A human focus for 21st century life sciences
Science+ meeting organised by Professor Geoffrey J Pilkington, Ms Rebecca Ram and Ms Kathy Archibald on behalf of the Alliance for Human Relevant Science.
This meeting showcased the transformative value of innovative, human-focused biomedical technologies, which are providing invaluable insights into human diseases and could underpin the development and optimal use of new and repurposed medicines. It brought together researchers, clinicians, industries, regulators and others to discuss key challenges and how to surmount them to to accelerate their advantageous development and implementation.
Image credit: iStock.com / sturti
Organisers
Schedule
Chair
Professor Michael Coleman
Aston University, UK
Professor Michael Coleman
Aston University, UK
Professor Coleman graduated with first-class honours in Pharmacology and a PhD in Biochemical Pharmacology from Liverpool University. He spent two years as a National Research Council Resident Research Associate at Walter Reed Army Institute of Research, Washington DC, then, after completing a Wellcome Fellowship in Toxicology he joined Aston University. His research interests since 1991 have been focussed on developing human experimental models in toxicology and latterly neurotoxicology.
| 09:00-09:05 |
Welcome by the Royal Society and lead organiser
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK Geoff Pilkington is an Emeritus Professor of Neuro-oncology, having held chairs at King's College, London and the University of Portsmouth, where he was Director of the Brain Tumour Research Centre which exclusively used human cells, tissues and biomaterials in their research. He is a Past President of the British Neuro-oncology Society and Honorary Treasurer, Executive Board Member and Scientific Board Member of the European Association of Neuro-oncology as well as being a Fellow of the Royal College of Pathologists and a Fellow of the Royal Society of Biology. His published work was largely focussed around brain tumour invasion, the blood brain barrier, the tumour microenvironment, brain tumour modelling and repurposed drugs. |
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| 09:05-09:25 |
A 50-year career reflection on models of human disease in research and pre-clinical testing: where are we now?
During Professor Pilkington’s long career in neuro-oncology, he developed novel models for the biology and development of brain tumours and pre-clinical testing systems for drug efficacy, delivery, and mechanisms of therapeutic resistance. In his early career, such models were developed in rats and mice, but he became aware that in addition to the ethical issues around this approach, such approaches, while of scientific interest, did not represent the situation in human neuro-oncology. Nor did they accurately represent other human neuropathologies, including dementias. He will allude to examples of where animal models were, and still are, imperfect. Later in his career, he used in vitro approaches derived from human biopsy tissues which, although initially suffering from other flaws, due to poor design, were adapted and improved. These models became increasingly sophisticated and more akin to human brain pathologies. Ultimately, he established a brain tumour research centre which focussed on human biopsy-derived cells in in vitro models of the blood-brain barrier, brain tumour invasion/microenvironment and pre-clinical testing in 3D multiple cell type assays, providing a more accurate representation of human disease. He will address the remaining flaws in both animal and ‘all-human’ modelling as well as how advanced human modelling is now ready to be exploited to determine the likelihood of well-designed clinical trials leading to successful outcomes for patients. He will close by suggesting that the orchestration of multiple new advanced methods will result in safer predictions for clinical trial outcomes, particularly in the testing of repurposed and re-formulated drugs.
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK Geoff Pilkington is an Emeritus Professor of Neuro-oncology, having held chairs at King's College, London and the University of Portsmouth, where he was Director of the Brain Tumour Research Centre which exclusively used human cells, tissues and biomaterials in their research. He is a Past President of the British Neuro-oncology Society and Honorary Treasurer, Executive Board Member and Scientific Board Member of the European Association of Neuro-oncology as well as being a Fellow of the Royal College of Pathologists and a Fellow of the Royal Society of Biology. His published work was largely focussed around brain tumour invasion, the blood brain barrier, the tumour microenvironment, brain tumour modelling and repurposed drugs. |
| 09:25-09:45 |
The new revolution in medicine: AI, imaging and the future of health care
The spreading of digital health innovation and personalized medicine together to the related regulatory shifts are the challenges of next future. The diffusion of AI worldwide appears to be a key point in the development of the most innovative devices allowing an easier and faster access to the cutting-edge technologies supporting the clinical practice and optimizing the management of health care related costs. AI techniques concerning process mining in medical procedures will be the cornerstone in the balance between resources and clinical needs, improving the standard of care for patients. Generative AI creates new data and opportunities especially when applied to medical imaging also supporting research and improving the development of new technologies in medical imaging through data analysis. Radiomic features may be used to allow an earlier disease detection, improving prognostic assessment as well as a better treatment strategy definition. The future of precision medicine relies on imaging, achieved by the implementation of radiomics technologies, and theranostics, with the same agent able to diagnose and treat simultaneously. This will be the best example individual personalized medicine. Generative AI has also demonstrated the power to increase the diagnostic performance, improving the accuracy and precision, potentially reducing costs and risks for population. Application of generative AI regulation changes is often slower than innovation. Anyway, AI requires legislative changes, and one of the most significant challenges will be managing technological progress as fast as it is need ensuring the optimal regulatory context while preserving every opportunity for progress within the healthcare system.
Professor Orazio SchillaciItalian Minister of Health
Professor Orazio SchillaciItalian Minister of Health Professor Orazio Schillaci MD, 59-year-old physician, is the Italian Minister of Health since October 2022. Before being appointed as Minister of Health, he held the position of Rector of the University of Tor Vergata (Rome) since 2019. He is a full professor of Nuclear Medicine and former Dean of the Faculty of Medicine at the same University. In 2020 he was appointed as a member of the Scientific Committee of the National Health Institute. He has held positions in numerous scientific bodies. He is the author of more than 350 scientific publications in peer reviewed journals and a member of the editorial boards of numerous international scientific journals. |
| 09:45-10:00 |
Discussion
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| 10:00-10:20 |
Break
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| 10:20-10:40 |
HNRNPD: Guardian of the (beta cell) galaxy
Cellular stressors are inducers of cell identity change. In the pancreatic islet, factors such as high glucose and dysregulated lipid can cause cellular transdifferentiation events. This has been observed in human beta cells in vitro and in islets of people with diabetes. Using a completely humanised system, we investigated the causes of pancreatic cell identity change by defining global gene expression patterns in normal and transdifferentiated cells after exposure to stress. We then tested the most dysregulated genes for causality using siRNA gene knockdown and hormone staining. We then investigated the impact of splicing-associated changes in the binding and expression of downstream targets. Finally, we explored the potential for reversal of cell identity changes by restoration of euglycaemia or manipulation of candidate isoform levels. We identified the heterogeneous ribonucleoprotein D (HNRNPD) gene as a potent protector of beta cell identity in the face of cellular stress. Cellular stressors caused a change in the splicing patterns of HNRNPD, with consequent disruption of the downstream beta cell development and function gene expression programs. We were able to demonstrate reversal of transdifferentiation events by removal of stress or manipulation of HNRNPD isoforms. We have identified HNRNPD as a key factor involved in the protection of beta cell identity. HNRNPD splicing changes induced by disrupted metabolism cause a reversible switch from a beta cell to delta cell profile. The nature of the change is human-specific and underlines the importance of using human-relevant experimental systems to identify human-relevant disease processes. These findings may represent a potential future point of traction for protection of beta cell mass in face of diabetes.
Professor Lorna HarriesExeter University, UK
Professor Lorna HarriesExeter University, UK Professor Lorna Harries is co-founder, director and Chief Scientific Officer at SENISCA Ltd, a spin out company from the University of Exeter Medical School, founded on the back of 10+ years of academic research from the Harries lab. Lorna gained her PhD from University College London in 1994 and holds a personal chair in Molecular Genetics at the University of Exeter Medical School. Lorna also heads the Exeter Animal Free Research Centre of Excellence (ARC 2.0) funded by Animal Free Research UK. The Harries lab have interests in -omics approaches to the study of ageing and age-related disease processes in humans, and her work takes a genes to systems approach, ranging from ‘big data’ analyses to detailed individual molecular analysis of particular genes. Her team were the first to report dysregulation of alternative splicing as a new, and druggable, hallmark of ageing; a finding which is now being developed as the basis for a new generation of interventions to treat the underpinning causes, not just the symptoms, of age-related chronic disease. |
| 10:40-11:00 |
Drug-induced liver injury: Genetic architecture to precision medicine
Liver is one of the most common organ toxicities observed during pre-clinical development, yet the signals of hepatotoxicity found in animal models are deemed unreliable. A systematic review demonstrated liver and immune related adverse events in combination accounted for 30% of the market withdrawal of medications. Among 62 drugs approved by FDA in 2021, 58% have been shown to have hepatotoxicity in clinical trials of listed in the official label. Host genetic factors are important in determining the susceptibility to idiosyncratic drug-induced liver injury (DILI) which is the most common cause for acute liver injury and the second 3rd in causes of jaundice presenting to secondary care hospitals. Through large international collaborations, and involvement of robustly evaluated patients with DILI, we have identified several human leucocyte antigen (HLA) and non-HLA genetic risk factors increasing susceptibility to DILI. Overlapping HLA alleles determine risk of DILI due to more than 20 drugs. Genetic risk scores using multiple genotypes have been developed to assist the diagnosis and causality assessment of DILI due to specific drugs. Genetic architecture determines the DILI potential of several drugs in organoids derived from primary hepatocytes or hepatocyte like cells derived from inducible pluripotent stem cells. This ‘polygenicity in dish’ concept will be the path to developing human relevant in-vitro models for the pre-clinical evaluations during the drug development. Recent cluster randomised controlled implementation trial demonstrated that a platform with 50 variants related to 12 genes contributing to pharmacogenomic alteration in 44 drugs can reduce adverse drug reaction by 30%. This has set a path for embedding precision medicine in the community.
Professor Guruprasad Padur AithalUniversity of Nottingham, UK
Professor Guruprasad Padur AithalUniversity of Nottingham, UK Guruprasad Aithal, MBBS (Mangalore University), MD (General Internal Medicine; Bengaluru University), FRCP (Edinburgh, London), PhD (University of Newcastle), Advanced International Fellow (Medical University of South Carolina) is Professor of Hepatology, University of Nottingham and Honorary Consultant Hepatobiliary physician, Nottingham University Hospitals. He was the Director of National Institute for Health Research (NIHR) Nottingham Digestive Diseases Biomedical Research Unit (2008-17) and is currently the Deputy Director/ Gastrointestinal and Liver Theme Lead of the NIHR Nottingham Biomedical Research Centre (2017-28). He was the President of British Association for the Study of the Liver: 2020-21. His research interests include the genomics of complex and monogenic liver disorders, metabolic physiology chronic liver diseases and multiorgan quantitative magnetic resonance imaging/ spectroscopy. He co-chaired international drug-induced liver injury (DILI) consortium that delivered several breakthroughs; he is now the Deputy Co-ordinator and DILI work package lead of the TransBioLine (2019-25), programme of the Innovative Medicines Initiative of the European Union. He was the joint winner of the NHS Innovation Challenge in 2013 for developing multi-award winning ‘Scarred liver project’. He has obtained major funding (>£147M) for research across the Liver and Gastrointestinal disease spectrum. He has authored over 360 publications with H index of 88. |
| 11:00-11:15 |
Discussion
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| 11:15-12:15 |
Roundtable discussion: Why should medicine be human-focused?
Dr David BuntonREPROCELL Europe Ltd, UK
Dr David BuntonREPROCELL Europe Ltd, UK David is CEO of REPROCELL Europe Ltd. David graduated from the University of Glasgow then undertook a PhD in pharmacology at Glasgow Caledonian University, which led to his appointment as a Lecturer in Physiology. In 2002, he co-founded Biopta and successfully grew its contract research business by providing lab services to the pharmaceutical industry, founding a US subsidiary, Biopta Inc. in 2011. Biopta Ltd and Inc. were acquired by REPROCELL Inc. (Yokohama, Japan) in Dec 2015. Following the acquisition, REPROCELL Europe was formed from the merger of two Group companies, Biopta Ltd and Reinnervate Ltd (Durham, UK), with Glasgow becoming REPROCELL’s European headquarters. The business moved to new premises on the West of Scotland Science Park, which were officially opened by the First Minister of Scotland in September 2017. David also co-chairs the Internationalisation Sub-Group of the Life Sciences Scotland Industry Leadership Group, is on the Board of Glasgow Economic Leadership, where he chairs the Life Sciences Workstream, and is a member of the Scottish Advanced Therapies Network. David’s recent focus has been on the use of AI in precision medicine, including a collaboration with the Hartree National Centre for Digital Innovation.
Professor John GreenmanUniversity of Hull, UK
Professor John GreenmanUniversity of Hull, UK John is a tumour immunologist at the University of Hull who has morphed into someone who spends most of his time developing lab-on-a-chip technology for personalized medicine against solid tumours. His interdisciplinary biomicrofluidics group, comprising clinicians, chemists and engineers, has been pioneering the development and adoption of bespoke microfluidic devices for almost 20 years. The group is equipped to design and fabricate devices from a range of materials depending on the desired end-use, recently moving more to using 3D-printed devices in biocompatible polymers. The group has extensive experience of maintaining different tumour types, in a viable state, including glioblastoma, head and neck squamous cell carcinoma, thyroid, renal and colorectal tissues. In addition, the group has undertaken extensive downstream analysis of effluents and tissue at the molecular (miRNA, mRNA, and circulating tumour DNA) and protein levels, allowing detailed analysis of clinically-relevant treatments of tissue on devices.
Professor Christine MummeryLeiden University Medical Center, The Netherlands
Professor Christine MummeryLeiden University Medical Center, The Netherlands Christine Mummery is Professor of Developmental Biology at Leiden University Medical Center. Following a PhD in Biophysics, she moved to the Hubrecht Institute to study ion channels and later, cardiovascular diseases using pluripotent stem cells from patients, developing organ-on-chip models. This has centred on safety pharmacology to predict toxic effects of drugs on the human heart and capturing cardiac and vascular disease phenotypes including identifying individual vulnerability and drug sensitivity. She was president of the International Society for Stem Cell Research (2020-2021) and co-founded the European Organ-on-Chip Society. She is also a member of the Royal Netherlands Academy of Science. In 2021, she was awarded the Lefoulon Delalande Prize jointly with Gordon Keller. She is currently on, or chairs, advisory boards of Sartorius AG (Germany), HeartBeatBio (Austria), Mogrify UK, Cellestic, Founding (now Associate) Editor of the ISSCR journal Stem Cell Reports, among others.
Professor Denis Noble CBE FMedSci FRSUniversity of Oxford, UK
Professor Denis Noble CBE FMedSci FRSUniversity of Oxford, UK Denis Noble is Emeritus Professor of Cardiovascular Physiology based at the Department of Physiology, Anatomy & Genetics at Oxford University. He is author of many books, including his latest ones: The Pacemaker Channels of the Heart (World Scientific 2025), and Understanding Living Systems (CUP 2023). The work for which he is most well-known is his demonstration that the origin of the heartbeat lies in its ability to excite itself spontaneously through the interaction of cellular electrical potential and the ionic channels responsible for generating ionic currents. During the last 20 years he has also published widely on evolutionary biology: https://www.denisnoble.com/evolution-publications/.
Professor Helen MaddockInoCardia, UK
Professor Helen MaddockInoCardia, UK Professor Helen Maddock is the Founder, Director and Chief Scientific Officer at InoCardia Ltd, a spin out company from Coventry University, supported by 15+ years of academic research from the Maddock lab. Helen is Professor of Cardiovascular Physiology and Pharmacology at Coventry University and an Honorary Professor at Warwick Medical School, University of Warwick. She has over 30 years of experience spanning academia, life sciences, clinical sciences, and industry — including time with AstraZeneca and GSK early in her career. Helen completed a BBSRC industry funded PhD studentship supported by GSK and received her doctorate from the Welsh School of Pharmacy, Cardiff University. Followed by post doctoral research funded by the British Heart Foundation, at the world leading Hatter Cardiovascular Institute, University College London. During this time she pursued novel pharmacological and therapeutic strategies to protect the heart from the consequences of ischaemic and reperfusion-induced injury. She is known for her work in industry for driving forward improved and predictive human relevant preclinical microphysiological system (native, engineered cells and tissue) and computational methods for cardiac efficacy and safety assessment. She is passionate about the development and use of clinically relevant New Approach Methodologies (NAMs) to deliver predictive data that has human clinical concordance. Helen’s goal is to support the improvement and transformation of how we assess cardiovascular safety and efficacy during pre-clinical development focussed on human-relevant methods — with the aim of making drugs safer and effective. She is the recipient of multiple industry awards including an Innovate UK Women in Innovation Award, and a leading advocate for translational approaches that connect science with real-world impact.
Dr Mona BoyéKsilink, France
Dr Mona BoyéKsilink, France Mona Boyé holds a PhD in molecular biology from Cardiff University. Shortly after her postdoc, she transitioned into technology transfer and bioscience innovation. She is currently Chief Business Officer at Ksilink, a French Tech company specializing in patient-based phenotypic drug discovery. |
Chair
Dr J Malcolm Wilkinson
Sheffield University, UK
Dr J Malcolm Wilkinson
Sheffield University, UK
Dr Wilkinson studied Physics at Oxford University and then worked in industry for 16 years developing microelectronics. In 1994 he started a consultancy in Cambridge supporting the development of microfluidics devices for medical diagnostics. From 2006 to 2019 he was CEO of Kirkstall Ltd, one of the world’s leading Organ on a Chip companies. Although now retired, he continues as a Director of Kirkstall Ltd., and is on the Scientific Advisory Panel of Animal Free Research UK supporting their work to replace the use of animals in medical research and drug testing. He has been a visiting Lecturer for FSRM, Neuchatel, Switzerland, on the subject of Micro and Nanotechnology in Biomedical Engineering for over ten years and was appointed as a Visiting Professor in the Department of Biomedical Engineering at Sheffield University in 2019.
Dr Wilkinson is co-author on several papers on in-vitro models of toxicity and a contributing editor of a recently published book on In-Vitro Testing. He has recently published a review article on the status of Organ on a Chip technology.
| 13:15-13:20 |
Chair's introduction
Dr J Malcolm WilkinsonSheffield University, UK
Dr J Malcolm WilkinsonSheffield University, UK Dr Wilkinson studied Physics at Oxford University and then worked in industry for 16 years developing microelectronics. In 1994 he started a consultancy in Cambridge supporting the development of microfluidics devices for medical diagnostics. From 2006 to 2019 he was CEO of Kirkstall Ltd, one of the world’s leading Organ on a Chip companies. Although now retired, he continues as a Director of Kirkstall Ltd., and is on the Scientific Advisory Panel of Animal Free Research UK supporting their work to replace the use of animals in medical research and drug testing. He has been a visiting Lecturer for FSRM, Neuchatel, Switzerland, on the subject of Micro and Nanotechnology in Biomedical Engineering for over ten years and was appointed as a Visiting Professor in the Department of Biomedical Engineering at Sheffield University in 2019. Dr Wilkinson is co-author on several papers on in-vitro models of toxicity and a contributing editor of a recently published book on In-Vitro Testing. He has recently published a review article on the status of Organ on a Chip technology. |
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| 13:20-13:35 |
Optional ornament or crucial code? The role of modelling and simulation in human safety assessment
Human safety assessment in drug development is undergoing significant change, driven by high attrition rates, ethical concerns about animal testing, and new global regulations encouraging alternative methods. Quantitative Systems Toxicology (QST), a key technology within New Approach Methodologies (NAM), uses mathematical modelling and experimental data to predict toxicological effects more mechanistically than traditional models. QST provides mechanistic, human-relevant evidence vital for decision-making, risk assessment, and regulatory submissions. International regulators increasingly recognise QST, as seen in recent guidelines from the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use on General Principles for Model-Informed Drug Development (ICH-M15) and updates like the FDA Modernization Act 2.0. QST offers mechanistic insights into toxicity, improves translational predictions of human responses, and supports efforts to reduce animal testing, aligning with the 3Rs principle (reduction, refinement, replacement). Case studies in organ-specific toxicities, and advanced therapies show QST’s impact on safer drug design and regulatory approval. As drug safety continues to evolve, QST is set to become a crucial, rather than optional, part of the drug development and safety assessment, advancing scientific and ethical standards in modern drug development.
Dr Ciarán FisherGSK, UK
Dr Ciarán FisherGSK, UK Ciarán Fisher is an accomplished quantitative systems toxicologist, presently holding the position of Associate Director in Systems Toxicology and Data at GSK. He earned his PhD in Molecular Toxicology from the University of Surrey. Throughout his career, Dr Fisher has made significant contributions to the advancement of mathematical models for predicting drug and chemical safety, with a focus on integrating and interpreting both in vitro and in vivo nonclinical data to support QST modelling strategies. He has participated in several high-profile projects, including the EUTOXRISK Horizon 2020 initiative and the IMI2 public-private partnership project, Translational Quantitative Systems Toxicology (TransQST). His academic experience includes a postdoctoral fellowship on a BBSRC-funded project investigating gene regulatory network dysregulation in the pathogenesis and progression of non-alcoholic diseases. Dr Fisher has authored or co-authored 25 peer-reviewed publications. He is a member of the British Toxicology Society (BTS) and serves as joint coordinator of the BTS in silico and computational specialty section. |
| 13:35-13:50 |
Human-based in silico medicine for drug development
Precision Medicine aims at providing the most accurate diagnosis and best treatments for each patient. Whereas this has primarily been genomic-centred so far, there is now a wide recognition of the need to consider a wide spectrum of lifestyle, environment, and biology conditions. Characterising such diversity of factors requires large quantity and quality of patients’ datasets, and at the same time, innovative approaches for their analysis, drawing on the increasing power of computers and algorithms. In this presentation, I will illustrate progress towards the vision of the Digital Twin and In Silico Trials for therapy testing, through human-based modelling and simulation for prediction medicine, therapy development, and the 3Rs of animals in research. These concepts are broadly applicable through all areas of medicine, and I will describe our own progress in cardiology and drug safety assessment. I will aim to demonstrate how combined computational approaches, including modelling and simulation and machine learning, can boost the capacity for diagnosis and prognosis, as well as testing of future treatments.
Professor Blanca RodriguezUniversity of Oxford, UK
Professor Blanca RodriguezUniversity of Oxford, UK Professor Blanca Rodriguez is Professor of Computational Medicine, Wellcome Trust Senior Research Fellow, and Head of the Computational Biology and Health Informatics Theme at the Department of Computer Science, University of Oxford. She aims to accelerate medical therapy development through augmenting clinical and experimental data with modelling and simulation, and machine learning. Her team has contributed pioneering case studies for the Digital Twin vision in precision medicine, and In Silico Trials for therapy testing. She actively collaborates with industry and leads the Computational Cardiovascular Science team at Oxford, an interdisciplinary, diverse and award-winning research team. Originally, she is from Valencia, Spain, where she trained in Engineering at the Universidad Politecnica de Valencia (MSc and PhD). She then worked as a postdoc at Tulane University in New Orleans, USA for two years. After this, she joined the University of Oxford, where she was awarded six competitive personal research fellowships and in 2014 the professorship in Computational Medicine. |
| 13:50-14:05 |
Enhancing preclinical risk assessment with computational tools
If impactful safety issues are first detected during the pre-IND GLP in vivo safety studies, this is far too late. The chemistry is already ‘locked-in’ to the candidate drug and there will have been significant investment in terms of compound synthesis of sufficient quantities for Phase I clinical trials and the cost of the tox IND package, not to mention the resource costs of getting to that point. To minimise the risk of this, toxicologists and safety pharmacologists generate in vitro safety data long before then. However, this only provides a patchy coverage of the entire spectrum of potential toxicities. There is a clear opportunity to (a) construct QSAR/QSTR models to flag-up various safety issues even before compound synthesis and then, once compounds are synthesised, (b) to apply computational safety tools to improve interpretation of in vitro readouts. By focusing on the most common and impactful preclinical safety issues (hepatotoxicity; QT prolongation; off-target safety), the discovery small molecule pipeline can be significantly de-risked during lead optimization. Going forwards, to accord with the FDA’s 2025 Roadmap to Reducing Animal Testing in Preclinical Safety Studies and the UK Government’s 2025 Replacing Animals in Science Strategy, and replace in vivo safety studies with New Approach Methodologies (NAMs), will require a coordinated involvement of both in vitro and in silico technologies. Some of these will have to be promoted from their current front-loading role to a frontline one, as well as the development of new ones.
Dr Will RedfernCertara, UK
Dr Will RedfernCertara, UK Will Redfern is Vice President of Quantitative Systems Toxicology & Safety (QSTS) in Certara. He is an experienced safety pharmacologist, having previously worked at Syntex, Quintiles and AstraZeneca, contributing to over 10 drugs that successfully reached the stage of product registration. Will has co-authored over 50 papers, review articles and book chapters, and is a Fellow of the Royal Society of Biology and of the British Pharmacological Society, a Diplomate in Safety Pharmacology, and an Honorary Senior Lecturer at the University of Manchester. Will is a former President of the Safety Pharmacology Society (SPS) and has received several of its awards, namely the Distinguished Service Award (2014), the Technological Innovation Award (2018) and the Translational Research Publication Award (2023). He is co-chair of the newly formed SPS Biological Modelling Group. Will has lectured on courses run by the universities of Manchester, Birmingham, Cardiff, Surrey, Cambridge, Liverpool and King’s College London. |
| 14:05-14:20 |
Discussion
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| 14:20-14:45 |
Break
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| 14:45-15:00 |
From promise to practice: enabling human relevant, pharma-ready life sciences through policy, regulation, standards, and platforms
Human relevant technologies such as New Approach Methodologies (NAMs), microphysiological systems (MPS), and organ-on-chip platforms are increasingly recognised as essential components of modern biomedical research and therapeutic development. Their growing importance reflects advances in experimental science, alongside supportive shifts in regulatory thinking, policy frameworks, AI and the development of shared standards that together are reshaping expectations of evidence and relevance. Recent policy and regulatory developments highlight this changing landscape. In the United States, the FDA Modernization Acts have moved regulatory guidance away from prescriptive animal testing requirements toward broader acceptance of nonclinical evidence, explicitly enabling the use of human relevant and computational approaches. In the UK the national strategy for alternative methods similarly emphasise innovation grounded in human biology, while seeking to accelerate validation, confidence, and uptake across research and development. These signals are reinforced by increasing attention to standards, qualification pathways, and cross sector alignment. At the same time, it is clear that human relevant platforms will not address every scientific or regulatory challenge, nor will they replace all animal models wholesale in the near term. NAMs, MPS, and organ on chip technologies are particularly powerful when applied to well defined questions, such as human specific mechanisms, integrated multi cellular and some system level functions, and aspects of safety assessment. Supporting technologies, including data driven and AI enabled analytical tools, play an important enabling role, helping to integrate evidence, manage complexity, and support decision making. This talk will take a strategic, forward-looking view of how policy, technology platforms, standards, and enabling analytics are supporting the transition from promise to practice in human relevant life sciences. It will highlight where momentum is building, where challenges remain, and how the next decade could see these approaches more effectively embedded to support more predictive and efficient drug discovery and development, and ultimately improve outcomes for human health.
Dr Dharaminder SinghFormerly CN Bio, UK
Dr Dharaminder SinghFormerly CN Bio, UK Dr Dharaminder Singh is a recognised leader in bioengineering and microphysiological systems (MPS), with a proven track record of driving innovation and strategy in next-generation drug discovery platforms. With deep expertise spanning engineering, biology, and translational science, he has directed high-impact collaborations across pharmaceutical, biotechnology, and academic sectors to accelerate the adoption of new approach methodologies (NAMs). Dharaminder’s work has shaped the development and global application of organ-on-a-chip technologies, advancing regulatory engagement, standardisation efforts, and qualification guidelines. A strong advocate for MPS integration in research and industry, he contributes to international initiatives that aim to transform drug development pipelines, and serves as a member of the UK Government’s Animals in Science Committee. Dharaminder holds an MEng in Biomaterials Science and Tissue Engineering and a PhD in Bioengineering from the University of Sheffield. |
| 15:00-15:20 |
Modelling health and disease on a chip: the rapidly evolving field of micro-physiological systems
Animal models, and mice especially, have proven critical in developing an understanding of human disease, but they are not without their limitations. Mice are short-lived relative to humans, their metabolism differs in significant ways and they genetics differs significantly enough that some human diseases simply cannot be meaningfully modelled. These limitations and the growing push to reduce the use of experimental animals has driven the rise of microphysiological systems (MPS), also called complex in vitro models (CIVM) and more colloquially, organ-on-chip. The vasculature is a multifunctional organ critical for life. In addition to facilitating the distribution of oxygen and nutrients across tissues of the body, blood vessels play a key role in the active transport of immune cells, metabolites, and therapeutics, and in the regulation of blood pressure and the maintenance of normal body temperature. MPS platforms that include perfused human vasculature thus offer a more physiologic model system in which to further advance our understanding of human disease. We have developed the Vascularized Micro-Organ platform (VMO), which incorporates a microfluidic artery and vein connected by a spontaneously assembled microvascular network. A blood substitute is driven through the vasculature by gravity and supports surrounding tissue, which can either be a generic stroma, organ-specific, or a tumour. Based on this platform we have developed models for several diseases including cancer, diabetes and vascular malformations. We are using these to investigate biological mechanisms underlying disease, and to identify and validate therapeutic targets.
Professor Christopher HughesUniversity of California, Irvine, US
Professor Christopher HughesUniversity of California, Irvine, US Professor Hughes is a Chancellor’s Professor and Associate Dean of Research and Innovation at UC Irvine. His research focuses on the development and growth of blood vessels, and he is a pioneer in the field of microphysiological systems (organ-on-chip), specializing in the creation of vascularized and perfused tissue-on-chip models. The technology is now licensed to Aracari Biosciences, for which Dr Hughes is a founder and CSO. He served for 10 years as chair of his department and previously served as Director of UCI’s Advanced Cardiovascular Technology Center, and as a Program Leader in the Cancer Center. He is an elected Fellow of the American Association for the Advancement of Science (AAAS), and works extensively with the non-profit organization, cureHHT, where he chairs their Scientific and Medical Advisory Council. In 2022 he received their Lifetime Achievement Award. |
| 15:20-15:30 |
Discussion
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| 15:30-16:30 |
Roundtable discussion: Is the government correct that the use of animals will be essential for some time to come?
Professor Richard LuxtonUniversity of the West of England, UK
Professor Richard LuxtonUniversity of the West of England, UK Richard Luxton, Director of the Institute of Bio-Sensing Technology (IBST) at UWE Bristol, specializes in rapid point-of-care measurements. Worked in the NHS as a clinical chemist and completed his PhD in neuro-immunology at the Institute of Neurology, London. He founded IBST in 2008 to drive industry–academia collaboration and helped launch Medilink South West (Chair since 2012). He co-created the Health Technology Hub (2018) and leads the NHS South West Centre of Digital Excellence (2024). Richard has led major multi-partner projects and is Conference Chair of the International Conference for Bio-sensing Technology and Editor-in-Chief of Sensor and Biosensor Research. His recent research interests include the contactless cell measurements, and the development of continuous monitoring of cell response to physiological and pathological challenges.
Dr Gerry KennaKenna Consulting, UK
Dr Gerry KennaKenna Consulting, UK Dr Gerry Kenna is a Drug Safety Consultant based in Cheshire, UK. His initial scientific training was in biochemistry, at the Universities of Leeds (BSc Hons) and London (PhD). He established and led academic research teams that explored mechanisms by which medicines and other chemicals can damage the liver. Dr Kenna then moved to industry (at Zeneca, Syngenta and AstraZeneca), where he supported human safety assessment of new medicines and agrochemicals and also led teams (and international consortia) that developed improved human safety testing methods. Subsequently, he worked with FRAME, Safer Medicines and as an independent scientific consultant to promote use of scientifically valid non-animal approaches for development of safe new medicines, plus human safety assessment of cosmetic ingredients. Dr Kenna has authored or co-authored >120 peer reviewed publications and book chapters, is a member of SOT and ISSX and is a Fellow of the British Toxicology Society.
Dr Katya (Tsaioun) HélinAkttyva Therapeutics, US
Dr Katya (Tsaioun) HélinAkttyva Therapeutics, US Dr Katya Hélin (formerly Tsaioun) is a life sciences entrepreneur and a founder of Akttyva Therapeutics. The focus of her career has been the translation of scientific innovations into therapies and technologies enabling improvements in human health. She spent two decades in pharmaceutical industry leading translational drug-discovery teams, subsequently founding Apredica, which became a leader in commercializing in vitro de-risking technologies for drug-discovery programs. After a successful acquisition of Apredica by Cyprotex, PLC (now Evotec), she served as a CSO and Board member in the merged company. Over the past decade Dr Hélin conducted academic research in toxicology at Johns Hopkins Bloomberg School of Public Health, where she led international multi-stakeholder efforts to establish evidence-based methodologies and practices in translational research. She also serves on advisory boards of companies, non-profits, and on scientific review committees at the NASEM, NIH, EFSA and private foundations. She earned her PhD in Human Nutrition Science from Tufts University and completed post-doctoral training in neurochemistry at Harvard Medical School.
Professor Michael ColemanAston University, UK
Professor Michael ColemanAston University, UK Professor Coleman graduated with first-class honours in Pharmacology and a PhD in Biochemical Pharmacology from Liverpool University. He spent two years as a National Research Council Resident Research Associate at Walter Reed Army Institute of Research, Washington DC, then, after completing a Wellcome Fellowship in Toxicology he joined Aston University. His research interests since 1991 have been focussed on developing human experimental models in toxicology and latterly neurotoxicology.
Professor Lorna HarriesExeter University, UK
Professor Lorna HarriesExeter University, UK Professor Lorna Harries is co-founder, director and Chief Scientific Officer at SENISCA Ltd, a spin out company from the University of Exeter Medical School, founded on the back of 10+ years of academic research from the Harries lab. Lorna gained her PhD from University College London in 1994 and holds a personal chair in Molecular Genetics at the University of Exeter Medical School. Lorna also heads the Exeter Animal Free Research Centre of Excellence (ARC 2.0) funded by Animal Free Research UK. The Harries lab have interests in -omics approaches to the study of ageing and age-related disease processes in humans, and her work takes a genes to systems approach, ranging from ‘big data’ analyses to detailed individual molecular analysis of particular genes. Her team were the first to report dysregulation of alternative splicing as a new, and druggable, hallmark of ageing; a finding which is now being developed as the basis for a new generation of interventions to treat the underpinning causes, not just the symptoms, of age-related chronic disease.
The Rt Hon George EusticeFormer Secretary of State for Environment, Food and Rural Affairs, UK
The Rt Hon George EusticeFormer Secretary of State for Environment, Food and Rural Affairs, UK George is a seasoned policy expert with nearly a decade of experience as a Minister in DEFRA, including three years in Cabinet. During his tenure, he championed groundbreaking legislation such as the Animal Welfare (Sentience) Act 2022, regulations mandating CCTV in slaughterhouses, and the Agriculture Act 2020, which established animal welfare as a public good. Since leaving Government, George has founded his consultancy business, Penbroath, which focuses on environmental policy. A passionate advocate for animal welfare, he continues to work towards policies that promote compassion and sustainability. |
| 16:30-16:50 |
Flash talks x 4
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| 16:50-17:00 |
Day 1 closing remarks
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK Geoff Pilkington is an Emeritus Professor of Neuro-oncology, having held chairs at King's College, London and the University of Portsmouth, where he was Director of the Brain Tumour Research Centre which exclusively used human cells, tissues and biomaterials in their research. He is a Past President of the British Neuro-oncology Society and Honorary Treasurer, Executive Board Member and Scientific Board Member of the European Association of Neuro-oncology as well as being a Fellow of the Royal College of Pathologists and a Fellow of the Royal Society of Biology. His published work was largely focussed around brain tumour invasion, the blood brain barrier, the tumour microenvironment, brain tumour modelling and repurposed drugs. |
| 17:00-18:15 |
Poster session
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Chair
Professor John Greenman
University of Hull, UK
Professor John Greenman
University of Hull, UK
John is a tumour immunologist at the University of Hull who has morphed into someone who spends most of his time developing lab-on-a-chip technology for personalized medicine against solid tumours. His interdisciplinary biomicrofluidics group, comprising clinicians, chemists and engineers, has been pioneering the development and adoption of bespoke microfluidic devices for almost 20 years. The group is equipped to design and fabricate devices from a range of materials depending on the desired end-use, recently moving more to using 3D-printed devices in biocompatible polymers. The group has extensive experience of maintaining different tumour types, in a viable state, including glioblastoma, head and neck squamous cell carcinoma, thyroid, renal and colorectal tissues. In addition, the group has undertaken extensive downstream analysis of effluents and tissue at the molecular (miRNA, mRNA, and circulating tumour DNA) and protein levels, allowing detailed analysis of clinically-relevant treatments of tissue on devices.
| 09:00-09:05 |
Opening remarks and Chair's introduction
Professor John GreenmanUniversity of Hull, UK
Professor John GreenmanUniversity of Hull, UK John is a tumour immunologist at the University of Hull who has morphed into someone who spends most of his time developing lab-on-a-chip technology for personalized medicine against solid tumours. His interdisciplinary biomicrofluidics group, comprising clinicians, chemists and engineers, has been pioneering the development and adoption of bespoke microfluidic devices for almost 20 years. The group is equipped to design and fabricate devices from a range of materials depending on the desired end-use, recently moving more to using 3D-printed devices in biocompatible polymers. The group has extensive experience of maintaining different tumour types, in a viable state, including glioblastoma, head and neck squamous cell carcinoma, thyroid, renal and colorectal tissues. In addition, the group has undertaken extensive downstream analysis of effluents and tissue at the molecular (miRNA, mRNA, and circulating tumour DNA) and protein levels, allowing detailed analysis of clinically-relevant treatments of tissue on devices. |
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| 09:05-09:25 |
Drug safety, pharmacogenomics and personalised medicine (online)
Adverse drug reactions (ADRs) remain a major, perhaps increasing, burden on healthcare systems. The burden often falls on the most vulnerable including the youngest and oldest (with multiple diseases) who are not usually included in clinical studies. Our latest data in the elderly with multimorbidity and polypharmacy indicates that 16.5% of admissions to our hospitals are due to ADRs, costing the NHS in England £2.21 billion per year. Although animal studies allow us to understand the pharmacokinetics of a drug in vivo, and potentially predict type A dose-dependent reactions, they are not usually designed to identify individual susceptibility factors. For example, it is not possible to undertake non-clinical studies that replicate the complexity encountered in a patient with multimorbidity and polypharmacy. This may however be possible by using modelling approaches but again limited because by the fact that few studies involve the elderly who are living with multiple long-term conditions. For type B (bizarre, unpredictable) reactions there are no good animal models to predict individual susceptibility. Because of the dearth of relevant studies during drug development, the identification of individual susceptibility to ADRs usually occurs during the post-marketing phase in real-world settings. Pharmacogenomics is the study of how genetic variation in individuals determines the response to a drug (both efficacy and safety). Pharmacogenomics represents one component of (a) factors that determine variability in drug responses; and (b) the overall field of personalized medicine. The presentation will discuss examples of how pharmacogenomics is being used in clinical practice to reduce ADRs and improve patient outcomes, and how this might be implemented in the future to benefit the whole population.
Sir Munir PirmohamedUniversity of Liverpool, UK
Sir Munir PirmohamedUniversity of Liverpool, UK Professor Sir Munir Pirmohamed (MB ChB, PhD, FRCPE, FRCP, FFPM. FRSB, FBPhS, FMedSci, DL) is David Weatherall Chair in Medicine at the University of Liverpool, NHS Chair of Pharmacogenetics, and a Consultant Physician at the Royal Liverpool University Hospital. He is Director of the Centre for Drug Safety Sciences, and Director of the Wolfson Centre for Personalised Medicine. He is also Director of HDR North. He also leads the Centre for Excellence in Regulatory Science in Pharmacogenomics (CERSI-PGx). He is an inaugural National Institute for Health and Care Research Senior Investigator, Fellow of the Academy of Medical Sciences in the UK, and Chair of the Commission Human Medicines. He is a medical trustee for the British Heart Foundation. He is Past-President of British Pharmacological Society and of the Association of Physicians. He was awarded a Knights Bachelor in the Queen’s Birthday Honours in 2015. His research focuses on personalised medicine, clinical pharmacology and drug safety in a variety of disease areas, including cardiovascular medicine. |
| 09:25-09:45 |
Cardiovascular diseases and drugs: Where are we with hiPSC models?
Derivation of cardiovascular cell types from human pluripotent stem cells (hPSCs) generated from patient tissues or introducing targeted mutations into healthy control hPSCs is an area of growing interest as a platform for disease modelling, drug discovery and (cardio)toxicity. Our lab has been investigating microtissue solutions in which cardiomyocytes and cardiac vascular and stromal cells are present. The presence of cardiac stromal cells promotes cardiomyocyte maturation. By combining cardiac microtissues with new methods for functional phenotyping, we have been able to quantify the outcomes of drug and disease mutation responses in situ in high throughput. The use of isogenic pairs of hiPSC lines with and without mutations has proven very important since variability between “healthy control” hiPSC lines is often greater than the difference between diseased cells and its isogenic control. Examples of studies investigating disease and drug responses in cardiac microtissues will be shown. These approaches have allowed us to identify which cell types in the heart are responsible for the disease phenotypes observed in vitro indicating which cells we might target for therapy. Moreover, we have been successful in automating the production of healthy and disease cardiac microtissues such that they can be used for high throughput drug screens. These complex cell systems are paving the way towards better understanding of disease mechanisms and drug discovery.
Professor Christine MummeryLeiden University Medical Center, The Netherlands
Professor Christine MummeryLeiden University Medical Center, The Netherlands Christine Mummery is Professor of Developmental Biology at Leiden University Medical Center. Following a PhD in Biophysics, she moved to the Hubrecht Institute to study ion channels and later, cardiovascular diseases using pluripotent stem cells from patients, developing organ-on-chip models. This has centred on safety pharmacology to predict toxic effects of drugs on the human heart and capturing cardiac and vascular disease phenotypes including identifying individual vulnerability and drug sensitivity. She was president of the International Society for Stem Cell Research (2020-2021) and co-founded the European Organ-on-Chip Society. She is also a member of the Royal Netherlands Academy of Science. In 2021, she was awarded the Lefoulon Delalande Prize jointly with Gordon Keller. She is currently on, or chairs, advisory boards of Sartorius AG (Germany), HeartBeatBio (Austria), Mogrify UK, Cellestic, Founding (now Associate) Editor of the ISSCR journal Stem Cell Reports, among others. |
| 09:45-10:00 |
Discussion
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| 10:00-10:20 |
Break
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| 10:20-10:40 |
3D cell culture models and their role in enhancing genotoxicity testing approaches for nano- and advanced-materials
Standard in vitro genotoxicity testing approaches have limitations for testing nanomaterials (NMs) and in 2013, an OECD expert panel concluded it was necessary to adapt the in vitro mammalian cell micronucleus test (OECD TG487) to facilitate evaluation of manufactured NMs. An OECD project was initiated, aimed at developing a new OECD Guidance Document detailing the necessary steps to adapt OECD TG487 for NMs, which was subsequently published in Sept 2022 (Series on Testing and Assessment No. 359; ENV/CBC/MONO(2022)15). Whilst ongoing efforts have focused on adapting existing OECD TGs for NMs testing, limitations remain as existing in vitro approaches lack physiological relevance, often do not consider long-term exposure effects, and do not cover all mechanisms of action underpinning genotoxicity. New approach methodologies (NAMs) provide the opportunity to overcome these issues and there have been substantial developments in this area over recent years. For example, co-culture models incorporating epithelial and macrophage cells can detect secondary genotoxicity, a key nanomaterial mode of action only detected in vivo. Additionally, 3D liver spheroids exhibit higher metabolic capacity, which is important for identifying pro-carcinogens. These models also support longer-term, repeated dosing, which is more representative of nanomaterial human exposure. The broad field of NAMs to better support regulatory decision making is therefore gaining momentum, with a variety of promising technologies emerging.
Professor Shareen H DoakMedicines and Healthcare products Regulatory Agency (MHRA), UK
Professor Shareen H DoakMedicines and Healthcare products Regulatory Agency (MHRA), UK Shareen Doak is a Deputy Director in Safety and Surveillance at the Medicines and Healthcare products Regulatory Agency (MHRA), leading the Benefit-Risk Evaluation I function. She is an Emeritus Professor of Genotoxicology and Cancer, and UK and EUROTOX Registered Toxicologist (UKRT, ERT). Prior to joining the MHRA, Shareen’s academic research experience focused on the genotoxic profiles of chemicals and advanced materials, mechanisms underlying their DNA damaging potential and subsequent consequences upon human health. Her interests extended to carcinogenicity, and development of advanced 3D culture models and mechanism-based bioassays for safety assessment to reduce the need for animal testing. Shareen has served as a member of the Committee on Carcinogenicity of Chemicals in Food, Consumer Products and the Environment (COC), the Scientific Advisory Group on Chemical Safety in Consumer Products (SAG-CS) for the Office for Product Safety and Standards (OPSS), and the Health & Safety Executive (HSE) Science Quality Assurance Group (SQAG). |
| 10:40-11:00 |
Which contexts of use arise from animal studies for new approach methods development? How does this change impact staff and infrastructure?
It is critical to understand the in vivo animal test (IAT) when developing and accelerating adoption of New Approach Methodologies (NAMs). The specialist animal staff understand the strengths and weaknesses of their IATs, and their knowledge and experience are needed when developing and implementing NAMs. Each IAT performed results in many outputs (eg liver weight changes, clinical signs, biomarkers, metabolites formed etc) that are used to decide whether there is an adverse event or not. Each output can be converted into a context of use (CoU), with adverse outcome pathways devised and used to create NAMs (eg in vitro or computational tests). Many NAMs, alone or as batteries, are required to replicate all animal tests endpoints. The same process can be followed to replicate the CoUs in human disease. Both IATs and human NAMs should predict the human disease to be of value in a risk assessment or submission in a regulatory package. How does this impact on the staff working on these tests and the infrastructure? Whilst NAMs are phased in, those staff working on the animal tests can be trained in cell husbandry, dosing, and sample collection. This is no different to when they are retrained to work with different species. The technologies and knowledge used by support services, eg pathology, immunology and bioanalysis, are similar for NAMs and minimum retraining is envisaged. When repurposing a vivarium to a tissue culture lab, smaller rooms are needed, but temperature and humidity control is essential. NAM development needs stakeholders with animal test knowledge to accurately develop CoUs for NAMs development.
Dr Clive RoperRoper Toxicology Consulting Limited, UK
Dr Clive RoperRoper Toxicology Consulting Limited, UK Clive graduated with a PhD in in vitro dermal absorption followed by a post-doctoral position at Newcastle University. In 1996, he joined CRL performing in vitro skin penetration studies, then promoted to Director, In Vitro Toxicology, leading the in vitro toxicology, genetox, safety pharmacology and advanced in vitro model’s teams. In 2021, Clive started a consulting business aiding clients in gaining adoption and regulatory acceptance of NAMs. Clive is the board chairperson for PeptiMatrix, a board member of the 3Rs Collaborative and NC3Rs, Vice President of ESTIV and Vice President Elect for the Dermal Toxicology Specialty Section of Society of Toxicology (SOT). He is member of the Roundtable of Toxicology Consultants, International MPS Society and SOT, a European Registered Toxicologist, and is a chartered biologist and chartered scientist and Fellow of Royal Society of Biology. |
| 11:00-11:15 |
Discussion
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| 11:15-12:15 |
Panel discussion: How would a transition to human-relevant methods make medicines safer?
Dr Stella CochraneUnilever SERS (Safety, Environmental and Regulatory Science), UK
Dr Stella CochraneUnilever SERS (Safety, Environmental and Regulatory Science), UK Regulatory Science Leader: Food Allergy and Immunology in Unilever’s Safety and Environmental Regulatory Science group (SERS). Dr Cochrane is an experienced science leader and risk assessor with over 20 years’ experience gained working in academia and industry. Dr Cochrane has expertise in immunotoxicology with a focus on protein allergy and provides scientific expertise and leadership, covering risk assessment and risk management of consumer products and ingredients. Dr Cochrane is also responsible for the development and application of new, non-animal, human-focused approaches to immunotoxicological risk assessment, including research into mechanisms of allergic sensitisation. Dr Cochrane is actively involved in allergy-related external, multi-partner, multi-national taskforces and research projects. Dr Cochrane is co-chair of Food Drink Europe’s Allergen Steering Group, chair of the UK Food and Drink Federation Allergen Steering Group and until recently was a member of UK Committee on Toxicity.
Professor Chris MolloyMedicines Discovery Catapult, UK
Professor Chris MolloyMedicines Discovery Catapult, UK Professor Chris Molloy was the founding CEO of Medicines Discovery Catapult, a national Life Sciences service, following an international career across a range of life sciences disciplines. Chris chairs the IP Advisory Committee for the Association of Medical Research Charities and the Industry Advisory Board for Manchester’s Biomedical Research Centre and Health Innovation Manchester. Chris is a Member and Trustee of the Institute of Cancer Research and holds an honorary chair at the University of Manchester.
Professor Helen MaddockInoCardia, UK
Professor Helen MaddockInoCardia, UK Professor Helen Maddock is the Founder, Director and Chief Scientific Officer at InoCardia Ltd, a spin out company from Coventry University, supported by 15+ years of academic research from the Maddock lab. Helen is Professor of Cardiovascular Physiology and Pharmacology at Coventry University and an Honorary Professor at Warwick Medical School, University of Warwick. She has over 30 years of experience spanning academia, life sciences, clinical sciences, and industry — including time with AstraZeneca and GSK early in her career. Helen completed a BBSRC industry funded PhD studentship supported by GSK and received her doctorate from the Welsh School of Pharmacy, Cardiff University. Followed by post doctoral research funded by the British Heart Foundation, at the world leading Hatter Cardiovascular Institute, University College London. During this time she pursued novel pharmacological and therapeutic strategies to protect the heart from the consequences of ischaemic and reperfusion-induced injury. She is known for her work in industry for driving forward improved and predictive human relevant preclinical microphysiological system (native, engineered cells and tissue) and computational methods for cardiac efficacy and safety assessment. She is passionate about the development and use of clinically relevant New Approach Methodologies (NAMs) to deliver predictive data that has human clinical concordance. Helen’s goal is to support the improvement and transformation of how we assess cardiovascular safety and efficacy during pre-clinical development focussed on human-relevant methods — with the aim of making drugs safer and effective. She is the recipient of multiple industry awards including an Innovate UK Women in Innovation Award, and a leading advocate for translational approaches that connect science with real-world impact.
Professor Christopher HughesUniversity of California, Irvine, US
Professor Christopher HughesUniversity of California, Irvine, US Professor Hughes is a Chancellor’s Professor and Associate Dean of Research and Innovation at UC Irvine. His research focuses on the development and growth of blood vessels, and he is a pioneer in the field of microphysiological systems (organ-on-chip), specializing in the creation of vascularized and perfused tissue-on-chip models. The technology is now licensed to Aracari Biosciences, for which Dr Hughes is a founder and CSO. He served for 10 years as chair of his department and previously served as Director of UCI’s Advanced Cardiovascular Technology Center, and as a Program Leader in the Cancer Center. He is an elected Fellow of the American Association for the Advancement of Science (AAAS), and works extensively with the non-profit organization, cureHHT, where he chairs their Scientific and Medical Advisory Council. In 2022 he received their Lifetime Achievement Award.
Professor Blanca RodriguezUniversity of Oxford, UK
Professor Blanca RodriguezUniversity of Oxford, UK Professor Blanca Rodriguez is Professor of Computational Medicine, Wellcome Trust Senior Research Fellow, and Head of the Computational Biology and Health Informatics Theme at the Department of Computer Science, University of Oxford. She aims to accelerate medical therapy development through augmenting clinical and experimental data with modelling and simulation, and machine learning. Her team has contributed pioneering case studies for the Digital Twin vision in precision medicine, and In Silico Trials for therapy testing. She actively collaborates with industry and leads the Computational Cardiovascular Science team at Oxford, an interdisciplinary, diverse and award-winning research team. Originally, she is from Valencia, Spain, where she trained in Engineering at the Universidad Politecnica de Valencia (MSc and PhD). She then worked as a postdoc at Tulane University in New Orleans, USA for two years. After this, she joined the University of Oxford, where she was awarded six competitive personal research fellowships and in 2014 the professorship in Computational Medicine.
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK Geoff Pilkington is an Emeritus Professor of Neuro-oncology, having held chairs at King's College, London and the University of Portsmouth, where he was Director of the Brain Tumour Research Centre which exclusively used human cells, tissues and biomaterials in their research. He is a Past President of the British Neuro-oncology Society and Honorary Treasurer, Executive Board Member and Scientific Board Member of the European Association of Neuro-oncology as well as being a Fellow of the Royal College of Pathologists and a Fellow of the Royal Society of Biology. His published work was largely focussed around brain tumour invasion, the blood brain barrier, the tumour microenvironment, brain tumour modelling and repurposed drugs. |
Chair
Dr Katya (Tsaioun) Hélin
Akttyva Therapeutics, US
Dr Katya (Tsaioun) Hélin
Akttyva Therapeutics, US
Dr Katya Hélin (formerly Tsaioun) is a life sciences entrepreneur and a founder of Akttyva Therapeutics. The focus of her career has been the translation of scientific innovations into therapies and technologies enabling improvements in human health. She spent two decades in pharmaceutical industry leading translational drug-discovery teams, subsequently founding Apredica, which became a leader in commercializing in vitro de-risking technologies for drug-discovery programs. After a successful acquisition of Apredica by Cyprotex, PLC (now Evotec), she served as a CSO and Board member in the merged company. Over the past decade Dr Hélin conducted academic research in toxicology at Johns Hopkins Bloomberg School of Public Health, where she led international multi-stakeholder efforts to establish evidence-based methodologies and practices in translational research. She also serves on advisory boards of companies, non-profits, and on scientific review committees at the NASEM, NIH, EFSA and private foundations. She earned her PhD in Human Nutrition Science from Tufts University and completed post-doctoral training in neurochemistry at Harvard Medical School.
| 13:15-13:20 |
Chair's introduction
Dr Katya (Tsaioun) HélinAkttyva Therapeutics, US
Dr Katya (Tsaioun) HélinAkttyva Therapeutics, US Dr Katya Hélin (formerly Tsaioun) is a life sciences entrepreneur and a founder of Akttyva Therapeutics. The focus of her career has been the translation of scientific innovations into therapies and technologies enabling improvements in human health. She spent two decades in pharmaceutical industry leading translational drug-discovery teams, subsequently founding Apredica, which became a leader in commercializing in vitro de-risking technologies for drug-discovery programs. After a successful acquisition of Apredica by Cyprotex, PLC (now Evotec), she served as a CSO and Board member in the merged company. Over the past decade Dr Hélin conducted academic research in toxicology at Johns Hopkins Bloomberg School of Public Health, where she led international multi-stakeholder efforts to establish evidence-based methodologies and practices in translational research. She also serves on advisory boards of companies, non-profits, and on scientific review committees at the NASEM, NIH, EFSA and private foundations. She earned her PhD in Human Nutrition Science from Tufts University and completed post-doctoral training in neurochemistry at Harvard Medical School. |
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| 13:20-13:40 |
Why we need social sciences to understand our behaviour and make faster progress together
As a newly graduated veterinarian in 1986, I aimed to improve animal welfare and scientific quality to enhance the translation of animal research to humans. At the time, I believed animal studies were necessary and evidence-based. Early in my career, I focused on implementing the 3Rs, particularly Reduction and Refinement. Over time, however, I realized that the 3Rs alone were insufficient. Systematic reviews proved more effective, as they directly supported 3R implementation by e.g. identifying unnecessary duplication. These reviews also revealed the poor quality of animal study publications and the lack of (predictors for) successful translation to humans. This led me to shift away from animal studies toward more human-relevant science, especially given advances in New Approach Methods (NAMs) such as organoids, AI, and better use of human-based data. Together with historians, I analysed animal testing requirements in legislation and found they were largely based on crisis-driven decisions rather than solid scientific evidence. The COVID-19 pandemic further demonstrated that development timelines for vaccines could be drastically shortened through increased use of NAMs, reduced animal testing, use of existing data, and earlier clinical trials. In the Netherlands, I joined the government-initiated Transition to Animal-Free Innovations program, applying transition /social sciences to accelerate this shift. This interdisciplinary approach brings together multiple disciplines in academia, industry, policymakers, and regulators. Supported by dedicated funding, I became co-chair of the SAFE consortium (Safety Assessment through animal-Free Evolution), which studies barriers to regulatory adoption of animal-free methods and how to accelerate progress toward more human-relevant science.
Professor Merel Ritskes-HoitingaUtrecht University, The Netherlands
Professor Merel Ritskes-HoitingaUtrecht University, The Netherlands Merel Ritskes-Hoitinga is Professor in Evidence-Based Transition to Animal-Free Innovations at Utrecht University. After her graduation as a veterinarian from Utrecht University in 1986, she has been committed to the goal of implementing the 3Rs, Replacement, Reduction and Refinement of animal studies. Systematic reviews turned out to be a valuable methodology to implement the 3Rs. Unfortunately, the evidence from systematic reviews demonstrates the generally low quality of publications and low translational value of animal studies for humans. This scientific fact indicates clearly that we need to find more human-relevant research. Not only is it important to develop new non-animal alternatives, ie more human-relevant methodologies, but also on how these will become generally accepted and taken up in legislation. This has implied taking steps to broadening her research and education, also teaming up with social sciences and multiple stakeholder groups. |
| 13:40-14:00 |
How much top-down policy in transitions beyond animal experimentation?
In recent years, several national governments, the European Commission, and several US government agencies have made efforts to strategize for a transition beyond animal experimentation. In debates about such strategies, the role of top-down policies is controversial. While animal advocates have called on governments to commit to specific reduction goals, or even to put an expiry date on some or all forms of animal experimentation, groups representing science and industry have warned that innovation cannot be planned and government should not try to steer research top-down. Synthesizing arguments on either side of this debate, this talk will suggest that policy mixes combining top-down and bottom-up approaches – drawing on frameworks such as Transformative Governance, Transition Management, and Mission-Oriented Research and Innovation – can retain most of what transition strategy advocates seek while avoiding most of the risks highlighted by their opponents.
Dr Nico MüllerUniversity of Basel, Switzerland
Dr Nico MüllerUniversity of Basel, Switzerland Dr Nico Müller is a philosopher based at the University of Basel. He is a Principal Investigator within the National Research Programme 79 «Advancing 3R» and leads a research project about the ethics of phase-out planning for animal experimentation. His work focuses on animal ethics, the philosophy of science, and science policy. His articles have appeared in journals such as ALTEX, Ethical Theory and Moral Practice, and Science and Engineering Ethics, addressing topics such as the limitations of the 3Rs framework, the applicability of transition-science approaches to animal experimentation, and the ethical questions that arise when seeking to advance the phase-out of animal experimentation. |
| 14:00-14:20 |
Ksilink – a patient-based phenotypic drug discovery engine
Late-stage clinical trial failures in drug development are often driven by poor efficacy, largely due to inappropriate animal models that fail to capture human disease complexity. Ksilink addresses this challenge through a patient-based phenotypic drug discovery engine, leveraging human cellular systems derived from patients for high-throughput screening. By automating complex human disease models through AI-driven image analysis, this platform enables physiologically relevant insights without relying on animal models. It supports screening of chemical and genetic perturbators, target deconvolution, and mechanism-of-action profiling directly in human cells. This approach not only accelerates discovery but also improves translational predictability, reducing ethical concerns and enhancing the likelihood of clinical success.
Dr Mona BoyéKsilink, France
Dr Mona BoyéKsilink, France Mona Boyé holds a PhD in molecular biology from Cardiff University. Shortly after her postdoc, she transitioned into technology transfer and bioscience innovation. She is currently Chief Business Officer at Ksilink, a French Tech company specializing in patient-based phenotypic drug discovery. |
| 14:20-14:30 |
Discussion
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| 14:30-14:45 |
Break
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| 14:45-15:15 |
Modernization at the US FDA
Learn about key modernisation initiatives and top priorities for 2026 at the new FDA.
Dr Martin MakaryFood and Drug Administration, US
Dr Martin MakaryFood and Drug Administration, US Prior to joining the FDA, Dr Makary worked at Johns Hopkins University School of Medicine, where he was a tenured professor, surgical oncologist, and Chief of Islet Transplant Surgery. He has led cross-disciplinary research on a range of subjects, including cancer care, obesity, frailty and psychologic reserve in older patients, adverse event monitoring, the Orphan Drug Act, antimicrobial resistance, and Alzheimer’s. Of particular note, he is the co-developer of the Surgery Checklist used in many operating rooms around the world today. He was the first to perform several novel surgical operations, including the first-in-the-world series of laparoscopic pancreas islet transplant operations. He is the author of three bestselling books, has published more than 300 scientific peer-reviewed articles, and in 2018 was elected to the National Academy of Medicine. |
| 15:15-15:35 |
The role of biological computational models in human-oriented medical research
My research has focussed on using experimental data from animal and human research to develop mathematical models of the heart, starting in 1960 with the first successful model explaining how heart rhythm arises from interactions between the cell membrane potential and the proteins carrying ionic currents. Those models, now including models specifically based on human cardiac cells, have been used extensively in the pharmaceutical industry to test for possible arrhythmic side-effects. They may also be used to clarify the difference between association scores in genome-wide association studies and physiological causation. In the case of the heart this can be as large as 85% causation with only a 10-15% association score (Noble, 2025). This difference is attributable to the presence of robust back-up processes, and would explain why the great majority of association scores are too small to be useful in predicting disease states. That fact is confirmed by the demonstration that the polygenic scores do not reliably predict either cancer or cardiovascular disease (Hingorani et al 2023). The outcome is what I call a gene-centric impasse: the failure of genomics to result in successful diagnoses and cures. I will conclude by showing how physiological modelling of causation may solve the gene-centric impasse. Hingorani, A.D., Gratton, J., Finan, C., et al 2023. BMJMedicine. 2:e000554. doi:10.1136/ bmjmed-2023-000554
Professor Denis Noble CBE FMedSci FRSUniversity of Oxford, UK
Professor Denis Noble CBE FMedSci FRSUniversity of Oxford, UK Denis Noble is Emeritus Professor of Cardiovascular Physiology based at the Department of Physiology, Anatomy & Genetics at Oxford University. He is author of many books, including his latest ones: The Pacemaker Channels of the Heart (World Scientific 2025), and Understanding Living Systems (CUP 2023). The work for which he is most well-known is his demonstration that the origin of the heartbeat lies in its ability to excite itself spontaneously through the interaction of cellular electrical potential and the ionic channels responsible for generating ionic currents. During the last 20 years he has also published widely on evolutionary biology: https://www.denisnoble.com/evolution-publications/. |
| 15:35-15:45 |
Discussion
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| 15:45-16:45 |
Panel discussion: How can we drive a transition to human-relevant science in academic research as well as preclinical testing?
Professor Merel Ritskes-HoitingaUtrecht University, The Netherlands
Professor Merel Ritskes-HoitingaUtrecht University, The Netherlands Merel Ritskes-Hoitinga is Professor in Evidence-Based Transition to Animal-Free Innovations at Utrecht University. After her graduation as a veterinarian from Utrecht University in 1986, she has been committed to the goal of implementing the 3Rs, Replacement, Reduction and Refinement of animal studies. Systematic reviews turned out to be a valuable methodology to implement the 3Rs. Unfortunately, the evidence from systematic reviews demonstrates the generally low quality of publications and low translational value of animal studies for humans. This scientific fact indicates clearly that we need to find more human-relevant research. Not only is it important to develop new non-animal alternatives, ie more human-relevant methodologies, but also on how these will become generally accepted and taken up in legislation. This has implied taking steps to broadening her research and education, also teaming up with social sciences and multiple stakeholder groups.
Dr J Malcolm WilkinsonSheffield University, UK
Dr J Malcolm WilkinsonSheffield University, UK Dr Wilkinson studied Physics at Oxford University and then worked in industry for 16 years developing microelectronics. In 1994 he started a consultancy in Cambridge supporting the development of microfluidics devices for medical diagnostics. From 2006 to 2019 he was CEO of Kirkstall Ltd, one of the world’s leading Organ on a Chip companies. Although now retired, he continues as a Director of Kirkstall Ltd., and is on the Scientific Advisory Panel of Animal Free Research UK supporting their work to replace the use of animals in medical research and drug testing. He has been a visiting Lecturer for FSRM, Neuchatel, Switzerland, on the subject of Micro and Nanotechnology in Biomedical Engineering for over ten years and was appointed as a Visiting Professor in the Department of Biomedical Engineering at Sheffield University in 2019. Dr Wilkinson is co-author on several papers on in-vitro models of toxicity and a contributing editor of a recently published book on In-Vitro Testing. He has recently published a review article on the status of Organ on a Chip technology.
Professor Paul L CarmichaelUnilever, UK
Professor Paul L CarmichaelUnilever, UK Professor Dr Paul L Carmichael has worked at Unilever in the UK since 2004, where he is responsible for the global development and implementation of novel non-animal-based approaches for assuring human and environmental health. He has over 45 years’ experience in toxicology and cancer research, largely in the academic arena, and prior to Unilever, he was a Senior Lecturer at Imperial College London in the Faculty of Medicine. A graduate of Surrey University with a BSc in Toxicology and a PhD from King’s College London, he was a postdoctoral scientist at the Institute of Cancer Research for seven years, exploring mechanisms of chemical genotoxicity and carcinogenicity. He currently has close academic links with Peking and Shenyang Medical Universities in China and is a past Assistant Professor of Pathology and Laboratory Medicine at Brown University in the USA. He has been an Endowed Professor at Wageningen University, Division of Toxicology, since March 2020 and is now Interim Director of the NAMs Qualification & Validation Unit of the Centre for Proefdiervrij Biomedical Translation at Utrecht University in the Netherlands. He has published over 150 research papers in peer-reviewed scientific journals and serves on many external committees in the UK, US, EU and China. His passion is the advancement of new safety assessment approaches using the inspiration of ‘Toxicity Testing in the 21st Century’ – this can be termed ‘next generation risk assessments’ or NGRA.
Dr Nico MüllerUniversity of Basel, Switzerland
Dr Nico MüllerUniversity of Basel, Switzerland Dr Nico Müller is a philosopher based at the University of Basel. He is a Principal Investigator within the National Research Programme 79 «Advancing 3R» and leads a research project about the ethics of phase-out planning for animal experimentation. His work focuses on animal ethics, the philosophy of science, and science policy. His articles have appeared in journals such as ALTEX, Ethical Theory and Moral Practice, and Science and Engineering Ethics, addressing topics such as the limitations of the 3Rs framework, the applicability of transition-science approaches to animal experimentation, and the ethical questions that arise when seeking to advance the phase-out of animal experimentation.
Dr Mona BoyéKsilink, France
Dr Mona BoyéKsilink, France Mona Boyé holds a PhD in molecular biology from Cardiff University. Shortly after her postdoc, she transitioned into technology transfer and bioscience innovation. She is currently Chief Business Officer at Ksilink, a French Tech company specializing in patient-based phenotypic drug discovery.
Steve RaceLabour MP for Exeter, UK
Steve RaceLabour MP for Exeter, UK Steve Race is the Labour MP for Exeter, and a Parliamentary Private Secretary in the Department of Health and Social Care. He has previously been a member of the Science, Innovation and Technology Select Committee, Chair of the APPG for Pharmacy, Chair of the APPG for ClimateTech, and Co-Chair of the APPG on Nutrition for Development. Steve has had a career in business, providing consultancy support in communications and policy to companies including Aston Martin, British Airways, HSBC, National Grid, and Nissan. He also spent three years on the UK Board of McKesson UK, owner of LloydsPharmacy and AAH, the largest medicines distributer in the country, leading a team of eight people covering crisis, policy, and government communications. |
| 16:45-17:00 |
Reflections, next steps and concluding remarks from co-organisers
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK
Professor Geoffrey J. PilkingtonUniversity of Portsmouth, UK Geoff Pilkington is an Emeritus Professor of Neuro-oncology, having held chairs at King's College, London and the University of Portsmouth, where he was Director of the Brain Tumour Research Centre which exclusively used human cells, tissues and biomaterials in their research. He is a Past President of the British Neuro-oncology Society and Honorary Treasurer, Executive Board Member and Scientific Board Member of the European Association of Neuro-oncology as well as being a Fellow of the Royal College of Pathologists and a Fellow of the Royal Society of Biology. His published work was largely focussed around brain tumour invasion, the blood brain barrier, the tumour microenvironment, brain tumour modelling and repurposed drugs.
Ms Rebecca RamSafer Medicines Trust, UK
Ms Rebecca RamSafer Medicines Trust, UK Rebecca Ram is an independent scientific consultant to the Safer Medicines Trust, working in the field of human-relevant science for almost two decades. Prior to this, she worked for a decade as a clinical data manager and programmer within phase 1-IV clinical trials for pharmaceutical and clinical research organisations(CROs). Rebecca has a Masters (MSc) in Toxicology (with Bioinformatics) and a BSc in Applied Biology. Her particular interest is in the field of clinical and scientific research and policy, including development and regulatory acceptance of advanced in-vitro and computational methods, improved use of clinical data and 'big data' analysis, as well as transition to human relevant approaches in fundamental research and disease modelling. Rebecca has written or co-authored occasional research papers and has presented at webinars, universities and colleges. She has also served on review committees for conference abstracts, publications and early career research awards.
Mrs Kathy ArchibaldSafer Medicines Trust, UK
Mrs Kathy ArchibaldSafer Medicines Trust, UK Kathy Archibald is the chair of Safer Medicines Trust. She graduated in genetics from Nottingham University and went on to work for Searle Pharmaceuticals and Genetics International (which became MediSense before acquisition by Abbott Laboratories). She then spent a decade working in conservation, including as a teacher-naturalist for the RSPB, followed by educational fundraising in schools for Action for Children. She founded Safer Medicines Trust in 2005, to confront the poor relevance of much pharmaceutical research (based on animals) to human medicine and its serious consequences for patient safety. The Trust has held conferences at the Royal Society and the House of Lords, initiated the Safety of Medicines Bill and published many peer-reviewed papers, book chapters and a popular science book, Rat Trap. Safer Medicines Trust was a founder member of the Alliance for Human Relevant Science, which helped establish the All-Party Parliamentary Group on Human Relevant Science in 2020. |