Speakers

Although the risk from TSEs in biopharmaceutical products have significantly decreased since the peak in the late 90s early 2000s era, controlling the TSE risk for such product still forms an important component of the product registration. TSE clearance studies can often provide useful data supporting product safety, but their implementation and execution requires careful adherence to regulatory guidelines. This presentation will focus on how TSE clearance studies fit into the overall TSE safety profile for a range of biopharmaceutical products and medical devices and will present key aspects related to their successful implementation.

Andy Bailey specialised in Virology while serving for nine years at the MRC Virology Unit, Glasgow University in Scotland. In 1995, he moved to the industry sector, initially as Director of Virus Validation services with Q-One Biotech Ltd, and later at the Global Pathogen Safety group of Baxter Healthcare in Vienna, Austria. Over the last 25 years Andy has been actively involved in the virus and prion safety field, presenting at numerous regulatory agencies either in support of products or as an invited speaker at expert workshops. In 2005 he founded ViruSure with the goal of providing a high quality science-based testing service to the biopharmaceutical industry.

The testing and approved release of raw materials for use in GMP manufacturing can be a process that takes weeks to accomplish, requiring companies to keep extensive inventories. To expedite the release of critical raw materials the Biogen RapidID platform was developed and implemented. The Biogen RapidID program describes an integrated IT and laboratory analytical instrumentation system, allowing incoming materials to be received, tested, and released in just a few hours. Two technologies make up the RapidID analytical platform: Raman and x-ray fluorescence to positively identify over 75 raw materials. To ensure compliance with the relevant agencies, a robust method validation and transfer strategy was implemented for the methods for both respective analytical instruments. The RapidID platform has been successfully implemented at Biogen since 2019.

Christie is a senior associate at Biogen, located in Research Triangle Park, North Carolina. As a member of the Analytical Technology department for the Quality Control organization she is responsible for the validation, implementation, and global transfer of new methods and programs in the raw material space. Christie joined in 2015 and has been in the pharmaceutical industry since receiving her bachelor’s degree in Biology from North Carolina State University in 2006.

Although often operating in the background, biobanking plays a critical role in the diagnosis and development of treatments for a variety of diseases. However, as we witness the urgent international efforts to develop vaccines and other therapies for the current COVID-19 pandemic, patient sample collection, processing, and analyses are at the forefront of this emergency. Biospecimen issues have long been important factors in handling the response to emerging infectious diseases. The US CDC has maintained a biobank for decades, and has found through analysis of its samples that infectious agents responsible for outbreaks such as the hantavirus and Legionnaires' disease were present years prior to the epidemic. Tissue samples from the 1918 Spanish influenza pandemic were used to determine that the origin of the virus was related to strains that commonly infected pigs and humans, and was not of avian origin as had been previously thought. Studies of the 1918 pandemic and subsequent influenza outbreaks have been instrumental in vaccine research as well. The Ebola outbreak in Africa in 2014–2016 presented some unique challenges, due to the high infectivity of patient blood samples. There were also security concerns, i.e. that Ebola patient samples could be used for bioterrorism purposes.

COVID-19 presents some unique issues in the biobanking world. The vast scope of the pandemic, with over 11 million patients globally as of early July, means that laboratories and biobanks are handling many samples involved in developing diagnostics and vaccines. The US CDC has published guidelines for handling COVID-19 samples. Some biobanks have developed their own guidelines, usually based on those of the CDC. Generally, these guidelines mandate the use of biosafety level 2 (BSL-2) rules for laboratories, including the use of protective equipment, use of class II biological safety cabinets, proper disinfection routines, etc. Also, a new US NIH COVID-19 Scientific Interest Group (SIG) has also been organized, where interested parties can join in an online discussion and exchange information about research and resources. At this time, we can expect that this pandemic will continue to be our most critical international concern for months to come, from a public health perspective, as well as from the resulting economic impact. As the number of cases continues to mount, as well as the unfortunate loss of life, biobanks will continue to work in the background to handle their roles in the response. This presentation will provide an update on biobanking activities during the pandemic.

Dr. Jim Vaught spent 14 years at the US National Cancer Institute, most recently as the Chief of the Biorepositories and Biospecimen Research Branch. He has been working in the field of biobanking and biospecimen science for over 20 years. In 1999, he was one of the founding members of the International Society for Biological and Environmental Repositories (ISBER) and served two terms as its president. In 2018 he was the recipient of ISBER’s Founder’s Award. His current work includes providing biobanking consulting services and serving on a number of advisory boards. He is a Senior Research Fellow at the International Prevention Research Institute in Lyon. He is the author of over 80 peer-reviewed articles and book chapters in chemical carcinogenesis and drug metabolism, biobanking, and biospecimen science. From 2006 to 2012 he was Senior Editor for Biospecimens and Biorepositories for the journal Cancer Epidemiology, Biomarkers and Prevention. He is the current Editor-in-Chief of Biopreservation and Biobanking, the official journal of ISBER. In 2017 he was appointed as a Guest Professor at Central South University in Changsha, China, where he is a lecturer for a post-graduate biobanking course. In 2018 he was appointed as a Guest Professor at the Children’s Hospital of Shanghai.

Human and other mammalian cell lines and xenograft tissues, including patient-derived xenografts (PDXs) and patient-derived organoids (PDOs), are essential tools as pre-clinical biomedical research model systems. They are used to study different aspects of biological processes (infection, enzymes, gene structure, and expression control), behavior of different types of human tissues and cells, diseases such as cancers and infection by microbes (e.g., viruses, bacteria), and effects of drugs on normal and abnormal biological processes.

A major concern in using cell lines and patient tissue samples as preclinical research models is that on average 22.5% (range 0–100%) of these samples are misidentified. This can compromise the validity and reproducibility of the research based on these model systems, waste valuable research funds and effort, and damage the reputations of research groups. This presentation will describe the enormity of the problem with examples from the literature and my own work that show how the scientific literature has been contaminated with inadequately authenticated cell lines. Current and new tools that can address this problem will be described.

Christopher Korch received his PhD at the University of California at Davis in Genetics and studied in France, Norway, Sweden, and the USA. In 2000, his University of Colorado Cancer Center core facility was one of the first to implement techniques for characterizing and authenticating human cell lines. In the last 19 years, he has published together with several groups on the identification and confirmation of the identity of authentic and over 80 imposter cell lines and clinical samples by DNA sequencing and STR genotyping. Some of the more salient findings include work with the following groups:
• Prostate Group showing about 30% of the prostate cell lines were misidentified and not suitable models of prostate cancer.
• Thyroid Group showing about 50% of the thyroid cell lines were misidentified and many were not suitable models of thyroid cancer. These finding shocked the endocrine field and have been seminal in the re-evaluation of earlier research. It initiated major efforts to establish authentic thyroid cell lines.
• Ovarian, Endometrial, and Breast Cancer Groups, which in 2012 published a major study on the identity of endometrial and ovarian cancer cell lines, showing extensive contamination, redundancy, and misidentification among these cell lines, raising questions about the validity of many publications using these cell lines.
• The International Cell Line Authentication (ICLAC) which has established that over 500 cell lines are misidentified, i.e., imposter cell lines.
• The ANSI/ATCC Standards group which is revising the Standard for Cell Line Authentication by STR profiling, which is due out in 2020.

Furthermore, Dr. Korch has been interviewed for a book, the Canadian Broadcasting Corporation, magazine articles including Science and Discover Magazine, and several documentaries (such as https://www.biocompare.com/Reproducibility/Cell-Line-Authentication/) and webinars about this problem.

Dr. Marian McKee is Vice President of Biosafety at Eurofins Lancaster Laboratories Inc. where she leads the Viral Clearance, Cell Banking, and Viral Safety departments. She has more than 20 years of experience driving research, design, and execution of services for biologics safety and characterization testing. In her current role, she provides guidance and the long range planning for biosafety, including development and strategic insight into the company’s gene and cell therapy offering. Prior to joining ELLI, Dr. McKee was at MilliporeSigma (BioReliance) most recently as Head of Global Testing R&D Services. She also worked at ATCC and as a Staff Scientist at the National Cancer Institute (NCI) after completing her postdoctoral training as a Cancer Research Training Award (CRTA) fellow at NCI. Dr. McKee earned her PhD in Microbiology and Immunology at the Uniformed Services University of the Health Sciences, Bethesda, Maryland and holds a BS in Zoology from Duke University.

Dr. Wei is an entrepreneur and chemistry professional in the area of supramolecular assembly and biomimetic nanoscience. She is the inventor of X-Therma’s core technology based on hyper-effective ice prevention materials. Her PhD work demonstrated the success of developing synthetic transmembrane nanopores with distinguished selectivity applying biomimetic and supramolecular chemistry principles. She founded X-Therma Inc. in 2014 to develop a state-of-the-art biopreservation formulation that incorporates a first-in-class hyper-effective (500x) and non-toxic proprietary antifreeze polymer. She led the X-Therma team as an industrial user at the Molecular Foundry, Lawrence Berkeley National Laboratory from 2015 to 2018 and gained multiple recognitions internationally and nationwide. She is the principle investigator of multiple SBIR awards to develop breakthrough cryoprotectants enabling complex tissue/organ cryopreservation with national initiatives. She is experienced in business development and fundraising and managed $7.5M in procurement projects while completing her PhD studies. She was a major author of eight peer-reviewed research papers before founding X-Therma.

Kelvin GM Brockbank, PhD is the President of KGB Associates, Inc., and Chief Executive Officer of Tissue Testing Technologies LLC. He is also a Research Professor of Bioengineering at Clemson University and Adjunct Professor of Regenerative Medicine and Cell Biology at the Medical University of South Carolina. His research interests include cell, tissue, and organ preservation for test systems and transplantation and automated manufacturing methods for cell-based tissue engineered products. His work has led to the establishment of two successful publicly traded low temperature technology platform companies, CryoLife, Inc. and Lifeline Scientific. Dr. Brockbank’s last company, Cell and Tissue Systems, Inc., was acquired in December 2014 for its intellectual property portfolio. He has published over 500 patents, book chapters, journal articles, and presentations at national and international conferences. He was the recipient of the “George W. Hyatt Memorial Award” for superior service in the fields of tissue banking and human transplantation in 2009 and is currently funded by several agencies of the National Institutes of Health and the Department of Defense.

Glyn Stacey’s background is in microbiology (Public Health Service, UK) and cancer research (University of Southampton) and he later progressed to work on the development of cell-derived products and cell-based assays at the Centre for Microbiology and Research, Porton Down, UK, where he established the Cell Development Group developing cell culture scale-up systems and cell-based assays. In 1998 he moved to the National Institute for Biological Standards and Control (NIBSC-MHRA, UK) where he set up a new cell biology division working on safety of cell substrates used for vaccine manufacture and establishment of genetic reference materials. In 2002, he won a UK national grant award to initiate the UK Stem Cell Bank to provide a source of ethically suitable and quality controlled human embryonic stem cell lines for research and clinical use. In 2007 he also initiated the International Stem Cell Banking Initiative which brings together pluripotent cell banks from around the world to discuss best practices. Glyn has been a principal investigator in numerous European Commission funded research programmes, scientific advisory boards, national health advisory committees, and was a member of the Horizon Scanning and Research Programme boards of the UK regulatory body Medicines and Healthcare products Regulatory Agency (MHRA). Glyn has also been involved in writing standards for cell culture applications in laboratory testing for the Organisation for Economic Co-operation and Development (OECD, 2018), cell-based manufacture of biotherapeutics and vaccines for the World Health Organization (WHO, 2010), and regenerative medicines for the UK Department of Health (2014). Glyn is currently the Director for the International Stem Cell Bank in Beijing, which is developing hESC lines for clinical trials and has seven registered clinical studies.

Xiaoming “Shawn” He is a Professor of Bioengineering at the University of Maryland (UMD), College Park. He received his PhD in Mechanical Engineering in 2004 from the University of Minnesota Twin Cities and conducted postdoctoral training from 2004 to 2007 at Harvard Medical School and Massachusetts General Hospital. He was an Assistant Professor at the University of South Carolina from 2007 to 2011, and Associate Professor and Full Professor at Ohio State University from 2011 to 2017. His current research is focused on developing micro and nanoscale biomaterials and devices to engineer totipotent, pluripotent, and multipotent stem cells for cancer theranostics and tissue regeneration. His research has been funded with him as the PI by various private foundations and government agencies including the American Cancer Society (ACS), National Science Foundation (NSF), and National Institutes of Health (NIH). He has published more than 110 peer-reviewed articles in high-ranking journals such as Nature Nanotechnology, Nature Communications, Advanced Materials, ACS Nano, ACS Central Science, and Advanced Functional Materials, in addition to one book and four book chapters. He is serving as the Chair of the American Society of Mechanical Engineers (ASME) Biotransport Committee and as an associate editor of the Journal of Medical Devices. He is a fellow of the ASME and the American Institute of Medical and Biological Engineering (AIMBE).

Audrey Chang earned her PhD in Biology at The Johns Hopkins University, Baltimore, Maryland and has over 20 years of government and industry experience in conducting biological products testing and in managing laboratories. In her current role as Executive Director of Development Services at MilliporeSigma she is responsible for managing and developing a team of PhD level scientific experts that provides greater focus to the development of new services and technologies while providing technical solutions and support to clients and operating laboratories. She and her department work to identify emerging products, technology trends, and global regulatory requirements and champion activities to implement new business plans.

Dr. Khan is Senior/Principal Investigator in the Division of Viral Products, Office of Vaccines Research and Review in the Center for Biologics Evaluation and Research (CBER), US Food and Drug Administration (FDA). She joined the FDA in 1991, after working in Dr. Malcolm Martin’s lab at National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH) since 1979, where she contributed significantly to research on pathogenic murine retroviruses, endogenous retroviruses, and simian immunodeficiency virus (SIV). In CBER, Dr. Khan established a program on the development of sensitive assays for adventitious virus detection, with a focus on the safety of novel cell substrates. She also expanded her research efforts to studying the role of naturally-occurring simian foamy retrovirus infections in SIV nonhuman primate models that are broadly used for preclinical studies of AIDS vaccines. Dr. Khan’s current research efforts focus on standardization of next-generation sequencing for adventitious virus detection for safety of biologics, including development of reference viruses and databases. Additionally, her program is investigating host and viral interactions regulating latent virus infections. Her regulatory responsibilities include review of Investigational New Drugs and Biological License Applications for a variety of candidate vaccines including HIV and emerging viruses, and novel cell substrates across different product categories. Dr. Khan has been involved in the licensure of several viral vaccines and development of FDA, International Conference on Harmonisation (ICH), Public Health Service (PHS), United States Pharmacopeia (USP), and World Health Organization (WHO) guidance documents related to cell substrates and viral safety of vaccines, therapeutics, and xenotransplantation. She is currently the FDA lead on adventitious virus detection using next-generation sequencing for cell substrates and product safety. Dr. Khan received her PhD in Microbiology from the George Washington University, Washington, DC.

Michael Brewer is the Director, Global Principal Consultant, Regulatory for the BioProduction Division (BPD) at Thermo Fisher Scientific. In this role, Michael is responsible for providing global support to BPD customers and serving as the regulatory thought leader and expert across all technology areas within BPD. Prior to moving to this role, he led the team responsible for product applications including microbiology, analytical sciences, and quality control. The products are fully integrated solutions for glycan profiling, bacterial and fungal identification, mycoplasma and viral detection, and host cell DNA and protein quantitation. Michael has over 30 years’ experience in the biopharma industry, including Scios, Synergen, and Amgen in a variety of roles including discovery research, analytical sciences, and quality control. Prior to joining Thermo Fisher Scientific, he led a group at Amgen that developed qualified, validated, and implemented molecular methods for host cell DNA quantitation, contaminant (mycoplasma, virus, and bacteria) detection, contaminant identification, strain typing, and genotypic verification of production cell lines.

Alissa M. Resch, PhD is Director of Biobanking Operations at Coriell Institute and a member of the Coriell Board of Trustees. Alissa provides oversight for all repository contracts and grants and supervises the project management teams to ensure project deliverables are met or exceeded. Alissa is also Principal Investigator of the National Human Genome Research Institute (NHGRI) Sample Repository, a diverse collection of cell lines and DNA that have contributed to several landmark initiatives, including the 1000 Genomes and International HapMap Projects.

Prior to joining Coriell, Alissa was an Assistant Professor of Genetics and Developmental Biology at the University of Connecticut, where she conducted research in the field of genomics and RNA biology. Alissa earned her doctorate in biochemistry at the University of California at Los Angeles. Upon completion of her graduate degree, she was awarded an NIH postdoctoral fellowship in bioinformatics at the National Center for Biotechnology Information (NCBI). She conducted a fellowship at the University of Connecticut before accepting a faculty position in the Department of Genetics and Developmental Biology.

John M. Baust, PhD is the Founder, President, and Lead Scientist of CPSI Biotech. Dr. Baust has over 20 years’ experience in research and medical device development and is a co-inventor on over 50 patents. Dr. Baust is a recognized innovator and entrepreneur in cryomedicine and is a pioneer in the area of molecular mechanisms of cell death and low temperature stress. Dr. Baust has published over 100 papers, reviews, book chapters, abstracts and patents in the area of low temperature biology and has been instrumental in the advancement of the field of cryobiology into the molecular biological era focusing on signal transduction and apoptosis. In this regard, Dr. Baust is credited with the discovery of cryopreservation-induced delayed-onset cell death (CIDOCD). In the area of research and technology development, Dr. Baust has led the development of numerous medical devices, including the Supercritical Nitrogen (SCN) and Pressurized Nitrogen (PSN) cryoablation devices for the treatment of cancer and cardiac arrhythmias. This is in addition to spearheading the development of the SmartThaw device for improved cell and tissue cryopreservation. Coupled with these technical engineering developments, he leads life science research programs focused on the cell-molecular actions of cryoablation. These efforts have resulted in the identification of a significant molecular stress response component to freezing injury which is responsible for the differential sensitivity of various cancers to thermal ablation. Through his previous work, Dr. Baust has been involved with the development and commercialization of several products including CryoStor and HTS-FRS (BioLife Solutions, Inc). In addition to these activities, Dr. Baust serves on the editorial boards of Biopreservation and Biobanking as well as Technology in Cancer Research & Treatment, and is a reviewer for several other scientific journals. Dr. Baust co-edited the book Advances in Biopreservation, is a past board member for the Society for Cryobiology, and board member of the American College of Cryosurgery. Dr. Baust completed his studies at Cornell University, State University of New York at Binghamton, and Harvard Medical School.

Marcin Łoś was born in Olsztyn, Poland. He began his studies at the Intercollegiate Faculty of Biotechnology of University of Gdansk and Medical University of Gdansk in Poland. He completed experiments for his MSc at University of Bradford (UK) and defended the MSc degree at University of Gdansk in August 1999. In September 1999 he started his PhD studies in molecular biology at University of Gdansk. He spent one year at the Fraunhofer Institute for Silicon Technology in Itzehoe, Germany (2002–2003) as a researcher involved in the construction of novel virus detection methods. He obtained his PhD degree in June 2004. In 2011 he also obtained a DSc degree. In 2007 he founded the company Phage Consultants, in which he holds the position of CEO. The company specializes in prevention and eradication of various contaminants from fermentation facilities and research laboratories and contract research in the field of phage biology, pharmacology, and biotechnology. His main scientific interests are bacteriophage biology and rapid detection methods. In 2017 Marcin became CEO of Acteryon, a company devoted to phage production and phage technology development.

Allen Burgenson was recently named the Global Subject Matter Expert for Testing Solutions at Lonza Walkersville Inc. in Walkersville, Maryland. Prior to this position he was the Regulatory Affairs Manager at that site for over 14 years. Allen has over 35 years of experience in endotoxin detection, both as an end-user and as a manufacturer of LAL assays, including 13 years as a Steering Committee Member for the LAL Users Group. He is currently the President of the Capital Area Chapter of the Parenteral Drug Association (PDA), and has served on several task forces to publish industry standard technical reports for that organization.

Antonio has over ten years of experience working in analytical testing positions for several industries, which include the pharmaceutical and medical device industries and criminal forensics. Antonio held positions in quality control for pharmaceutical repackaging and laboratory testing for criminal and industrial forensics before joining Gateway Analytical. He held roles as both a scientist and laboratory manager at Gateway Analytical before moving into business development. Antonio received his BS in Biochemistry from the Indiana University of Pennsylvania. He is currently a senior sales representative for Gateway Analytical and acts as a technical resource to help companies solve issues involving drug formulation and medical device development and foreign particulate identification and source determination investigations.