Preclinical Rethinking Webinar

In the last session before the summer break, Prof. Thomas Hartung (Johns Hopkins University, USA; Center for Alternatives to Animal Testing (CAAT)) will share his insights into "Organoid intelligence", an emerging multidisciplinary field working to develop biological computing using 3D cultures of human brain cells (brain organoids) and brain-machine interface technologies.

About the Talk:

The presentation introduces the new field of organoid intelligence, where human brain organoids are combined with advances in artificial intelligence, bioengineering, and computing. It explores how living neural systems may offer new models for studying cognition, disease, and learning, while raising important scientific, ethical, and societal questions about the future of biological intelligence and AI.

Smirnova, Lena et al. Organoid intelligence (OI): the new frontier in biocomputing and intelligence-in-a-dish. Frontiers in Science Vol 1 (2023): 2813-6330. https://doi.org/10.3389/fsci.2023.1017235

About the Speaker:

Thomas Hartung, MD PhD, is the Doerenkamp-Zbinden-Chair for Evidence-based Toxicology in the Department of Environmental Health and Engineering at Johns Hopkins Bloomberg School of Public Health and the Whiting School of Engineering, Baltimore. He also holds a joint appointment for Molecular Microbiology and Immunology at the Bloomberg School and as faculty in the Cellular and Molecular Medicine program in the Johns Hopkins School of Medicine. He is adjunct affiliate professor at Georgetown University, Washington D.C.. In addition, he holds a joint appointment as Professor for Pharmacology and Toxicology at University of Konstanz, Germany. He is faculty affiliate of the Johns Hopkins Data Science and AI Institute (DSAI), of the Wendy Klag Center for Autism and Developmental Disabilities (WKC) and the Health Innovation Translation Council. He also is Director of Centers for Alternatives to Animal Testing (CAAT, http://caat.jhsph.edu) of both universities. CAAT hosts the secretariat of the Evidence-based Toxicology Collaboration (http://www.ebtox.org) and manages collaborative programs on Good Read-Across Practice, Good Cell Culture Practice, Green Toxicology, Developmental Neurotoxicity, Developmental Immunotoxicity, Microphysiological Systems and Refinement. As PI, he headed the Human Toxome project funded as an NIH Transformative Research Grant and the series of annual Microphysiological Systems World Summits starting in 2022 by 60+ organizations. He has initiated a Human Exposome Moonshot Forum in Washington in May 2025, which has been joined by 60+ organizations so far. He is the founding Field Chief Editor of Frontiers in Artificial Intelligence, which published 1700+ articles since 2018. He is the former Head of the European Commission’s Center for the Validation of Alternative Methods (ECVAM), Ispra, Italy, and has authored more than 770 scientific publications with more than 58,000 citations (h-index 131). His toxicology classes on COURSERA had more than 23,000 active learners.

Join this session focused on Refinement (3Rs: Replace, Reduce, Refine). Dr. Paulin Jirkof (3Rs Coordinator, University of Zurich, Switzerland) will give an overview of different refinement strategies and will talk more in detail about the Micropipette-Guided Drug Administration (MDA) method, a novel approach to conventional oral gavage.

About the Talk:

Refinement of animal experiments is a key principle of the 3Rs, aiming to reduce animal burden, improve welfare, and enhance data quality. In rodent research, oral gavage is widely used for compound administration but can induce stress, carries a risk of injury, and in some cases poorly models voluntary intake in humans. This presentation evaluates voluntary oral administration methods, with a focus on Micropipette-Guided Drug Administration (MDA) as a refined alternative. For the MDA, mice are trained to voluntarily ingest experimental compounds mixed into palatable solutions delivered via a micropipette.

Studies show that mice can be reliably trained to voluntarily ingest a range of compounds, including tamoxifen and analgesics, although sex- and strain-dependent differences and challenges with poorly palatable substances have been observed. Most behavioural and metabolic measures appear unaffected by MDA, though some may be influenced and should be considered during experimental design.

Overall, MDA offers a refined alternative to oral gavage that better reflects voluntary intake and has the potential to improve both ethical standards and translational relevance. This talk presents successful use cases of MDA alongside its limitations, and provides practical training tips and troubleshooting guidance.

About the Speaker:

Paulin Jirkof earned her PhD in Neuroscience from the University of Zurich, Switzerland, and holds a Master of Advanced Studies in Management, Technology, and Economics from ETH Zurich, Switzerland. She obtained her venia legendi from the medical faculty of the University of Zurich in 2020. Her research focuses on refinement measures for laboratory rodents, with a particular emphasis on assessing animal welfare of mice. Currently serving as the 3R coordinator in the Office for Animal Welfare and 3R at the University of Zurich, Paulin is a member the executive board of the Swiss 3R Competence Center and chairs the Swiss Culture of Care Network. Additionally, she is a board member of the Swiss Society for Laboratory Animal Science and serves as the Editor-in-Chief of the journal Laboratory Animals.

Scarborough, Joseph et al. Preclinical validation of the micropipette-guided drug administration (MDA) method in the maternal immune activation model of neurodevelopmental disorders. Brain, behavior, and immunity vol. 88 (2020): 461-470. https://doi.org/10.1016/j.bbi.2020.04.015

In this session, Prof. Dr. med. Stephan Rosshart (Universitätsklinikum Erlangen, Germany) will present the concept of Wildlings: Laboratory mice engrafted with wild mouse-derived microbiota that display a more mature immune system sharing similar characteristics to those of adult humans.

About the Talk:

Mouse models are indispensable for biomedical research, yet two major limitations continue to impede global scientific progress: (I) a reproducibility crisis, primarily driven by divergent, institution-specific, low-resilience microbiota, and (II) a translatability crisis, as findings regularly fail to predict human outcomes.

Microbiome research offers a compelling route to address both challenges. While interspecies genetic differences have long been emphasized as the underlying reason, paradigm-shifting work by us and others shows that conventional laboratory specific pathogen-free (SPF) mice are also excessively removed from natural microbial and pathogen exposure. Because mammalian immunity evolved in a microbe-rich environment, sanitized housing conditions distort immune development and function, fostering misleading inferences about human physiology.

To mitigate these issues, natural microbiota-based mouse models, including wildlings, were established. Wildlings more closely reflect the human metaorganism by combining co-evolved microbes and pathogen exposure with the controlled genetics of laboratory strains (Rosshart et al., 2017; Rosshart et al., 2019). Notably, in settings where rodent and even non-human primate models failed to anticipate harmful clinical outcomes – such as the CD28 superagonist trial and anti-TNF strategies in sepsis – wildlings correctly recapitulated key human-like immune responses and, in principle, could have prevented these catastrophically failed clinical trials (Rosshart et al., 2019; Suntharalingam et al., 2006; Fisher et al., 1996). Moreover, wildling microbiota show multi-generational stability, cross-site robustness, and resilience to environmental challenges, supporting long-term, reproducible experimentation (Bruno et al., 2025; Runge et al., 2025).

Collectively, natural microbiota-based mouse models may enable the discovery of novel therapeutics that are unlikely to emerge from conventional SPF mice, improve the reproducibility of biomedical research, strengthen the safety and success of bench-to-bedside efforts, reduce attrition and costs, and support more ethical animal research aligned with the 3R principles, ultimately accelerating the development of new treatments for the benefit of human health.

About the Speaker:

Stephan P. Rosshart studied biology and medicine at the Albert-Ludwigs-University of Freiburg, Germany. In 2010, he received his M.D. with summa cum laude from the University of Freiburg. Following postgraduate training in internal medicine at the Medical Center – University of Freiburg, he joined the National Institutes of Health (NIH) in Bethesda, USA, for a six-year postdoctoral fellowship in basic immunology. During this period, he conceived the “natural microbiota theory” and created natural microbiota-based mouse models, including wildlings, to improve the reproducibility as well as the predictive value of basic and preclinical research for human diseases.

In 2019, he became an Emmy Noether Group Leader, founding the Translational Microbiome Research Laboratory and serving as Head of the Gnotobiotic Mouse Facility at the Department of Medicine II, Medical Center – University of Freiburg. In 2023, he was appointed Full Professor and Head of the Department of Microbiome Research, University Hospital Erlangen, Friedrich-Alexander-Universität Erlangen-Nürnberg (FAU), Erlangen, Germany.

His research focuses on mammalian microbiota and their roles in health and disease. Leveraging natural microbiota-based mouse models such as wildlings and a bench-to-bedside strategy, his work aims to (I) elucidate disease-relevant mechanisms and identify novel therapeutic opportunities for a wide range of human diseases of global relevance, including but not limited to transplant rejection, graft-versus-host disease, cancer, infectious diseases and vaccines, allergies, autoimmune and inflammatory disorders, neurological diseases, and cardiovascular disease; (II) enhance the reproducibility and modelling of human immune responses in basic and preclinical studies; and (III) support drug development in close collaboration with the pharmaceutical industry.

Runge, S., von Zedtwitz, S., Maucher, A.M. et al. Laboratory mice engrafted with natural gut microbiota possess a wildling-like phenotype. Nat Commun 16, 5301 (2025). https://doi.org/10.1038/s41467-025-60554-2

Join us for another round of Preclinical Rethinking, kicking it off with Dr. Maria Economou (Berlin Institute of Health at Charité – Universitätsmedizin Berlin, Germany) and Apl.-Prof. Dr. phil. nat. Julia Steitz (RWTH Universitätsklinikum Aachen, Germany) who will present Cancer-SOLES.

About the Talk: 

Cancer is a leading cause of death worldwide and new therapies are needed, necessitating testing in animal models. Although a wide variety of tumor models have been described in the literature, their limitations, as well as unsuccessful translation attempts, are often not reported. Yet, choosing the right animal model for the drug to be tested and successfully translated into the clinic is critical.   

We systematically reviewed studies testing targeted therapies with the goal of finding animal models which have been successfully tested in cancer research and to identify variables that contribute to successful translation and markers of high external validity. Given the substantial number of studies potentially relevant to this topic, we developed and validated an automated approach to categorize and prioritize studies prior to in-depth review of disease domains with potential for greatest impact. We established the approach with a manageable number of publications, to demonstrate the feasibility of extension to other tumor entities. Results were implemented in a systematic online living evidence summary (SOLES), an interrogatable database accessed through a user-friendly online dashboard. The dashboard can be used to filter studies based on study design features of interest, supporting the conduct of specific systematic reviews and highlight gaps in the literature for future research.  The Cancer-SOLES platform provides a novel opportunity to identify drug and model combinations that show concordance with human outcomes, optimize the model selection process for preclinical cancer studies and to reduce the number of animals used in cancer research.  

Hair K, Wilson E, Wong C, Tsang A, Macleod M, Bannach-Brown A. Systematic online living evidence summaries: emerging tools to accelerate evidence synthesis. Clin Sci (Lond). 2023 May 31;137(10):773-784. https://doi.org/10.1042/CS20220494  
 

About the Speakers:

Maria Economou is a postdoctoral researcher at the BIH QUEST Center for Responsible Research in Berlin. She has a background in biomedical sciences and neuroimaging and received her PhD in the field of cognitive neuroscience. Since 2023 she has been working in meta-research, focusing on systematic review methodology in the preclinical field, as well as the development and implementation of workflows for evidence synthesis at scale. 

Julia Steitz is an adjunct professor at the Institute for Laboratory Animal Science of the Medical Faculty, RWTH Aachen University, Germany. She studied Biotechnology (Dipl. Engineer) and received her PhD in Biochemistry from the Johann Wolfgang Goethe-University of Frankfurt, Germany, working in the field of melanoma research at the Johannes-Gutenberg University in Mainz. Following postgraduate training at the Medical Faculty of the Rheinischen Friedrich-Wilhelms University Bonn, Germany, she habilitated 2006 in the field of experimental dermatology. In 2005, she joined the Department of Surgery at the School of Medicine at the University of Pittsburgh, USA for an almost five-year postdoctoral fellowship in vaccine development. With the experience of working with various animal models in the cancer and vaccine development field she joined 2010 the Institute for Laboratory Animal Science at the RWTH Aachen University, Germany, serving as the director of the service laboratories for hematology, clin. Chemistry and microbiology and established her own cancer research group. Her research focusses on the establishment of tumor models for preclinical testing of new cancer drugs and the application of the 3R principle. In addition, as a lecturer, tutor and course organizer she is involved in the establishment and accreditation of FELASA Function A & D training courses and co-directed the Master program for Laboratory Animal Science (2015-2025) at the RWTH Aachen. Since 2025 she has also been serving as an external expert for the Education and Training Board of the FELASA and more recently of the GV-SOLAS. 

Mirzaei Y, Hüffel M, McCann S, Bannach-Brown A, Tolba RH, Steitz J. Animal models in preclinical metastatic breast cancer immunotherapy research: A systematic review and meta-analysis of efficacy outcomes. PLoS One. 2025 May 7;20(5):e0322876. doi: 10.1371/journal.pone.0322876.

Villwock S, Mirzaei Y, Dahl E, Steitz J. Preclinical pancreatic cancer mouse models for treatment with small molecule inhibitors: a systematic review and meta-analysis. Sci Rep. 2025 Oct 27;15(1):37358. doi: 10.1038/s41598-025-25191-1. 

In this session, Dr. Marcus Meinhardt will share his insights on conducting multi-center preclinical trials, a novel approach in the drug discovery pipeline to enhance translation.   

Meinhardt, Marcus W et al. "A new module in the drug development process: preclinical multi-center randomized controlled trial of R-ketamine on alcohol relapse." Neuropsychopharmacol. 50, 886–894 (2025). DOI: 10.1038/s41386-025-02071-w 

About the Speaker:

Dr. Marcus Meinhardt is the head of the research group Translational Psychopharmacology, bridging preclinical research (Institute for Psychopharmacology) to clinical work (Department of Molecular Neuroimaging) at the Central Institute of Mental Health in Mannheim. His background is in biotechnology and neuroscience with particular expertise in the field of preclinical behavioral neuroscience, molecular biology, and neuroimaging research methods. Additionally, he has intensive pharmaceutical industry experience, including preclinical target development, CNS pharmacokinetics and quality management. The core vision of his research is to promote preclinical research data quality and integrity in order to develop novel therapeutic approaches in the field of substance disorders (SUD), ultimately providing go/no-go decisions for clinical development.

Join us for the first session of 2026, with a focus on research reproducibility. Our expert speaker Dr. Michèle Nuijten (Tilburg University, Netherlands) will present the R package and web app statcheck to automatically detect statistical reporting inconsistencies in primary studies and meta-analyses. 

About the Speaker:

Michèle Nuijten is an Assistant Professor at the Meta-Research Center at Tilburg University, where she studies reproducibility and replicability in psychology. She obtained her PhD in Methodology and Statistics at Tilburg University in 2018. 
Michèle received an innovation grant from the Dutch Research Council to develop an efficient protocol to assess and improve robustness of psychological findings.
Furthermore, as part of her research, Michèle co-developed the free tool statcheck; a “spellchecker” for statistics. Statcheck has gained popularity as a pre-publication check: since its launch in 2016, the web app was visited tens of thousands times, and two psychology journals have made statcheck a standard element in their peer review process.
Beside her research, Michèle serves on the Editorial Boards of several psychology and meta-science journals. She is on the advisory boards of several initiatives related to improving robustness in psychology and other scientific fields, such as the Institute for Replication, the ERROR program, and the Dutch Reproducibility Network. Throughout all her work, she is an advocate for open science an robust research methods.

About the Talk:

Half of the psychology papers contain inconsistent statistical results in which the reported p-value does not match the reported test statistic and degrees of freedom. Most of these inconsistencies seem to be small and inconsequential, but in over 12.5% of the papers there are inconsistencies that might change the statistical conclusion. Such statistical reporting inconsistencies affect the reproducibility and quality of scientific findings.
We developed the R package "statcheck" and the accompanying web app https://statcheck.io to automatically find these inconsistencies. In my talk, I will briefly explain how statcheck works and discuss its potential in preventing statistical errors through self-checks and peer review. Finally, I will highlight some additional manual consistency checks that require only the information in the published article.

Nuijten, Michèle B, and Joshua R Polanin. ""statcheck": Automatically detect statistical reporting inconsistencies to increase reproducibility of meta-analyses." Research synthesis methods vol. 11,5 (2020): 574-579. DOI: 10.1002/jrsm.1408  

In this session, PD Dr. rer. nat. Steven R. Talbot of Medizinische Hochschule Hannover (Germany) will speak about the RELSA algorithm and its implementation in preclinical severity assessment of laboratory animals. The talk will focus on examples of where and how to implement the algorithm in animal research and routine monitoring. We will explore the idea of continuous quantitative severity assessment to more objectively identify animals at risk. Further, different applications such as forecasting and severity mapping will be discussed. The talk will illustrate how quantitative severity assessment informs research protocols and animal care practices, helping stakeholders make better informed decisions, supporting regulatory compliance while improving welfare outcomes and data quality.

About the Speaker:

Dr. Talbot is a biochemist, data scientist, and group leader (Preclinical Data Science) at the Institute of Laboratory Animal Science at Hannover Medical School (MHH), where he specialises in the intersection of artificial intelligence, statistics, and translational laboratory animal medicine. His research centres on developing data‑driven decision‑support tools that advance both scientific rigor and animal welfare in preclinical research. A significant contribution has been the development of the RELSA (Relative Severity Assessment) score, a quantitative framework for burden assessment in animal experiments that has also been successfully translated to pediatric clinical care as the Patient Vital Score (PVS). This work exemplifies his commitment to translational research that bridges veterinary and human medicine. As co‑founder of the GMDS “AI in Research” working group and an active member of the research groups R2N and FOR2591 – Severity Assessment in Animal‑Based Research, he continues to shape the integration of data science methods and artificial intelligence in biomedical research, working toward intelligent systems that enhance both scientific discovery and ethical standards in preclinical studies.

Talbot, Steven R et al. "RELSA - A multidimensional procedure for the comparative assessment of well-being and the quantitative determination of severity in experimental procedures." Frontiers in veterinary science vol. 9 937711. (2022) DOI:  10.3389/fvets.2022.937711 

Ohland PLS, Jack T, Mast M, Melk A, Bleich A, Talbot SR. Continuous monitoring of physiological data using the patient vital status fusion score in septic critical care patients. Sci Rep. 2024 Mar 26;14(1):7198. doi: 10.1038/s41598-024-57712-9. PMID: 38531955; PMCID: PMC10965972.

In this session, Dr. Natasha Karp (Data Sciences & Quantitative Biology, Discovery Sciences, R&D, AstraZeneca, Cambridge, UK) will present the interactive Sex Inclusive Research Framework (SIRF) that supports the evaluation of in vivo and ex vivo research proposals to address the risk of sex bias in preclinical research.

About the Talk:

A culturally embedded practice of studying only one sex in health research introduces a bias into the scientific knowledge base and contributes to poor translation and irreproducible findings. It is also inefficient and unsustainable, resulting in an estimated overproduction of >25 million mice and rats globally.   

Research has shown that scientists perceive sex as an important biological variable but believe sex-inclusive research represents an insurmountably difficult challenge. Despite this, funders and regulators are driving change in the research landscape by moving from encouragement to mandates, with key bodies now requiring a justification for exclusion.

Unfortunately, many of the perceived obstacles are rooted in misconceptions that permeate the entire research hierarchy. Frequently, these obstacles are rooted in statistical misconceptions and lead researchers to believe that inclusive designs will increase the number of animals needed and would thus be unethical and prohibitively expensive. Moving the dial to improve inclusive research practice requires a systematic approach to investigate and challenge these obstacles.

Here we present the Sex Inclusive Research Framework (SIRF) which supports the evaluation of in vivo and ex vivo research proposals from a sex inclusive research perspective by delivering a traffic light classification, indicating whether a proposal is appropriate, risky, or insufficient with regards to sex inclusion. This tool is designed for use by researchers, (animal) ethical review boards, and funders to generate a rigorous and reproducible assessment of sex inclusion at the proposal level, thus helping address the embedded sex bias in preclinical research.  

Karp, Natasha et al. "The Sex Inclusive Research Framework to address sex bias in preclinical research proposals." Nat Commun 16, 3763 (2025). DOI: 10.1038/s41467-025-58560-5  

About the Speaker:

Natasha is a Director of Hit Discovery and Biostatistics within AstraZeneca leading a team of statisticians and bioinformaticians supporting preclinical research. Natasha is an active researcher publishing papers with a focus on the challenges within preclinical research with a particular interest in improving replicability, reproducibility, and generalizability of the studies.   In recent years, the research has focused on meta-research exploring how to enable and nudge scientists into better research practice. 

Join us for the first session after the summer break with Dr. Manoj Lalu (University of Ottawa, Canada) and learn about patient engagement in preclinical research. 

About the Talk:
Patient engagement in research, defined as “research being carried out with or by patients, caregivers, or the public, rather than to, about, or for them” ensures that those with lived experience of a health condition (and their families or caregivers) become active members of research teams. Because patients are the end-users of healthcare innovations, their perspectives help guide research design, conduct, and dissemination, making studies more relevant, meaningful, and impactful.
While patient engagement has gained significant traction in clinical research, its application in preclinical (laboratory-based) research remains relatively uncommon. Dr. Manoj Lalu will discuss the opportunities and challenges of preclinical patient engagement, sharing insights from his work on involving patients in laboratory studies.

About the Speaker:
Dr. Manoj Lalu is an Associate Professor in the Department of Anesthesiology and Pain Medicine at the University of Ottawa, with a cross-appointment to the Department of Cellular and Molecular Medicine and an adjunct appointment at the School of Epidemiology and Public Health. He serves as Lead of the Blueprint Translational Research Group and Associate Scientific Director of Training at the Ottawa Hospital Research Institute, where he is also a Scientist in the Acute Care Research and Regenerative Medicine Programs. His research focuses on cell therapy for perioperative medicine and critical illness, patient engagement in clinical and preclinical research, and preclinical multi-laboratory studies to reduce bias and improve rigor in preclinical science. Dr. Manoj Lalu is also active in meta-research and contributes to initiatives addressing predatory journals.

Policy Brief: Engaging Patients as Partners in Preclinical Laboratory Research by The Blueprint Translational Research Group 

Fox, Grace et al. "Patient engagement in preclinical laboratory research: A scoping review." EBioMedicine vol. 70 (2021): 103484. DOI: 10.1016/j.ebiom.2021.103484 

Join the seventh session with Dr. Fanglian He (Bio-protocol) to learn about methods and practices that improve research reproducibility in preclinical research.

Reproducibility remains a critical challenge in preclinical research. The Reproducibility Project: Cancer Biology, as reported in eLife (Errington et al., 2021), reveals that inadequate protocols are a major factor critically impeding the replication of findings in preclinical cancer biology. In this talk, Dr. Fanglian He, co-founder and publisher of Bio-protocol, will discuss practical solutions for enhancing reproducibility, with a focus on open and reusable protocols. She will share recommended actions that stakeholders can take to address irreproducibility, as proposed by PRO-MAP, an initiative led by the EU's Joint Research Centre. Dr. He will also provide insights into how, over the past decade, Bio-protocol has supported the scientific community in improving reproducibility by publishing and sharing detailed, step-by-step life sciences protocols.

Attendees will gain a clearer understanding of the role of open protocols in addressing irreproducibility, and leave with actionable steps for fostering a more open and trustworthy preclinical research community.

About the Speaker: 

Fanglian He is the publisher of Bio-protocol. She co-founded Bio-protocol in 2011 while at Stanford University, after experiencing firsthand the difficulties of reproducing published experiments. Motivated to help researchers share detailed, step-by-step protocols, she received strong support from the Stanford community. After completing her postdoc at the Carnegie Institution for Science at Stanford, Dr. He transitioned full-time to Bio-protocol in 2013, dedicated to improving the accessibility and reliability of protocols in the scientific community. She holds a PhD in Biology from the University of Pennsylvania.

In the sixth session, Dr. Nikki Osborne (Responsible Research) and Dr. Nathalie Percie du Sert (NC3Rs) will explore various resources that support researchers to improve the reliability of in vivo and in vitro experiments including the Experimental Design Assistant (EDA), ARRIVE guidelines, and DRIVER recommendations.

About the Speakers:

Dr Nikki Osborne is a developmental neurobiologist with over 25 years of experience in the lab animal science sector. She is the founding director of Learning Development & Research Training Ltd (trading as Responsible Research & Mindset Action) and works globally with research organizations, funders, regulators, and publishers. She is passionate about empowering researchers and institutions to improve the rigor and reproducibility of life science research. 

Dr Nathalie Percie du Sert is the Director of Research Practice at the NC3Rs, where she leads efforts to improve research quality and reproducibility. She serves on the Advisory Board of the Swiss Reproducibility Network, the Steering Board of the Reproducibility by Design program at the University of Bristol, and the Scientific Advisory Board for the DECIDE project. She has played a central role in the development of key resources such as the Experimental Design Assistant (EDA), a free online tool for designing robust animal studies, and the ARRIVE 2.0 guidelines, which promote transparent reporting.

In the fifth session, Dr. Christoph Conrad and Dr. Michael Moles of the Regulatory Support Unit at the Berlin Institute of Health will provide an overview of the regulatory requirements for the preclinical stage of Gene and Cell Therapy (GCT) product development. 

About the Speakers:

Dr. Christoph Conrad (Head of Staff Office Regulatory Affairs and Regulatory Support Unit, BIH) brings a wealth of regulatory and scientific expertise from a variety of fields. He was responsible for the evaluation of vaccines at the Paul Ehrlich Institute (PEI) for many years. Later he headed the Office for Scientific and Regulatory Advice (OSRA), an infrastructure of the German Center for Infection Research (DZIF) at the PEI. In addition to his regulatory work, Christoph Conrad gained several years of experience at the WHO in Geneva, the BMBF and the Federal Chancellery. In July 2022, he joined the BIH as Head of Staff Office Regulatory Affairs, and is also Head of the Regulatory Support Unit that has been established in June 2024.

Dr. Michael Moles (Regulatory Affairs Specialist for Biologicals) completed his PhD in Cancer Studies at the University of Glasgow, specializing in targeted therapy for chronic lymphocytic leukemia. He later pursued a Postdoc at the Max Delbrück Center, where his main focus was the development of CAR-T and CAR-NK cell therapies for blood cancer. In April 2024, he joined the Regulatory Support Unit at the Berlin Institute of Health as a Regulatory Affairs Specialist, where, alongside support for academic ATMP developers, he is participating in the EU Join4ATMP consortium and is a member of the International Rare Disease Research Consortium Task Force on regulatory convergence.

In the fourth session, Dr. Samuel Pawel (Epidemiology, Biostatistics and Prevention Institute, University of Zurich) will explore the concept of replicating null findings and present methods for evaluating their replication.
Pawel S. et al. (2023). Replication of "null results" – Absence of evidence or evidence of absence? eLife12:RP92311 DOI:10.7554/eLife.92311.2

About the Speaker:

Samuel Pawel is a postdoctoral researcher at the University of Zurich specializing in (bio)statistics and meta-research. His work focuses on statistical methods for evaluating scientific evidence, with a particular interest in replication studies. He has contributed to methodological advances in statistical inference and methods for assessing replication success. Beyond research, he is involved in promoting open and reproducible science principles through his contributions to the Center for Reproducible Science at the University of Zurich and the Swiss Reproducibility Network.

In the third seminar, Dr. Vibeke Fosse (Department of Clinical Science, University of Bergen) will present a set of recommendations designed to enhance the robustness of preclinical methods in translational research for personalized medicine.

Fosse V. et al. (2023). Recommendations for robust and reproducible preclinical research in personalised medicine. BMC Med 21, 14. DOI:10.1186/s12916-022-02719-0

About the Speaker:

Vibeke completed a professional veterinary degree from the Royal Veterinary College, University of London in 2003, and specialized in companion animal oncology in 2019 from the University of Copenhagen, Denmark. Since 2016, Vibeke has also been working as an academic researcher at the University of Bergen, in a research group that focuses on the development of clinically relevant preclinical models for cancer, combined with the development of preclinical imaging modalities. From 2020-2022, Vibeke was involved in the PERMIT project funded by the European Clinical Research Infrastructure Network (ECRIN), to lead explorations into the optimal exploitation of preclinical research to drive personalized medicine trials.

In the second seminar session, Prof. Peter Loskill (Organ-on-Chip Research, University of Tübingen) will share his expertise on new approach methods (NAMs), with a particular focus on how microphysiological systems are advancing translational preclinical research.

About the talk:

Drug discovery and development to date has relied on animal models, which are useful, but fail to resemble human physiology. Technological advances at the interface of microsystem engineering and cell biology have provided an entirely new class of human relevant in vitro models – Organ-on-Chips (OoCs) – that have led to the emergence of a new paradigm of drug development using patient- and disease-specific models. OoCs integrate human µ-tissues into microfluidic devices tailored to create physiological microenvironments and recapitulate organ-level functionality. Moreover, OoCs provide the opportunity to mimic aspects of the human immune system, which is a multi-faceted challenge and a major opportunity at the same time: The complexity of the immune system, significant species-differences in immune response, and large population heterogeneities have created an immense need for human-relevant immunocompetent models.

Using microfabrication techniques, the team of Prof. Loskill has developed diverse OoCs with complex, immunocompetent 3D human tissues that remain viable and functional for weeks, including Tumor-, Retina-, Choroid-, Heart-, Pancreas-, Lymphoid-tissue-, and Adipose-on-chip models. The OoCs generally consist of three functional components: organ-specific tissue chambers; "vasculature-like" media channels enabling a precise and computationally predictable delivery of soluble compounds; "endothelial-like" barriers protecting the tissues from shear forces while allowing diffusive transport. To recapitulate human immune responses, Prof. Loskill’s team has successfully integrated components of the innate and adaptive immune system into the OoCs demonstrating the complex interplay of different cell populations as well as their importance for pharmaceutical safety and efficacy assessment. 

About the speaker:

Prof. Dr. Peter Loskill is W3-Professor for Organ-on-Chip Research at the Eberhard Karls University Tübingen and the Natural and Medical Sciences Institute (NMI) as well as Past-Chair of the European-Organ-on-Chip-Society (EUROoCS). He graduated in 2012 from Saarland University with a PhD in Physics and thereafter worked as a postdoctoral fellow in the Healy Lab at UC Berkeley. In 2015, he was named as one of Technology Review’s "Innovators under 35 Germany" and awarded an ATTRACT starting grant. He now heads the interdisciplinary µOrgano-Lab and the 3R Center Tübingen for In vitro Models and Alternatives to Animal Testing. 

Join the first seminar session with Randall J. Ellis, PhD, (Department of Biomedical Informatics, Harvard Medical School) and learn about the importance of pre-registrations and registered reports in preclinical research.

Ellis R. J. (2022). Questionable Research Practices, Low Statistical Power, and Other Obstacles to Replicability: Why Preclinical Neuroscience Research Would Benefit from Registered Reports. eNeuro, 9(4), DOI: 10.1523/ENEURO.0017-22.2022

About Randall J. Ellis:

He completed his BS in Neuroscience at Florida Atlantic University in 2014, followed by a postbac at the National Institute on Drug Abuse, where he studied cocaine’s effects on behavior and developed computational biology skills. He earned his PhD at Mount Sinai in 2022, focusing on opioid use disorder and neurocognitive risks. Currently, he is a postdoc at Harvard Medical School, using statistical modeling to assess dementia risk and exploring metascience issues in biomedical informatics. A lot of his work focuses on the intersection of metascience and biomedical informatics, specifically how the performance of machine learning models can be reported in misleading ways.

Meet the Speaker:

If you'd like to meet our speaker in person outside of the webinar to discuss enhancing quality in preclinical research, Randall will be at the BIH QUEST Center from January 28–30, 2025. Feel free to contact decide@charite.de to set up a meeting.