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BIH Lecture 27: „Basic science, novel modalities and innovative therapies for heart failure“

Cardiovascular diseases are the major cause of morbidity and mortality worldwide. Despite major recent breakthroughs regarding the underlying genetics and molecular mechanisms, our abilities to therapeutically interfere remain limited.

In the BIH Lecture on November, 25 2022, Prof. Dr. Ralph Knöll (Chief Scientist at AstraZeneca) gave a brief overview regarding the genetics of heart failure, the downstream effects for physiology and myocardial performance as well as novel modalities including those directly interfering with RNA and gene expression. He also discussed novel targets for future drug development, particularly regarding the sarcomere, cardiomyocyte and other cell types. The power of and potential use of therapeutic genome editing approaches will be discussed.

The Lecture was moderated by Dr. Stefanie Seltmann (BIH).

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Lecture 26: "From Understanding Human Pluripotency to Disease Modelling and Cell-Based Therapy"

In the BIH Lecture on September 30, 2022, Ludovic Vallier (Einstein Strategy Professor of Stem Cells in Regenerative Therapies at the Berlin Institute of Health at Charité) spoke about how he and his team are researching the development of new therapeutics for liver disease. End-stage liver disease is one of the leading causes of death in modern countries and can only be treated by liver transplantation. Therefore, the development of new therapeutics against liver diseases is urgently needed. The team led by Ludovic Vallier aims to address this global public health challenge by studying the mechanisms that control liver development and, in particular, the production of cholangiocytes and hepatocytes, the two major functional cell types of the liver. They are using human pluripotent stem cells and primary liver organoids to study the mechanisms that drive the progression of nonalcoholic fatty liver disease and to develop new regenerative therapies.

Lecture 25:"Novel Approaches To Targeting Hematopoietic Stem Cells for Ex Vivo and in Vivo Gene Therapy"

In the BIH Lecture on September 16, 2022, the world-renowned expert in stem cell gene therapy, Hans-Peter Kiem (Director, Associate Head, and Professor of different programs at the Clinical Research Center of the Fred Hutchinson Cancer Center in Seattle, WA/USA), talked about HSC gene therapy and addressed current limitations his team encountered regarding HSC characterization, engraftement and targeting. In his talk, Kiem focused on gene editing approaches and disease applications. He explained that many diseases could be treated with HSC gene therapy and elaborated that the largest population are patients with sickle cell disease, thalassemia or HIV and the highest incidents regarding mortality are in located in low and middle-income countries. So the Kiem lab works intensively on how to make gene therapy more accessible and potentially be able to move it to the beforementioned settings.

The lecture was moderated by Christopher Baum (BIH) and followed by a Meet the Speaker-session, led by Stefanie Seltmann (BIH).

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Lecture 24: "Democratising Drug Development Through Human Genomics"

In his BIH Lecture on June 24, 2022, Aroon Hingorani (Chair of Genetic Epidemiology, UCL) talked about the high failure rate in drug development processes and the undesirable consequences for healthcare systems and the pharmaceutical industry. About 96% of programs fail and reviews conclude that the major reason is the lack of efficacy in the intended indication – often unveiled at the final stage. One of the flaws lies in the target-disease matching system: model organisms are often an unreliable predictor of treatment efficacy in humans. Hingorani showed the high potential of human genomics as a tool for drug target identification and validation - an approach that would eventually need a wider societal engagement but could provide a lever for more needs-led than profit-led approach to drug development.

The lecture was moderated by Birgit Sawitzki (BIH Professor for Translational Immunology).

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Lecture 23: "Insider Advice From a Scientific Editor"

On April 1, 2022, Cherilyn M. Sirois was the BIH Lecture speaker and talked about her career path and her work as a Scientific Editor at Cell. Sirois provided insider tips on communicating with editors and reviewers - from cover letters and abstracts to submission and formatting. What exactly do journals want and how do Cell editors really think about papers? Following the lecture, Sirois answered numerous questions from participants about publishing.

The lecture was moderated by Birgit Sawitzki (BIH Professor for Translational Immunology) and Rosario Astaburuaga Garcia (Charité Institute of Pathology | Integrative Research Institute for the Life Sciences at HU Berlin).

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Lecture 22: "Is the Scientific Literature Better Today Than It Was 10 Years ago?"

In her BIH Lecture on March 25, 2022, Anita Bandrowski (neuroscientist at University of California at San Diego and Founder and CEO of SciCrunch Inc.) explored the types of metrics that can be gathered about the scientific literature, why the metrics exist and how people use them. After a broad look at multiple metrics, she discussed RRIDs (Research Resource Identifiers). She explained why and how to use these identifiers for key biological reagents to tell others exactly what materials, model organisms or software and tools were used to complete the research. During the lecture, Bandrowski also examined the elements of the RTI (Rigor and Transparency Index) and explained why we think that putting a number on a manuscript is useful.

Bandrowski is currently BIH Visiting Fellow of the Stiftung Charité. The lecture was moderated by Ulrich Dirnagl (BIH QUEST Center for Responsible Research)

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Lecture 21: "The Transferbarometer"

On February 18, 2022, Andrea Frank (Deputy Secretary General and Member of the Executive Board at Stifterverband) gave a BIH Lecture on the project ‘Transferbarometer’ – a tool to evaluate and expand knowledge exchange at universities and research institutions. The transfer concept was developed by a group of five universities and six Helmholtz-Centers to clarify the range of possible transfer and cooperation activities as well as institutional preconditions. Frank presented which indicators were identified and tested to systematically assess, present and communicate knowledge exchange achievements.

The lecture was followed by a lively discussion during the Meet the Speaker-session, hosted by the BIH QUEST Center for Responsible Research and moderated by Ulrich Dirnagl and Miriam Kip.

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Lecture 20: "Engineering Organoid Development"

On December 17, 2021, Matthias Lutolf (Roche Institute for Translational Bioengineering, EPFL Lausanne) was a guest in the BIH Lecture Series and spoke about the central topic of his lab: improving organoid technologies through various engineering approaches. Lutolf discussed the efforts in developing next-generation organoids that are assembled by guiding cell-intrinsic self-patterning through engineered stem cell microenvironments. He presented several examples of his group's research to improve the potential for real-life applications to make organoid models useful, for example, for drug development and ultimately for regenerative medicine.

The lecture was organized in cooperation with the German Stem Cell Network (GSCN) and moderated by Christopher Baum (BIH) and Hans Schöler (President GSCN)

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Lecture 19: "A Prognostic Atlas for Clinical Medicine"

On November 26, 2021, Harry Hemingway (Research Director HDR UK | Director UCL Institute of Health Informatics | Charité Guest Professor) spoke about the systematic interrogation of data for the benefit of healthcare systems across the world. Although each patient leaves a digital trace in the electronic health record (EHR), in practice there has been a lack of systematic approaches to generating research insights across common and rare diseases using EHR. Healthcare systems know remarkably little about which patients have which diseases, in which combinations, and with what outcomes. In his lecture Hemingway presented opportunities and challenges in developing a prognostic atlas for clinical medicine. The BIH Lecture was moderated by Prof. Christopher Baum (BIH) and Prof. Christof von Kalle (Charité BIH Clinical Study Center).

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Lecture 18: "Impact of Clinical Trial Data Sharing Policies"

On October 8, 2021, Florian Naudet (Professor of Therapeutics from Rennes 1 University in France) reported in the BIH Lecture on policies, practices, and problems related to the sharing and reuse of clinical trial data. The topic of the talk was related to the news in June 2021 when the European University Hospital Alliance, of which Charité is a member, signed the Sorbonne Declaration on Research Data Rights. Naudet showed that data sharing policies of journals are still poorly implemented, and clinical data sharing is not common. The lecture was moderated by Prof. Dr. Ulrich Dirnagl (BIH Chair, QUEST Center for Responsible Research).

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Lecture 17: "From Molecules to Medical Records – Insights From Large-Scale, Multi-Omic Studies"

On September 10, 2021, we welcomed Claudia Langenberg (BIH Professor of Computational Medicine at the Berlin Institute of Health at Charité (BIH)) as speaker of our Online BIH Lecture. Together with her team, she combines genomic with metabolomic and proteomic data to define the genetic architecture of thousands of ‘omic’ traits and integrate data from different platforms and studies to maximize power for the identification of genetic variants associated with life-long differences in metabolite and protein levels and enable investigation of their relevance for disease aetiology and prediction. While giving insights from large-scale, multi-omic studies, she spoke about her work, its importance to translational medicine, and the related challenges.

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Lecture 16: "Current Status and Future Vision of Retinal Cell Therapy"

On May 28, 2021, the BIH Lecture Series took place for the second time in cooperation with the German Stem Cell Network (GSCN). This time the lecture came directly from Japan by the speaker and pioneer of iPS Therapy Masayo Takahashi (MD, PhD). Takahashi is president of the startup Vision Care Inc. and was involved in the world's first transplantation of retinal cells derived from induced pluripotent stem (iPS) cells in her role as project leader at the Riken Center for Developmental Biology. In her talk, Takahashi discussed the current status and fascinating development potential of retinal cell therapy and was available for a "Meet the Speaker" afterwards. The BIH Lecture was moderated by Prof. Hans Schöler (President GSCN) and Prof. Georg Duda (BIH Chair/Charité).

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Lecture 15: "Gene Edited Human Muscle Stem Cells as Advanced Therapies"

On April 23, 2021, Simone Spuler was guest in the BIH lecture series "Frontiers in Translational Medicine - Scientific and Structural Challenges". Spuler is Head of the University Out-Patient Clinic for Muscle Disorders and Senior Group Leader of the Myology Research Group at the Experimental and Clinical Research Center Charité (ECRC) of Charité - Universitätsmedizin Berlin and the Max Delbrück Center for Molecular Medicine (MDC). In her talk, she explained how muscle stem cells are returning to the focus of attention in the treatment of muscle wasting and demonstrated the unprecedented efficacy of gene correction strategies derived from CRISPR/Cas9 techniques in primary human muscle stem cells.

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Lecture 14: "Why Translational Medicine Depends on Interoperability"

On March 12, 2021, Sylvia Thun held an online lecture in the framework of our BIH Lecture Series “Frontiers in Translational Medicine – Scientific and Structural Challenges”. Thun is the Director of the Core Unit eHealth and Interoperability (CEI) at the Berlin Institute of Health (BIH) at Charité – Universitätsmedizin Berlin. In her talk, she explains why interoperability of medical data is fundamental to digital innovation and how it facilitates digital transformation in research and medicine, which in turn benefits patients.

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Lecture 13: "The 100,000 Genomes Project – Transforming Healthcare"

On January 29, 2021, Sir Mark Caulfield MD FRCP FMedSci gave a great talk as part of our BIH Lecture Series “Frontiers in Translational Medicine – Scientific and Structural Challenges”. Professor Caulfield is Chief Scientist for Genomics England and Director of the NIHR Barts Biomedical Research Centre. In our Online BIH Lecture, he talked about the 100,000 Genomes Project, the exciting opportunities it offers researchers, and how it is advancing medical translation.

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Lecture 12: "Keys to Successfull Technology Translation by Entrepeneurs in Academia and Biotech"

On December 11, 2020, Angelika Fretzen was a speaker at the BIH Lecture Series. She is Technology Translation Director, COO Wyss Institute for Biologically Inspired Engineering at Harvard University. The Wyss Institute for Biologically Inspired Engineering at Harvard University has developed a unique model that takes the most innovative ideas from 'blue sky' research by leading academic groups in the Boston area and translates them into risk-free technologies.

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Lecture 11: "Cerebral Organoids - Modeling Human Development and Disease"

On November 27, 2020, Jürgen Knoblich (Senior Scientist and Scientific Director of the Institute of Molecular Biotechnology of the Austrian Academy of Sciences - IMBA) gave a talk at the BIH Lecture Series. In his research group he is investigating cerebral organoids to recapitulate diseases of the human brain in vitro and to test risk factors. Cerebral organoids derived from patients suffering from a neurological developmental disorder can recapitulate the developmental defects leading to these diseases and allow us to unravel the mechanistic complexity of disorders such as epilepsy and autism.

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Lecture 10: "Metabolic Aspects of Angiogenesis in Physiology and Disease"

On October 30, 2020, Michael Potente (BIH Professor for Translational Vascular Biomedicine) was speaker in the BIH Lecture Series. He spoke about the influence of metabolic processes on the formation of blood vessels in health and disease. Aberrant angiogenesis is a characteristic and trigger of cancer or inflammation, but therapeutic approaches have so far shown little success. New insights into the metabolic processes that small blood vessels use to form networks open up new possibilities for intervention.

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Lecture 9: "The BIH - an Institute Dedicated to the Science of Translation"

On October 2, 2020, the new BIH CEO Christopher H. Baum took office and took the opportunity to speak at our BIH Lecture Series. His lecture dealt with BIH and the question of how translational medicine and a science of translation can succeed.

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Lecture 8: "Frontiers in Translational Mecine - An Industry Perspective"

On July 31, 2020, Florian Gantner (Senior Vice President Translational Medicine & Clinical Pharmacology at C.H. Boehringer Sohn AG & Co. KG) was guest in our BIH Lecture Series. He gave a lecture on the industrial view on the scientific and structural challenges of translation.

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Lecture 7: "How to Create a Scientific Spin-off"

On June 5, 2020, Sonja Jost (CEO of the start-up company DexLeChem) was a guest in our BIH Lecture Series. She spoke in her lecture about the ways and possibilities to apply scientific knowledge about a start-up company. The seventh session of the Lecture Series was the second one that we offered completely as video conference.

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Lecture 6: "What does it take to translate? – Lessons Learned in Regenerative Medicine"

On March 27, 2020, Georg N. Duda (co-speaker of the BCRT and director of the Julius-Woff-Institute for Biomechanics and Musculoskeletal Regeneration at the Charité - Universitätsmedizin Berlin) gave an insight into the experiences of the BIH Center for Regenerative Therapies (BCRT) with translation in regenerative medicine. The sixth lecture of the series was also a premiere - in the time of the COVID 19 pandemic it was the first lecture organized by the BIH as a video conference with about 170 participants*.

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Lecture 5: "Translating Data into Health – Clinical Translational Research in the Digital Age"

On February 28, 2020, Christof von Kalle (BIH-Chair for Clinical Translational Sciences and Director of the Clinical Study Center of the BIH and Charité - Universitätsmedizin Berlin) gave an introduction to the challenges of Clinical Translational Research in the Digital Age. At our fifth session of the lecture series "Frontiers in Translational Medicine - Scientific and Structural Challenges" he spoke on "Translating Data into Health - Clinical Translational Research in the Digital Age".

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Lecture 4: "From Infection Research to New Antiviral Drugs"

On February 14, 2020, Susanne Herold (Universities Giessen & Marburg Lung Center (UGMLC)) presented the perspective of Clinician Scientists on translational research. She is Professor of Lung Diseases at the University Hospital Gießen-Marburg, where she heads the Department of Infectiology. At our fourth session of the lecture series "Frontiers in Translational Medicine - Scientific and Structural Challenges" she spoke on the topic "From infection research to new antiviral drugs" and gave an insight into her current research.

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Lecture 3: "Immunological Challenges of Regenerative Therapies - what, why, how?"

On January 31, 2020, the BIH again invited participants to reflect and discuss the challenges of translational research in the historic Virchow lecture hall of the Charité – Universitätsmedizin Berlin. At the third lecture of the series and the first one this year, Hans-Dieter Volk (speaker of the BIH Center for Regenerative Therapies) spoke on the topic "Immunological Challenges of Regenerative Therapies - what, why, how?"

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Lecture 2: "Debunking Common Myths about Translation"

On November 22, 2019, the BIH invited to the second lecture of the series. Ulrich Dirnagl (founding director of the BIH QUEST Center) spoke on "Debunking Common Myths about Translation". He argued that, over the last twenty years, ideas of translation have become ingrained that were not true and distracted from different or even better approaches to translation medicine.

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Lecture 1: "Frontiers in Translational Medicine - Scientific and Structural Challenges"

Axel Radlach Pries (Chairman of the BIH Board (interim) and Dean of the Charité - Universitätsmedizin) opened the new lecture series on November 8, 2019 in the Hans Virchow lecture hall of the Charité Campus Mitte.  With questions and answers on the structures and conditions of successful translational medicine, he addressed all those interested in translation and the work of BIH.

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