SPOTlights 2021

  • Preclinical/translational tools and strategies
    Feb 2021

    Preclinical/translational tools and strategies

    Bruce Bunnell
    Guest Editor:
    Bruce Bunnell, Professor and Chair at UNT Health Science Center
    Bruce Bunnell
    Bruce Bunnell
    Professor and Chair at UNT Health Science Center
    Bruce A Bunnell is a professor and Chair of the Department of Microbiology, Immunology and Genetics at the University of North Texas Health Science Center in Fort Worth, TX. Previously, he served as Director of the Tulane Center for Stem Cell Research and Regenerative Medicine and Professor in the Department of Pharmacology in the Tulane University School of Medicine. Dr. Bunnell obtained his PhD in Microbiology from the University of Alabama at Birmingham School of Medicine. He then pursued Postdoctoral Fellowship research at the Howard Hughes Medical Institute in the School of Medicine at the University of Michigan and the National Human Genome Research Institute at the National Institutes of Health in Bethesda, MD. Dr. Bunnell was an Assistant Professor at the Nationwide Children’s Hospital Research Institute, part of the Ohio State University School of Medicine prior to joining the faculty at Tulane University in 2002. Dr. Bunnell’s research program is focused on both the basic science and translational applications of adult stem cells isolated from the bone marrow and adipose tissue. Dr. Bunnell investigates use of mesenchymal stem cells (MSCs) isolated from the bone marrow or adipose tissue as a therapeutic intervention for both multiple sclerosis (MS) wound repair, lung injury and bone repair. He is particularly interested the interactions of MSC with the immune system and how the cells elicit robust anti-inflammatory effects in vivo. He is currently working towards a human clinical trials for the treatment of osteoarthritis, traumatic brain injury and MS with these cells. He has served as a reviewer of stem cell, regenerative medicine and tissue engineering grants for the National Institutes of Health, Department of Defense and several state funded programs including Maryland, New York, Virginia and Pennsylvania. He has also served on grant review panels for several foreign countries including Denmark, Ireland, Poland, Germany and Spain. Dr. Bunnell serves as an Editorial Board Member for several journals, including Stem Cells, BMC Genomics, and Regenerative Medicine.

    Assessing emerging technological innovation and collaborative strategies to drive time/cost efficiencies and valuable translational insights in non-clinical development

    • As the cell and gene therapy field expands into new, more complex diseases, pathways and targets, what are the key methods and tools, and where are most pressing innovation requirements, for preclinical/ translational R&D?
    • Where is tangible progress being made in developing animal models that can deliver clinically translatable insights?
    • How are novel/alternative non-clinical models and methods shaping up in terms of enhancing translatability to human diseases?
    • Preclinical assay 101: defining the key considerations/requirements and clarifying timeframes relating to successful preclinical assay development
    • Integrating early process development with preclinical/translational development

  • Raw and starting materials
    Mar 2021

    Raw and starting materials

    Elizabeth Read
    Guest Editor:
    Elizabeth Read, Principal Consultant
    Elizabeth Read
    Elizabeth Read
    Principal Consultant
    Elizabeth Read, MD is an independent consultant focusing on CMC development and CMC regulatory issues for cell- and tissue-based therapies. Dr. Read received her M.D. from the State University of New York (Buffalo, NY). After clinical training in Internal Medicine, Hematology, Oncology, and Blood Banking/Transfusion Medicine, Dr. Read worked at the National Cancer Institute and later in the Clinical Center’s Department of Transfusion Medicine at the National Institutes of Health (NIH; Bethesda, MD), where she served as Section Chief and Medical Director of the Cell Therapy Core Facility from 1995-2006. Initially engaged with novel cellular therapies in the context of hematopoietic transplantation, she later worked on more complex cell, tissue, and gene therapies for a range of clinical indications. From 2007-2010, she headed the Cell Therapy Program at Blood Systems Research Institute (San Francisco, CA), collaborating with UCSF investigators on grant-funded stem cell projects. She previously served as Medical Director at the American Red Cross Blood & Tissue Services (Los Angeles, CA). Over the past 10 years, she held leadership positions at small biotech companies, including Fate Therapeutics (San Diego, CA), StemCyte (Baldwin Park, CA), Medeor Therapeutics (San Mateo, CA), and Adicet Bio (Menlo Park, CA). Dr. Read has authored over 100 scientific publications, and has served as a faculty lecturer at UCSF and for the American Course on Drug Development and Regulatory Science. She has served on advisory committees focused on quality, safety, and efficacy of blood products and cell, tissue, and gene therapies, including the American Association of Blood Banks, the US DHHS/HRSA Advisory Council for Blood Stem Cell Transplantation, and the US Pharmacopeia. She currently serves as an advisor on clinical-stage cell therapy projects funded by the California Institute of Regenerative Medicine.

    Examining risk mitigation best practices and exploring emerging challenges/opportunities for an expanding cell and gene therapy sector

    • What lessons has the cell and gene therapy field learned about the raw and starting materials supply chain from the COVID-19 pandemic?
    • What is the latest progress in ensuring a sufficient supply of starting materials for specific, rapidly expanding cell and gene therapy technology areas?
      • Eg. how are donor management strategies and cell conversion methods evolving to enable the allogeneic cell therapy field?
    • Driving improvements in the consistency, reproducibility and traceability of reagents and other critical raw/ancillary materials for cell and gene therapy manufacture
    • How to predict, measure and manage the impact downstream of variability in cell therapy starting materials?
    • Key considerations and preparations for effectively managing materials supply risk and meeting regulatory requirements in the post-market approval setting.

  • Viral vector bioprocessing
    Apr 2021

    Viral vector bioprocessing

    Anindya Dasgupta
    Guest Editor:
    Anindya Dasgupta, Director, Vector Development at Expression Therapeutics
    Anindya Dasgupta
    Anindya Dasgupta
    Director, Vector Development at Expression Therapeutics
    Anindya is the director of vector development at Expression Therapeutics. He obtained his PhD from University of South Carolina, USA. His post-doctoral training and research associateship at the school of medicine, Emory University, Atlanta, USA, were focussed on the evaluation of novel anti-cancer therapies and the development of strategies for expansion and lentivirus based bioengineering of gamma delta; T cells in serum free media. Anindya is a co-inventor of a patent on anti-cancer strategy. At his recent role at Cincinnati Childrens Hospital Medical Centre he led vector development to manufacture high titer lentiviral vectors.

    Two decades on from the advent of commercial-scale protein therapeutics manufacture, is the viral vector space ready to make its own rapid advance towards industrialization?

    • Are we ready to deliver improvements required in upstream yield and downstream throughput to finally alleviate the current bioprocessing bottlenecks affecting both AAV and lentiviral/retroviral vector production?
    • Customized versus repurposed bioprocessing tools – what has been the impact on key steps in gene therapy processing from the emergence of more fit-for-purpose enabling technologies (eg. in the separation area)?
    • What is the latest progress in terms of cost and time savings in viral vector bioprocessing, and what elements should we target for further gains moving forward?
    • Key considerations for both in-house and outsourced viral vector manufacture in the current environment

  • Cell therapy CMC and quality control
    May 2021

    Cell therapy CMC and quality control

    Christiane Niederlaender
    Guest Editor:
    Christiane Niederlaender, Vice President Technical at Parexel
    Christiane Niederlaender
    Christiane Niederlaender
    Vice President Technical at Parexel
    Christiane Niederlaender. has spent 9 years at the UK medicines regulator MHRA, most recently as the Unit Manager of the Biologicals Unit. In that capacity she chaired and oversaw all formal scientific advice meetings and had in-depth involvement of Brexit preparedness. At the same time Christiane was a Senior Assessor and the UK delegate at the EMA committee for advanced therapies (CAT). She was UK CAT Rapporteur responsible for managing the portfolio of all UK ATMP products (cell and gene therapies). She was also the Rapporteur for the EMA CAT Gene Therapy Guideline and quality lead for gene therapy section of new draft guideline for investigational ATMPs. She was a drafting group member in all other quality related ATMP drafting groups, including GMP for ATMPs. Prior to joining MHRA, Christiane worked with the UK regulator for the European Tissues and Cells Directive as a quality manager and inspector. Christiane has experience in CMC and regulatory strategy for all types of biological products, with a particular focus on gene and cell therapies, novel biotherapeutics, biosimilars and vectored vaccines. While at MHRA, she was the principal quality/CMC assessor for 10 centralized MAAs, including two full dossier applications for ATMPs as sole UK quality assessor (including CAR-T, see publications). She has in depth knowledge of all European procedure types, both centralized and MR/DCP, and can give advice on ATMP classification, ODD and the PRIME program. As an ex-member of the EMA CAT committee, Christiane is familiar with the thinking of all European regulators in the ATMP space and is used to dialogue and negotiation with many regulatory agencies, as well as presenting her thinking during committee discussions. Christiane undertook her undergraduate studies in biochemistry, neurobiology and molecular biology at Heidelberg University in Germany before obtaining her PhD in molecular developmental neurobiology at Kings College London in the UK. Christiane has published her academic research in many high impact journals, including Nature.

    Learning lessons from recent setbacks, utilizing novel technological innovation, and defining key steps from early development towards delivering robust data packages

    • How will evolving regulatory guidance and manufacturing strategy trends impact approaches to demonstrating comparability and potency of cell therapy products?
    • Examining challenges in, and benefits from, implementing Quality by Design and Design of Experiments methodologies for cell therapy manufacture
    • Technology evolution: what efficiencies and consistency/quality related benefits are emerging process analytical tools delivering in practice to cell therapy developers and manufacturers?
    • Automation and associated analytics: what is the current state-of-the art in cell therapy?
    • How to effectively harness and extract value from large analytical data sets to the benefit of cell therapy manufacturers?
    • What progress in accelerating release testing to help further reduce manufacturing time frames?

  • Cellular immuno-oncology – overcoming manufacturing and development obstacles to commercial success
    Jun 2021

    Cellular immuno-oncology – overcoming manufacturing and development obstacles to commercial success

    Fritz Fiesser
    Guest Editor:
    Fritz Fiesser, Director, C&GT Engineering and Informatics at GlaxoSmithKline
    Fritz Fiesser
    Fritz Fiesser
    Director, C&GT Engineering and Informatics at GlaxoSmithKline

    How sustainable are the manufacturing and commercialization models of leaders in the cellular immunotherapy field?

    • What can we glean from macro investment, biophama innovation, and cancer healthcare trends to inform future cell and gene therapy clinical development, manufacturing and commercialization strategies?
    • How are leading developers of both autologous and allogeneic cellular immunotherapies and supporting tool and service providers alike addressing challenges relating to:
      • Cost of goods control?
      • Minimizing manufacturing and supply chain timeframes?
      • Optimizing bioprocess and supply chain quality/robustness?
    • What has the cellular immunotherapy sector learned from the COVID-19 pandemic?

  • Gene delivery platform evolution
    Jul 2021

    Gene delivery platform evolution

    How are cutting-edge R&D/technological innovation and a growing understanding of human and disease biology driving the expansion of gene therapy and gene editing into new, larger markets and indications?

    • Predicting next steps for gene therapy R&D in the traditional stronghold of rare monogenic diseases
    • Continuing the drive of gene editing into clinical application:
      • How are genome editing platforms evolving in the context of their applications in the burgeoning allogeneic cell therapy field?
      • Reviewing emerging gene editing platforms and their relative pros and cons versus established tools
    • How successfully are current R&D approaches, such as next-gen vector engineering, at tackling key obstacles to the widespread and long-term success of in vivo gene therapy?
    • Brave new world: assessing evidence of early progress in terms of gene therapies delivering both clinical efficacy and safer delivery strategies in larger indications

  • Clinical trial design, supply chain and operations
    Jul 2021

    Clinical trial design, supply chain and operations

    Ensuring strategic and operational excellence in the increasingly competitive world of cell and gene therapy clinical development

    • Assessing the longer-term repercussions of the COVID-19 pandemic for cell and gene therapy clinical development
    • How is competition for patients in key indications driving innovation in patient recruitment within the cell and gene therapy space?
    • Weighing up the pros and cons of recent innovation in both clinical development tools and trial designs
    • How should the cell and gene therapy field capitalize on the potential of Patient Reported Outcomes to bring both efficiencies and increased robustness to clinical development?
    • Adaptive trial designs
    • Biomarkers and surrogate markers linked to evidence of clinical effectiveness and response to treatment in cell and gene therapy: Recent progress and regulatory implications.
    • Capitalizing on increasing interaction and convergence between the cell and gene therapy, in vitro diagnostics, and medical device fields

  • Scale-up and scale-out - what do we really need and how will we get there?
    Sep 2021

    Scale-up and scale-out - what do we really need and how will we get there?

    Jan Thirkettle
    Guest Editor:
    Jan Thirkettle, Chief Executive Officer at Transine Therapeutics
    Jan Thirkettle
    Jan Thirkettle
    Chief Executive Officer at Transine Therapeutics
    Chief Executive Officer of Transine Therapeutics, UK based biotech developing SINEUPs (a novel lncRNA platform which upregulates protein expression) in ocular and neurodegenerative diseases. He was previously Chief Development Officer at Freeline, a clinical phase AAV gene therapy company with a novel AAV capsid platform. Jan has extensive experience in the development and commercialisation of novel platforms including NCEs, natural products, biologics and gene therapies, and has played a role in launching products in all these classes. Prior to joining Freeline Jan led the establishment of GSK&rsquo;s Cell and Gene Therapy platform and was responsible for CMC/supply for Strimvelis, the first&nbsp;<em>ex vivo</em> gene therapy to receive an EU Marketing Authorisation Application. Jan holds an MA in Chemistry and a PhD in Biological Chemistry from Oxford University.

    Identifying and addressing critical scalability bottlenecks in cell therapy and viral vector bioprocessing and supply chain to enable large-scale manufacture of advanced therapies

    • Considerations for effective viral vector scale-up and scale-out with both insect and mammalian cell culture systems
    • Improving the scalability of allogeneic cell therapy platforms (eg. iPSCs, extracellular vesicles)
    • What should the future of scaled-out commercial autologous cell therapy manufacture actually look like?
    • Assessing the advantages and limitations of current tools and processes through this lens
    • How to ensure that PAT-derived data management and integration doesn’t apply the handbrake to rapid, robust scale-up/-out?
    • What progress in delivering turnkey manufacturing platforms and solutions for the cell and gene therapy space to enable a stronger early-stage focus on scale-up?
    • Key do’s and don’ts for an effective integrated approach to materials supply chain and bioprocess scale-up

  • Gene therapy CMC and quality control

    Gene therapy CMC and quality control

    Christine Le Bec
    Guest Editor:
    Christine Le Bec, Head, CMC, Gene Therapy at Sensorion
    Christine Le Bec
    Christine Le Bec
    Head, CMC, Gene Therapy at Sensorion
    Christine Le Bec joined Sensorion Pharma in early 2020 as Head of CMC Gene Therapy. She is responsible for all CMC activities, including pre-clinical development, CMC transfer to CMOS, manufacturing and supplying of Phase 1 and 2 clinical trails. Before joining Sensorion Pharma, she worked for more than 20 years at Genethon in the field of Gene Therapy vectors (AAV, Lentivirus, Baculovirus) for rare diseases. She has a strong expertise in the development, qualification, validation of analytical methods for product characterization, release testing of gene therapy products and in stability studies. She has also a solid knowledge of International regulations and reviewing CMC documents for clinical trial applications.

    Is recent innovation in analytical tools delivering the required improvements in cost, speed and accuracy for vector manufacturing?

    • How and where are process analytical tools being successfully incorporated/integrated into viral vector bioprocess tools/steps, and where is further innovation required in this regard?
    • What is the state-of-the-art in accelerated release testing for gene therapy products?
    • Manufacturer and regulator perspectives on viral vector purity testing requirements and the utility of current tools
    • How will evolving regulatory guidance and manufacturing strategy trends impact approaches to demonstrating comparability and potency of gene therapy products?
    • Examining challenges in, and benefits from, implementing Quality by Design and Design of Experiments methodologies for gene therapy manufacture
    • Is it time to prepare for continuous manufacture of gene therapy products? If so, what are the initial steps?

  • New horizons for cell therapy: emerging platforms

    New horizons for cell therapy: emerging platforms

    Tamara Laskowski
    Guest Editor:
    Tamara Laskowski, Scientific Project Director at MD Anderson Cancer Center
    Tamara Laskowski
    Tamara Laskowski
    Scientific Project Director at MD Anderson Cancer Center
    Dr. Tamara J. Laskowski received her doctorate degree in Human Molecular Genetics and Immunology from University of Texas Health Science Center where her work focused on genome editing of patient-derived stem cells to correct genetic mutations causative of Wiskott-Aldrich Syndrome, an immunodeficiency disorder that results in severe impairments to the immune system. Subsequently, Tamara joined Dr. Laurence J.N. Cooper&rsquo;s laboratory at MD Anderson Cancer Center as a fellow. Her work focused on engineering stem cells with the goal of generating off-the-shelf NK and T-cell immunotherapies for targeting solid tumor malignancies. Though Tamara remains as a collaborator in the Cooper laboratory, her work has led her to transition to Dr. James Allison&rsquo;s Immunotherapy Platform at MD Anderson. In her new role as Senior Research Scientist, Dr. Laskowski&rsquo;s work primarily involves immune-monitoring of patients undergoing clinical trials in Immunotherapy and development of novel immunoassays. Dr. Laskowski also shares an interest in technology innovation, and has developed multi-plex assays for testing therapies against solid tumors. In 2017, this work led to an invitation to participate in the National Science Foundation (NSF) Innovation Corps, an exclusive program through which she was trained to develop strategies for expanding the economic and societal benefits of innovative ideas which have commercialization potential. She was the sole recipient of an award for outstanding performance upon completion of the program. In addition to her research work, Dr. Laskowski participates in several educational programs at MD Anderson. She is a lecturer at MD Anderson&rsquo;s School of Health Professions and also serves as a scientific communications coach teaching students and fellows how to effectively communicate science and medicine with scientific and lay audiences.

    An exploration of the cutting-edge in next-gen cell therapy technology platforms and applications spanning the cellular immunotherapy and stem cell therapy realms

    • Update on preclinical and early clinical-stage cell therapy platforms, with analysis of data generated to date, and discussion of how specific challenges and considerations relating to their ongoing development will be addressed – to include:                
      • Cellular Immunotherapies (eg. novel CAR T, TCR, Treg and NK cell therapy approaches) 
      • Exosomes                            
    • Progress on the migration into solid tumor indications – how are obstacles presented by the tumor microenvironment being approached?  
      • The future is now: how and where are novel single cell analysis and bioinformatics tools being applied to generate new targets and drive progress in tackline solid tumors with cellular immunotherapy?  
      • What is the latest thinking on the potential role/utility of cellular immunotherapy in the combination therapy setting?                       
    • Profiling the continuing expansion of engineered cells (eg. T cells, NK cells, macrophages) into non-cancer therapeutic areas and indications                               
      • Cell therapies against COVID-19 and beyond - examining the spread of cell therapy into more complex, challenging diseases.              
    • How will we expand patient access to tomorrow's cell therapies?  
      • Preparing at an early stage of R&D to ensure your cell therapy will have a viable and durable commercial future 

  • Cell therapy bioprocessing and automation

    Cell therapy bioprocessing and automation

    Qasim Rafiq
    Guest Editor:
    Qasim Rafiq, Associate Professor of Cell & Gene Therapy Bioprocess Engineering at University College London
    Qasim Rafiq
    Qasim Rafiq
    Associate Professor of Cell & Gene Therapy Bioprocess Engineering at University College London
    Associate professor at UCL&rsquo;s Advanced Centre for Biochemical Engineering, Qasim&rsquo;s research focus is on addressing translational challenges of robust manufacture of stem cells that are needed to take regenerative medicines into the clinic. This includes the &ldquo;whole bioprocess&rdquo; from sourcing of raw materials and cells, through cell expansion in bioreactors and ultimately the downstream processing needed to purify and concentrate cell product for delivery to the patient. He previously lectured in Bioprocess Engineering at Aston University, where he was Principal Investigator on 4 grants with funding from RCUK, EU Horizon 2020, InnovateUK and commercial sources. Qasim completed his PhD at Loughborough.

    Assessing progress to date and planning next steps in increasing robustness of cell therapy manufacture, and in reducing COGs and cell therapy bioprocessing/analytical timeframes

    • Learnings for the cell therapy manufacturing space from the experiences of COVID-19 vaccine manufacturers
    • How far have we come in automating/industrializing cell therapy manufacture, and how far have we got to go? Assessing successes and setbacks to date, as well as key areas for future focus    
      • Bioprocess technology update: how is the increasing availability of closed, modular bioprocessing equipment tailored for the cell therapy field changing the picture for the sector?
      • Automation of analytics: what progress has been made in advancing cell therapy towards ‘Bioprocess 4.0’?
      • Regulatory update: what impact will recent guidance and ongoing trends in regulatory scrutiny have on the field?
    • What do the latest cost analyses for centralized and various distributed cell therapy manufacturing models tell us about their current relative efficiency? How is this picture evolving?             
      • Just how far away – and how desirable - is point-of-care manufacturing? Healthcare sector, industry, academic and regulatory perspectives           
    • Exploring the interface between tool development and trends in cell therapy manufacturing facility design – what do they mean for the cell factory of the future?  

  • Tools of tomorrow

    Tools of tomorrow

    Cell & Gene Therapy Insights’ annual review of the year just gone, with trends analysis providing pointers to the likely big stories and developments in the 12 months ahead.

    Combined with an exploration of enabling tools and therapeutic technology platforms likely to make a splash in 2022.