc., a company that is creating a new living system that emulates human biology, for understanding how different diseases, medicines, chemicals and foods may affect human health. Prior to joining Emulate’s founding team, Dr. Levner was a Senior Staff Scientist with the Wyss Institute for Biologically Inspired Engineering at Harvard University, where he led the advanced engineering team responsible for developing Emulate’s Organs-on-Chips platform. There, Dr. Levner played a key leadership role, formulating approaches to foster close collaboration between biologists and engineers. Prior to the Wyss Institute, Dr. Levner worked with world-renowned Harvard geneticist Prof. George M. Church in programs related to medical diagnostics, DNA/RNA sequencing tools, and multiplexed analysis techniques. As an entrepreneur, Dr. Levner co-founded a medical diagnostics startup company, and earlier in his career, an optical telecommunications startup company. Dr. Levner received his Ph.D. in electrical engineering from Stanford University as well as an M.S. in aeronautics and astronautics, also from Stanford. He has authored numerous publications and more than 25 issued and pending patents.

William Mattes, PhD – Director, Division of Systems Biology, National Center for Toxicological Research/FDA

Dr. Mattes is the Director of the Division of Systems Biology, part of the FDA’s National Center for Toxicological Research in Jefferson, Arkansas. Dr. Mattes developed and directed the Predictive Safety Testing Consortium (PSTC), a collaboration of 16 of the world’s major pharmaceutical companies, with FDA and EMEA advisors, with the goal of qualifying new biomarkers for drug safety in a regulatory setting. He also developed the COPD Biomarkers Qualification Consortium, serving as its Senior Director and overseeing interactions with the FDA. Dr. Mattes other positions included senior scientific director of Toxicogenomics at Gene Logic, Associate Director of Toxicogenomics and Group Leader of Genetic Toxicology at Pharmacia Corp, Kalamazoo, MI, Group Leader of Experimental Toxicology and Metabolism at Ciba Pharmaceuticals, Summit, NJ, and Group Leader of Molecular and Cellular Toxicology, Ciba-Geigy Agricultural Chemical Division, Farmington, CT. Dr. Mattes received his BA from the University of Pennsylvania and PhD in biological chemistry from the University of Michigan, Ann Arbor.  He did his postdoctoral training in biochemistry at the Johns Hopkins University, and was a staff fellow at the National Cancer Institute, the National Institutes of Health (NCI/NIH).  In 1997 Dr. Mattes became a diplomate of the American Board of Toxicology.  He is a full member of the Society of Toxicology and the American College of Toxicology (ACT) and has severed on committees for both organizations.

William Murphy, PhD – Professor, University of Wisconsin

Bill Murphy is the Harvey D. Spangler Professor of Biomedical Engineering, Professor of Orthopedics & Rehabilitation, Co-Director of the Stem Cell and Regenerative Medicine Center, and Director of the Human MAPs Center at the University of Wisconsin. His research interests focus on creating new biomaterials inspired by the materials found in nature. Murphy’s research group is using new biomaterials to understand stem cell behavior and to induce tissue regeneration. He has published more than 150 scientific manuscripts, filed over 40 patents, co-founded multiple start-up companies, and received awards that include the National Science Foundation Career Award, the Wisconsin Vilas Associate Award, the H.I. Romnes Fellowship, and induction as a Fellow in the American Institute for Medical and Biological Engineering.

Monicah Otieno, PhD, DABT - Scientific Director & Head, Mechanistic & Investigative Toxicology, Janssen Pharmaceuticals

Dr. Otieno has held positions in the pharmaceutical industry for 16 years as a Discovery/Mechanistic Toxicologist and is presently the Director of Mechanistic & Investigative Toxicology group at Janssen Pharmaceuticals in Pennsylvania. Monicah received her PhD in Pharmacology from University of Rochester in Rochester, New York, followed by a postdoctoral fellowship from the Department of Environmental Health Sciences, Division of Toxicology at Johns Hopkins University in Baltimore, Maryland. Monicah is presently leading scientific efforts at Janssen to explore the utility and value of Organs-on-Chip technology and is also actively involved in the IQ-consortium of industry partners on microphysiological models.

Anne Pariser, MD – Deputy Director, Office of Rare Diseases Research, NCATS/NIH

Anne Pariser has joined NCATS in January 2017 as the deputy director of the Office of Rare Diseases Research (ORDR). She works with the ORDR director and team to advance diagnosis and treatment for rare diseases through research. Pariser comes to NCATS from the Food and Drug Administration (FDA), where she had worked since 2000, mainly on the development of drug and biological products for rare diseases.  Most recently, she served as associate director for knowledge management in the FDA’s Office of Translational Sciences, which is part of the Center for Drug Evaluation and Research (CDER). Prior to serving in that role, Pariser was the associate director for rare diseases in the Office of New Drugs (OND) at CDER, where she established the Rare Diseases Program (RDP) in 2010 to support, facilitate and accelerate the development of therapeutics for rare diseases. Before founding RDP, she was a medical officer and team leader in OND, where she worked almost exclusively on the review and regulation of products for rare genetic disorders. Pariser has been involved in numerous collaborations within the FDA and with drug developers, governmental agencies, patient groups and other stakeholders to further the development of treatments for rare diseases. In addition to her government service, she works as a physician volunteer in the Arlington Free Clinic. Pariser received her bachelor’s degree in chemistry from Bates College in Lewiston, Maine, and her medical degree from Georgetown University School of Medicine in Washington, D.C., where she also completed her training in internal medicine. Pariser is board certified in internal medicine.

Christopher Penland, PhD – Vice President of Research, Cystic Fibrosis Foundation

Chris Penland, Ph.D. Earned a B.Sc. at Pfeiffer College in Sports Medicine and Management, a M.Sc from Appalachian State University in Exercise Science and completed his doctoral studies at East Carolina University in the Department of Physiology where  he examined ion transport of bronchiolar tissues.  A postdoctoral fellowship at Stanford University followed in the laboratory of Jeffrey Wine where Dr. Penland was part of a larger team seeking to identify a naturally occurring primate model of cystic fibrosis.  Although no such animal was identified Dr. Penland was given a chance to further his efforts in CF, this time behind a desk rather than standing at a bench, when he joined the US Cystic Fibrosis Foundation as Director of Research in 1999.  For the next 15 years Dr. Penland oversaw the basic science investments the Foundation made and served on the advisory committees of numerous drug discovery and development projects.  In 2015 Dr. Penland transitioned to a new role in the Foundation where he now facilitates relationships between it and biopharmaceutical companies engaged in CF-related drug discovery and development.

Anne Plant, PhD – Chief, Biosystems and Biomaterials Division, NIST

Anne Plant received her PhD in Biochemistry from Baylor College of Medicine in Houston TX, USA.  She has published in various areas of research including biosensors, biomolecular materials, quantitative cell imaging, and theoretical modeling. She is a Fellow of the American Institute for Medical and Biological Engineering, and a AAAS Fellow.  Currently she serves as Chief of the Biosystems and Biomaterials Division at the National Institute of Standards and Technology.  The Division’s work is focused on quantitative measures of living systems and the molecular basis of cellular response in health and disease.  The Division develops materials and methods that help to assure confidence in measurements of cell phenotype, cancer genomic biomarkers, genomic editing, flow cytometry, and microbial genomics for applications including regenerative medicine, biomaterials, engineered biology and pathogen detection.

William Proctor, PhD – Senior Scientist, Genentech

Will Proctor is the Head of Investigative Toxicology group in the Department of Safety Assessment at Genentech, Inc. Prior to joining Genentech in December of 2013, Will earned his PhD in Pharmaceutical Sciences from the University of North Carolina at Chapel Hill in the laboratory of Dr. Dhiren Thakker with a focus on drug transport and pharmacokinetics.  He then performed postdoctoral training at the NIH/NHLBI in laboratory of Dr. Lance Pohl, focusing on immune mechanisms of drug-induced liver injury (DILI).  His research interests in mechanisms of DILI continued at Genentech, where his group has worked to qualify new in vitro tools to assess hepatotoxicity risk as well as evaluating exploratory biomarkers of liver injury.

Michael Shuler, PhD – President/CEO, Hesperos, and Professor, Cornell University

Michael L. Shuler is the CEO and President of Hesperos, Inc., a company founded to further develop and implement the “Body on a Chip” system in collaboration with James Hickman. Shuler is also the Samuel B. Eckert Professor of Engineering in Biomedical Engineering and in the School of Chemical and Biomolecular Engineering at Cornell University, Ithaca, New York. He was the director for a NCI funded Physical Sciences- Oncology Center (Center for the Microenvironment and Metastasis) and is currently director of Cornell’s Nanobiotechnology Center. Shuler received both of his degrees in chemical engineering (BS, University of Notre Dame, 1969 and Ph.D., University of Minnesota, 1973) and has been a faculty member at Cornell University since January 1974. Shuler’s research is focused on biomolecular engineering and includes development of “Body-on-a-Chip” or microphysiological system for testing pharmaceuticals and chemicals for toxicity, creation of production systems for useful compounds, such as paclitaxel from plant cell cultures, and constructions of computer models of cells relating physiological function to genomic structure. Shuler has been elected to membership in the National Academy of Engineering and the American Academy of Arts and Science and is a fellow of numerous other professional societies.

George Truskey, PhD – Professor, Duke University

George A. Truskey, PhD, is the R. Eugene and Susie E. Goodson Professor of Biomedical Engineering and the Senior Associate Dean for the Pratt School of Engineering at Duke University. He received his PhD in Chemical Engineering in 1985 and has been a member of the faculty at Duke University since 1987.  His current research interests include microphysiological systems, cardiovascular tissue engineering and the role of physical forces in atherosclerosis. From 2003-2011, he was Chair of the Department of Biomedical Engineering at Duke University. During that time, he directed Duke’s Translational Research Partnership with the Coulter Foundation and the successful transition to an endowed program. He is the author of over 125 peer reviewed research publications, a biomedical engineering textbook entitled Transport Phenomena in Biological Systems, six book chapters, 1 patent and 2 patent applications. He is a Fellow of the American Association for the Advancement of Science (AAAS), Biomedical Engineering Society (BMES), the American Institute of Medical and Biological Engineering, and the American Heart Association. He was president of BMES from 2008 to 2010. In 2007, he received the Capers and Marion McDonald Award for Excellence in Mentoring and Advising from the Pratt School of Engineering at Duke.

Gordana Vunjak-Novakovic, PhD – University Professor, Columbia University

Dr. Vunjak-Novakovic is the University Professor at Columbia University, and the Mikati Foundation Professor of Biomedical Engineering, Professor of Medical Sciences and a faculty in the Irving Comprehensive Cancer Center and the Center for Human Development. The focus of her research is on engineering functional human tissues for regenerative medicine, stem cell research and study of disease. With ~35,000 citations and h=108, she is one of the most highly cited individuals of all times. She has 79 licensed, issued or pending patents, and has founded with her students three biotech companies: epiBone (epibone.com), Tara (tarabiosystems.com), and East River Biosolutions (http://eastriverbio.com). Among her many recognitions, she is a member of the National Academy of Engineering (officer), National Academy of Medicine, and National Academy of Inventors.