Thursday, November 29th

The FORUM Translational Medicine 2018 is the first event of a biennial series with focus on the challenges and possibilities of the translational process of biomedical research, clinical development, and precision therapies. The conference will present highlights in research & development of New Therapies– from academia, clinic, spin-offs, and industry.

Conference Program

Welcome and KeyNote

09:00 am
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Welcome note and conference opening

Chair: Prof. Horst Domdey, Managing Director
BioM Biotech Cluster Development GmbH
 
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Prof. Caroline Kisker, Chair Rudolf Virchow Center for Experimental Biomedicine
University of Würzburg
09:20 am
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KeyNote Talk
Prof. Jochen Maas, Director Research & Development
Sanofi-Aventis Deutschland GmbH
Sanofi’s model for R&D cooperation in Translational Medicine Projects >>

Session A: New Targets and Success Stories in Immunotherapy

 
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Chair: Dr. Gerhard Frank, Project manager and start-up consultant
Innovations- und Gründerzentrum Würzburg
10:00 am
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Prof. Christina Zielinski, Head of Cellular Immunoregulation lab - TranslaTUM
Technical University Munich
From cellular immunology to targeted therapies in dermatology >>
10:20 am
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Prof. Philipp Beckhove, Director Center of Interventional Immunology RCI
University of Regensburg
New targets in Immunotherapy >>
10:40 am
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Prof. Ralf Bargou, Chair Translational Oncology, Director Compreh. Cancer Center
Universitätsklinikum Würzburg
The Blinatumomab-Story and far beyond – the clinician perspective
11:00 – 11:30 am
Coffee break and exhibition

Session B: Pathways for New Therapies

 
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Chair: Dr. Ulrike Kaltenhauser, Managing Director
BioSysNet
11:30 am
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Prof. Caroline Kisker, Chair Rudolf Virchow Center for Experimental Biomedicine
University of Würzburg
The Good and the Bad of NER or the Double Edged Sword of DNA Repair >>
11:50 am
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PD Dr. Oliver Pullig, Head of Implants & GMP
Fraunhofer Institute for Silicate Research ISC
Innovative cell-based therapies in the treatment of cartilage defects >>
12:10 pm
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Prof. Martin Ungerer, Cardiologist
advanceCOR GmbH
Antigen-specific therapy of hormone receptor-targeting autoimmune disease >>
12:30 pm
Lunch break, exhibition walk and poster session

Break out Session: Hubs in Translational Medicine and Start up Pitches

 
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Chair: Dr. Georg Kääb, Managing Director Bavarian Biotech Cluster
BioM Biotech Cluster Development GmbH
02:00 pm
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Prof. Christoph Maack, Director of Translational Research CHFC
Universitätsklinikum Würzburg
Translational concepts in heart failure research and treatment >>
 
Pitch arena at FORUM Translational Medicine
02:15 pm
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Dr. Nadja Fenn, Gene Center and Department of Biochemistry
LMU München
OPSYON – Fusing tumor targeting and immune checkpoint blockade >>
 
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Prof. Matthias Mack, Department of Internal Medicine II - Nephrology
Universitätsklinikum Regensburg
IL-3 as therapeutic target in autoimmune diseases >>
 
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Dr. Valentin Bruttel, Project Leader
AIM Biologicals, Universitätsklinikum Würzburg
AIM Biologicals: rethinking targeted therapeutics for autoimmune diseases >>
 
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Dr. Yelena Wainman, Project Leader
Targeted Cancer Chemotherapeutics, Department of Pharmacy, LMU Munich
Development of light-responsive cancer chemotherapeutics >>
 
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Dr. Sebastian Kreß, Project Leader
Tissue Engineering und Regenerative Medizin, University Hospital Würzburg
Biologically Vascularized Scaffold for Tissue Generation and Translation >>
02:45 pm
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Dr. Jutta Heix, Head of International Affairs
Oslo Cancer Cluster
Rescuing the lost in translation: SPARK Norway and the global SPARK Network >>

Session C: Enabling New Technologies for Therapies & Diagnostics

 
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Chair: Dr. Ilja Hagen, Project Manager Healthcare
BioPark Regensburg GmbH
03:00 pm
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Dr. Konstantin Zhernosekov, CSO / Managing Director ITM Oncologics
ITM Isotopen Technologien München AG
Theranostics - Targeted Radionuclide Diagnostics & Therapy for cancer treatment >>
03:20 pm
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Prof. Christoph Klein, Chair Experimental Medicine and Therapy Research
University Regensburg and Fraunhofer Center Regensburg
Detection, characterization and monitoring of early and advanced systemic cancer >>
03:40 pm
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Dr. Christine Günther, Chief Executive Officer
apceth Biopharma GmbH
Cell and Gene Therapy – from research to patients >>
04:00 pm
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Final KeyNote
Prof. Harald Schmidt
, Head of Department of Pharmacology
and Personalised Medicine, Maastricht Universität Niederlande
The end of medicine as we know it >>

04:30 pm
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Closing remarks

Dr. Georg Kääb, Managing Director Bavarian Biotech Cluster
BioM Biotech Cluster Development GmbH
until 06:00 pm
GET TOGETHER: COFFEE AND BEER

PD Dr. Oliver Pullig

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Head of Implants & GMP
Fraunhofer Institute for Silicate Research ISC

PD Dr. Oliver Pullig is Head of Regulatory Affairs and vice chair of the Translation Center Regenerative Therapies – Fraunhofer ISC in Würzburg (TLZ-RT). He is designed as Qualified Person and Head of Manufacturing for processing variant ATMPs at University Hospital Würzburg production site.

Oliver Pullig studied biology at the University in Mainz and completed his PhD thesis at the University of Erlangen. He worked in variant orthopedic departments and has as an over 10 years’ experience in the field of musculoskeletal diseases and in the development and coordination of advanced therapies.

At TLZ-RT, he established GMP conform infrastructure including QM system, SOP-implementation, and special educated staff.

Dr. Valentin Bruttel

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Project Leader
AIM Biologicals, Universitätsklinikum Würzburg

Valentin Bruttel leads the AutoImmunity Modifying Biologicals (AIM Biologicals) project at the University Hospital of Würzburg together with Prof. Jörg Wischhusen. He has discovered the underlying mechanism for targeted modifications of defined immune responses during his PhD in the work group of Prof. Wischhusen. He obtained a M.Sc. in Molecular Medicine from Trinity College in Dublin and a Bachelor of Science in Biomedicine from the University of Würzburg.

He co-founded two companies, authored several publications and has received awards including a DFG PhD fellowship, the m4 award by the Bavarian Ministry for Economic Affairs and a “new ideas for bioeconomy” grant by the German Ministry for Science and Education. Valentin Bruttel was born in 1984 in Baden-Württemberg and is father of three children.

Dr. Sebastian Kreß

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Project Leader
Tissue Engineering und Regenerative Medizin, University Hospital Würzburg

Sebastian Kreß, studied biology, Bachelor and Master of Science, at the Julius-Maximilians-University of Würzburg (Germany). I conducted my Master thesis at the National University of Singapore (Singapore) studying the ‘Extracellular matrix influence in fibrogenesis - investigating in vitro effects of dynamic reciprocity’.

Staying in the field of tissue engineering, I started doing my doctoral thesis at the chair of Tissue Engineering und Regenerative Medicine on the ‘Development and proof of concept of a biological vascularized cell-based drug delivery system’.

Dr. Yelena Wainman

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Project Leader
Targeted Cancer Chemotherapeutics, Department of Pharmacy, LMU Munich

Dr. Yelena Wainman studied Natural Sciences at University College London and the Ecole Normale Supérieure de Lyon, specializing in organic chemistry. She completed her PhD in medicinal chemistry at the University of Cambridge in the area of novel agents for cancer imaging. She then did a post-doc at Harvard Medical School working on photoswitchable epigenetic inhibitors. She was active in the biotech/start-up scene in both Cambridge and Boston (Kaleidoscope, Palletech, Oxbridge Biotech Roundtable).

After having worked in the emerging area of photopharmacology at Harvard, this led her to come to Munich to co-start the CytoSwitch start-up project together with junior group leader Dr. Oliver Thorn-Seshold at the LMU Munich. CytoSwitch is a biotech startup project developing novel light responsive cancer chemotherapeutics with reduced side-effects for poorly treatable solid tumors. CytoSwitch received the Flügge funding from the Bavarian State Ministry in as well as the pre-seed EXIST grant from the German ministry for Economics for proof-of-concept/early preclinical work until 2020.

Dr. Valentin Bruttel

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Chief Executive Officer
apceth Biopharma GmbH

Dr. Christine Günther has been Chief Executive Officer, Chief Medical Officer and Qualified Person at apceth Biopharma since 2008 (then apceth). She is a specialist in clinical hematology and oncology with extensive experience in clinical stem cell transplantation, transplantation immunology and cell therapies. Her particular area of expertise is pharmaceutical development of complex cell-based and gene therapy products.

Before joining apceth, Christine Günther worked as qualified person, head of quality control and medical director at the German public stem cell and cord blood bank. Her work there focused on stem cell and tissue procurement, donor testing and characterization of cell products – including cord blood products – and their application to the patient in the treatment of malignant diseases. Before that she gained extensive experience as a hematologist/oncologist at the University Hospital in Munich, Germany.

Dr. Christine Günther

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Chief Executive Officer
apceth Biopharma GmbH

Dr. Christine Günther has been Chief Executive Officer, Chief Medical Officer and Qualified Person at apceth Biopharma since 2008 (then apceth). She is a specialist in clinical hematology and oncology with extensive experience in clinical stem cell transplantation, transplantation immunology and cell therapies. Her particular area of expertise is pharmaceutical development of complex cell-based and gene therapy products.

Before joining apceth, Christine Günther worked as qualified person, head of quality control and medical director at the German public stem cell and cord blood bank. Her work there focused on stem cell and tissue procurement, donor testing and characterization of cell products – including cord blood products – and their application to the patient in the treatment of malignant diseases. Before that she gained extensive experience as a hematologist/oncologist at the University Hospital in Munich, Germany.

Prof. Christina Zielinski

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Head of Cellular Immunoregulation lab - TranslaTUM
Technical University Munich

Professor Zielinski (b. 1979) conducts research on the immunological principles underlying chronic inflammatory diseases and infections in humans. Through a better understanding of the regulation of human T-cells she seeks to identify molecular switches that might provide new diagnostic and therapeutic options when dealing with infections and autoimmune diseases.

Professor Zielinski studied medicine at the University of Heidelberg and at Harvard and Duke University. During her studies she spent a year as a research fellow at Yale University supported by a grant from the German National Academic Foundation. As a recipient of a DFG grant she spent over three years as a postdoctoral fellow at the Institute for Biomedicine in Bellinzona (Switzerland) in the research group of Federica Sallusto.

She acquired her German medical board certification in dermatology in Tübingen and at the Charité in Berlin where she also headed the cellular immune regulation research group for more than three years. Since 2015 she has been a professor at the Institute for Virology at TUM.

Dr. Ilja Hagen

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Project Manager Healthcare
BioPark Regensburg GmbH

Since 2016 Ilja Hagen is working as Project Manager Healthcare at the BioPark Regensburg GmbH which is the management and administrative headquarter of the life science cluster BioRegio Regensburg in East Bavaria.

Before that, he spent 10 years working as Network Manager for the Forum MedTech Pharma at Bayern Innovativ GmbH in Nuremberg.

Ilja is biologist by training and received his PhD from the Department of Cell Biology at the University Regensburg.

Prof. Martin Ungerer

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Cardiologist
advanceCOR GmbH

Medical school at the University of Munich, Germany, and at the Universities of Marseille and Nice, France

Post-doc in the lab of Prof. Martin Lohse and Prof. Ernst Winnacker, Gene Center, Max-Planck-Institute for Biochemistry, Martinsried, Germany

Resident/Senior House Officer at the1st Medical Clinic Rechts der Isar and German Heart Center, TUM University of Munich, training in internal medicine and cardiology

Since 2000: Founder and member of the company management of ProCorde GmbH, Corimmun GmbH and advanceCOR GmbH - biotech companies which established novel technologies and drugs for the treatment of thrombosis and cardiac diseases

2005 Procorde merged with Trigen plc, London, UK

2012 Corimmun was sold to Janssen pharmaceuticals Inc, New Jersey, USA, name change of remaining company activities to advanceCOR GmbH

Since 2001 lecturer in Internal Medicine and Cardiology at the University of Würzburg

2005 approved professor of internal medicine and cardiology at the University of Würzburg

Since 2014 member of the „Translational Research Group“ steering committee of the German Centre for Cardiovascular Research, Berlin

Dr. Ulrike Kaltenhauser

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Managing Director
BioSysNet

Ulrike Kaltenhauser is the Managing Director of the “Bavarian Climate Research Network” (bayklif) and the “Research Network for Molecular Biosystems” (BioSysNet), which are both funded by the Bavarian State Ministry of Science and the Arts. Previously, she was the Managing Director of the “Bavarian Genome Research Network” (BayGene), one of the first large network programs for basic research of the Bavarian Government. In addition to the other network programs, she also took over the management of the research association "Plant Fit for the Future" (FORPLANTA).

After completing her PhD in Biology at the Technical University in Munich in 1994 she worked as a postdoc at the Max-Planck-Institute of Biochemistry in Martinsried in the Department of Protein Chemistry. This was followed by a position as a laboratory manager at the University Clinic of the Ludwig-Maximilian-University in Großhadern in the field of stroke research. Since 1999, she has turned to the field of science management and initially coordinated two research associations FORGEN and FORIMMUN funded by the Bavarian Research Foundation.

Her primary aim is bundling expertise in certain fields of science on current issues and to connect the scientists across disciplines.

Prof. Horst Domdey

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Managing Director
BioM  Biotech Cluster Development GmbH

Managing Director/CEO of BioM Biotech Cluster Development GmbH and of BioAG, Martinsried. Spokesman of "Bavarian Biotechnology Cluster" mandated by the Bavarian Government, Spokesman of "Leading Edge Cluster Munich m4 - personalized medicine" (2010-2015).

Prof. Heike Walles

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Head of Regenerative Therapies in Oncology and Musculoskelettal Disease, Fraunhofer-Institut für Silicatforschung

 

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Dr. Konstantin Zhernosekov

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CSO / Managing Director ITM Oncologics
ITM Isotopen Technologien München AG

Konstantin joined ITM as Head of Product Management in 2012, and he is responsible for developing and controlling strategic concepts in business development and marketing authorization procedures, respectively. Prior to this, Konstantin worked as a Group Leader at the Paul Scherrer Institute (Switzerland) where he acquired extensive expertise in radio- and radiopharmaceutical chemistry, bringing radioisotope technologies to clinical applications. From 2006 to 2009, he earned his first spurs as Postdoc at the Technical University of Munich (TUM).

Konstantin holds a PhD in Chemistry from the Johannes Gutenberg University, Mainz (Germany).

Dr. Nadja Fenn

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Gene Center and Department of Biochemistry

Nadja Fenn studied biochemistry at the University of Bayreuth (Germany), University of Stockholm (Schweden) and the Cold Spring Harbor Laboratory (USA). Afterwards she performed her doctoral thesis in the laboratory of Prof. Erich Nigg, department of cell biology at the Max-Planck-Institute of Biochemistry in Martinsried (Germany) where she graduated in 2010. Since then, she is working on the development of new antibody-based therapies at the Gene Center of the Ludwig-Maximilians-University Munich in the group of Prof. Karl-Peter Hopfner.

Her work focuses on the generation of effective immuno-oncology therapeutics with reduced systemic toxicities to provide cancer patients a long-term possibility for progression-free survival and low risk of side effects. The project is supported by the M4 Award 2015 from the Bavarian State Ministry. 

Prof. Matthias Mack

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Department of Internal Medicine II - Nephrology
Universitätsklinikum Regensburg

Prof. Mack has studied medicine at the Ludwig-Maximilians University Munich and completed his doctoral thesis at the Institute of Immunology at the University of Munich with Prof. Riethmüller. During this time he developed the first bispecific single-chain antibodies (now called BiTEs) that engage T cells against cancer cells. This technology led to the generation of Blinatumomab, the first approved bispecific antibody. He completed residency and training in Internal Medicine and Nephrology at the University of Munich and became associate professor of internal medicine and nephrology at the Univeristy of Regensburg in 2004.

He is member of the German National Academic Foundation and received several awards inlcuding a Howard Hughes Scholarship at the Rockefeller University in Molecular and Cellular Biology, the Franz-Volhard Award of the German Society of Nephrology and the Innovation Award of the German Biotechnology-Regions. Focus of his research is the identification of new targets and development of therapeutic approaches for autoimmunity, inflammation and fibrosis using a variety of animal models and validating the results in well defined cohorts of patients.

Dr. Gerhard Frank

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Project manager and start-up consultant
Innovations- und Gründerzentrum Würzburg

Gerhard Frank holds a doctoral degree in biology and has been working as a project manager and start-up consultant at the Innovation and Entrepreneurship Center (IGZ) in Würzburg since 2012. From his previous professional career, he has extensive experience in the areas of patents, technology transfer and early-stage financing of high-tech companies. The IGZ Würzburg is the largest incubator of lower Franconia with a focus on the life sciences.

Prof. Jochen Maas

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Director Research & Development
Sanofi-Aventis Deutschland GmbH

Jochen Maas was appointed General Manager, Research & Development (R&D) at Sanofi-Aventis Deutschland GmbH in 2010. He is a member of the Global R&D Management Board and of the German Management Board of Sanofi-Aventis.

Jochen Maas started his career in PK and later expanded his responsibilities to Preclinical Development, Preclinical and Clinical Development and Research & Development. He was also responsible for Global Research & Development in the Diabetes Division and acted as Vice President R&D Europe at Sanofi-Aventis.

Jochen Maas is a professor of pharmacokinetics and administering medication at Gießen-Friedberg University of Applied Sciences. He has studied biology and veterinary medicine at the Universities of Zurich, Heidelberg and Munich.

Prof. Ralf Bargou

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Chair Translational Oncology, Director Comprehensive Cancer Center
Universitätsklinikum Würzburg

Ralf Bargou is Full Professor (W3), Chair for Translational Oncology of the University Hospital of Würzburg

since 2011 and Director of theCCCMainfranken since 2010 . He is since 2007 Head of the Interdisciplinary Phase-I/II Unit of the CCCMainfranken.

Professor Bargou studied molecular biology and medicine in Heidelberg and is hematologist and medical oncologist by training. His major research focus I on immuno-oncology, personalized medicien and the development of bispecific antibodies.

He received the following awards: Inventor of the Year Award in Immuno-Oncology, IPO Foundation, Washington DC, USA (2016), Paul Martini Award for Clinical Pharmacology,Germany (2009), Rudolf Virchow Award of the Charité Berlin, Germany (1996).

Prof. Philipp Beckhove

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Director Regensburg Center of Interventional Immunology RCI
University of Regensburg

Prof. Dr. med. Philipp Beckhove, born in 1969 in Freiburg im Breisgau, studied medicine in Hamburg and Heidelberg and graduated at the University of Heidelberg. As internist with focus on hematology/oncology, he established a research division on the subject of “translational immunology” at the DKFZ (Deutsches Krebsforschungszentrum) in Heidelberg, where he studied mechanisms of tumor control by T cells and developed novel immune therapies against cancer.

Since 2015 Prof. Beckhove is owner of the chair of Interventional Immunology at University of Regensburg and Director of Regensburg Center for Interventional Immunology (RCI).

Research focus: Regulation of spontaneous T cell responses against tumors, tumor immunotherapy

Dr. Jutta Heix

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International Advisor
Oslo Cancer Cluster

Dr. Jutta Heix is a life science professional with more than 20 years of industry experience. She holds a PhD in molecular biology from the University of Heidelberg/ German Cancer Research Center and has worked in different Business Development roles in international pharma and biotech companies prior to joining the Oslo Cancer Cluster.

Her current responsibilities and initiatives include:
Business Development interface for Oslo Cancer Cluster’s pipeline and partnering opportunities towards international partners from industry, academia and the investment community.

Planning and execution of national and international meetings, partnering events and conference sessions.

Innovation projects in Precision Medicine and Immuno-Oncology incl. Consortium partner in Horizon2020 Project PERMIDES (Personalized Medicine Innovation through Digital Enterprise Solutions) - a cross-sectoral cluster collaboration initiating and funding novel collaboration projects between biopharma and IT SMEs.

Co-Founder of the Norwegian SPARK Academic Innovation Program in Oslo and member of Management Team (SPARK Norway is based on Stanford University’s SPARK Program and part of the SPARK Global Network).

Prof. Caroline Kisker

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Chair Rudolf Virchow Center for Experimental Biomedicine
University of Würzburg

Throughout her career Caroline Kisker pursued the analysis of medically relevant proteins, starting with the tetracycline repressor (Science 1994) during her PhD followed by the essential enzyme sulfite oxidase (Cell 1998) as a postdoc. After becoming an independent investigator she focused on the molecular machinery maintaining the genomic integrity.

Defects in these systems lead to a dramatically increased predisposition to cancer and accelerated ageing but also counteract the successful treatment of patients with antitumor agents. Her focus on nucleotide excision repair (NER), the most versatile DNA repair mechanism with respect to the repertoire of lesions being targeted, started initially with the prokaryotic system. Dr. Kisker elucidated several steps within this pathway (EMBO J 1999, EMBO J 2004, EMBO J 2005, NSMB 2006, EMBO 2007).

In recent years she targeted the eukaryotic system and solved the structure of the XPD helicase which is involved in the damage verification process during NER. She then analyzed XPD in the presence of DNA (Plos Bio 2008, EMBO J 2011), providing insights how damaged DNA could be perceived. Importantly, her subsequent analysis of XPD in the context of transcription and DNA repair revealed that all enzymatic activities of XPD are linked to the repair process but are not relevant for transcription.

This result has important consequences for the development of anti-tumor drugs targeting XPDs enzymatic activity (PLos Bio 2014). Her very recent analysis of XPA in the presence of damaged DNA showed how this protein could potentially also be involved in damage verification (PNAS 2015).

In addition to the work on the nucleotide excision repair proteins Dr. Kisker has also expanded her interest towards other genome caretakers such as the RecQ helicases. Very recently her laboratory could show for the first time how the RecQ4 protein exerts its helicase mechanism (Nat. Comm. 2017).

Prof. Christoph Klein

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Chair Experimental Medicine and Therapy Research
University Regensburg and Fraunhofer Center Regensburg

Christoph Klein studied medicine in Munich, Toronto and New Orelans and performed his doctoral thesis at the Institute of Immunology in Munich (Prof. Gert Riethmüller). In 2004 he received his postdoctoral lecturing permit (Habilitation) and became professor for Oncogenomics in 2006 at the University of Regensburg.

In 2010 he became Chair of Experimental Medicine and Therapy Research, University of Regensburg. In 2011 he started to set up the Fraunhofer project group for „Personalized Tumor Therapy“ which became a Division of the Fraunhofer ITEM in 2017.

Prof. Christoph Maack

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Director of Translational Research Comprehensive Heart Failure Center
Universitätsklinikum Würzburg

Christoph Maack received his MD at the University of Cologne (Germany) in 2000. Between 2000 and 2017, he worked at the Department of Cardiology at the University of the Saarland in Homburg, Germany, and from 2002-2005 as a post-doctoral research assistant in the lab of Brian O’Rourke at the Department of Cardiology at Johns Hopkins University in Baltimore, MD, US. In 2017, he became Director of the Comprehensive Heart Failure Center (CHFC) at the University Clinic in Würzburg, Germany, where he also chairs the Department of Translational Science.

His work focuses on cellular defects in chronic heart failure, with a special emphasis on the mechanisms of contractile, mitochondrial and metabolic dysfunction in heart failure.

For his work, Maack received several awards, such as the Albert-Fraenkel- (2014) and the Arthur-Weber Awards (2015) of the German Cardiac Society, respectively. He served on the Board of the Heart Failure Association (HFA) of the European Society of Cardiology (ESC) from 2010-2016, where he served as the Coordinator of the Translational Research Committee (2011-2014) and the Chair of the Basic Science section (2014-2016). In 2018, he entered the Council of the International Society of Heart Research (ISHR), European Section (ES).

Innovative cell-based therapies in the treatment of cartilage defects

Cell based therapies are mainly initiated by academia in the anticipation to develop effective therapies where only unsatisfying therapeutic options exists. Cell-based therapies have highly innovative potential but data on efficacy are limited due to regulatory, financial, and time-consuming aspects.

Taken into account that cartilage itself have a low healing capacity, compensation of cartilage loss by cell-based therapies has been actively performed on the pre-clinical and clinical level. Three products reached EU market authorization however, two of these products are not or only limited available on the EU market. National authorizations under the regulatory conditions of the so-called hospital exemption allow further four treatment alternatives in Germany.

New products with different therapeutic strategies are currently investigated in advanced clinical trials. In the ADIPOA trial MSC-therapy is targeting osteoarthritis the most widespread musculoskeletal disorder in adults resulting in progressive loss of articular cartilage and most frequently in pain and disability. The First-In-Human application of adipose‐derived stromal cells (ASCs) in knee OA was mainly performed under safety aspects to ensure that adverse events are minor and serious adverse events are not related to ASC treatment. Furthermore, preliminary data on efficacy were obtained to support our proposed hypothesis of beneficial effect after ASC treatment. A phase II clinical trial with ambitious design is currently ongoing in five EU member states.

A variant approach addressing focal cartilage defects has been proven in another phase I clinical trial. Innovative potential came from the cell source using nasal chondrocytes purified from nasal septum biopsy. A reduced donor side morbidity increased metabolic and proliferative activity are the major advantages of this cell source. The promising safety profile in the phase I led to the onset of a phase II trial including two arms diverging in short-time maturation versus long-time.

Both trials are representative for the upcoming participation from academics and small biotech companies in the translational field of innovative therapies. However, regulatory expertise and manufacturing in a GMP environment are essential requirements for the successful translational process.

These projects have received funding from the European Union’s Horizon 2020 research and innovation program under grant agreement No. 681103, BIO-CHIP and No 643809 ADIPOA-2.

AIM Biologicals: rethinking targeted therapeutics for autoimmune diseases

AutoImmunity Modifying Biologicals (AIM Biologicals) use a newly discovered mechanism that selectively induces tolerance towards embryonic antigens during pregnancy. This mechanism has been adapted and optimized for therapeutic use in autoimmune diseases. The current aim of the project is to validate the efficacy of AIM Biologicals for multiple sclerosis and neuromyelitis optica and to prioritize candidate molecules for clinical development.

Biologically Vascularized Scaffold for Tissue Generation and Translation

This study demonstrates the capability to generate a vascularized platform technology based on decellularized segments of the rat intestine for a clinically applicable cell-based drug delivery system. The in vitro established mBioVaSc-TERM® indicated promising results in short term in vivo studies while revealing current limitations for the translation into clinical application.

Cell and Gene Therapy – from research to patients

The field of cell and gene therapy is rapidly evolving and provides hope for diseases with high unmet medical need. However, gene and cell therapies constitute a new level of biological and pharmaceutical complexity. Dr Guenther will comment on challenges in the translational process from research to GMP-compliant manufacturing, clinical supply and reimbursement.

Development of light-responsive cancer chemotherapeutics

Our light-responsive chemotherapeutics can be specifically activated to their cytotoxic potential in areas irradiated with blue light. We will present data confirming their cytotoxic potential in vitro as well as preliminary in vivo data. With this light "switching" principle, we are developing these light controlled targeted chemotherapeutics towards the clinic, that would have reduced side-effects to healthy organs compared to conventional chemotherapy.

The end of medicine as we know it

Department of Pharmacology and Personalized Medicine, Faculty of Health, Medicine and Life Sciences, Maastricht University, the Netherlands

Existing drugs fail to provide benefit for most patients. The efficacy of drug discovery is in a constant decline. This poor translational success of biomedical research is due to false incentives, lack of quality/reproducibility and publication bias. The most important reason, however, is our current concept of disease, i.e. mostly by organ or symptom, not by mechanism.

Systems Medicine will lead to a mechanism-based redefinition of disease, precision diagnosis and therapy eliminating the need for drug discovery and a complete reorganization of how we teach, train and practice medicine.

Theranostics - Targeted Radionuclide Diagnostics & Therapy for cancer treatment

Targeted Radionuclide Therapy involves the use of very small amounts of radioactive compounds, called radiopharmaceuticals, to diagnose and treat diseases like cancer. Targeted radiopharmaceuticals contain a targeting molecule and a medical radioactive isotope.

This radio-conjugate is injected into the patient’s body, where it accumulates in the affected organs or lesions. The targeting molecule binds to a tumor-specific receptor or antigen and is absorbed by the tumor cells. The targeting molecule can be used for both diagnosis and therapy – only the radioisotope must be changed.

From cellular immunology to targeted therapies in dermatology

Prof. Christina Zielinski conducts research on the fundamental principles that govern the regulation of human memory T cell responses in health and disease. Through a better understanding of the molecular switches that shape T cell identities, tissue tropism and longevity as well as the T cell dialogue with the tissue microenvironment, she works towards strategies that will provide new diagnostic and therapeutic options for cancer, autoimmunity and chronic infections.

New targets in Immunotherapy

Spontaneous T cell responses against tumor antigens occur regularly in cancer patients. Such responses and associated T cell activity within the tumor tissue impact on tumor cell biology, tumor progression and on patient prognosis. T cell - tumor cell communication has many bi-directional consequences which are determined by multifold signaling pathways on both sides. Novel approaches in cancer immunotherapy aim to exploit these circuits to overcome T cell suppression and tumor cell resistance.

In order to systematically unravel critical signaling cascades during tumor – T cell interactions we apply genome wide gene knock downs in various tumor types and assess their impact on tumor cell destruction by T cells. The obtained results suggest the existence of numerous genes and related pathways that induce immune modulation in T cells and immune resistance in tumor cells and represent novel targets for cancer immunotherapy.

IL-3 as therapeutic target in autoimmune diseases

Various studies in disease models and analysis of human patients indicate that IL-3 could be a highly effective and safe approach for treatment of systemic lupus erythematosus (SLE) and rheumatoid arthritis (RA). We have developed a fully humanized antibody against IL-3 that blocks primary human IL-3 with high efficancy.

IL-3 belongs to the family of hematopoietic cytokines that also includes GM-CSF and IL-5. IL-3 is mainly produced by activated T cells and binds to the unique a-receptor CD123 and the common signal transducing b-receptor CD131. IL-3-receptors are expressed on plasmacytoid dendritic cells, basophils, CD16+ monocytes, activated B cells and endothelial cells. Under basal conditions IL-3 KO-mice have no overt phenotype and no hematopoietic abnormalities, but are more susceptible to infections with parasites.

In recent years IL-3 was identified as important regulator and inducer of autoimmunity and inflammation. In several models (lupus nephritis, collagen-induced arthritis and autoimmune encephalitis) blockade or knock-out of IL-3 markedly improved disease activity including histological and functional parameters. In a sepsis model IL-3 deficient mice did not develop the typical cytokine storm and were largely protected from death.

In contrast, overexpression of IL-3 or injection of recombinant IL-3 aggravated disease activity in all of these models. In healthy rhesus monkeys injection of recombinant IL-3 induced a pronounced inflammation with polyarthritis.

Animal models and human in vitro data show that IL-3 has 4 main mechanisms of action: (1) IL-3 strongly acts on plasmacytoid dendritic cells (pDC), basophils, B cells and monocytes. These cell types play an important role in SLE due to production of type I interferons, IL-4, BAFF, autoantibodies and generation of immune complexes. (2) IL-3 is a central and early inducer / regulator of important proinflammatory cytokines, such as IL-6, TNF and IL-1. (3) IL-3 has pronounced anti-apoptotic effects on IL-3 receptor expressing cells. (4) IL-3 activates endothelial cells and enables leukocyte migration into tissues thus promoting inflammation and tissue destruction.

IL-3 is markedly over-expressed in patients with active SLE, rheumatoid arthritis and multiple sclerosis. Moreover, IL-3 expression correlates with disease activity and was not reduced by currently approved drugs, including biologicals. There is a high medical need for more effective treatments of SLE, beyond cytotoxic drugs and high dose steroids. Also in RA antibodies against single effector cytokines like TNF or IL-6 fail to induce good responses in a considerable percentage of patients. The broad immuno-regulatory properties make IL-3 a promising target for SLE and RA.

We have generated a humanized monoclonal antibody that blocks IL-3 with high potency. This was also confirmed with primary IL-3 from patients with autoimmune diseases. Proposed next steps are GMP-production of the antibody and clinical studies in patients with SLE, including lupus nephritis and RA. First proof of concept in a clinical phase IIa study is estimated to cost about 12-13 Mio Euros and to take about 5 years.

OPSYON – Fusing tumor targeting and immune checkpoint blockade

OPSYON generates new and effective immuno-oncology therapeutics with reduced systemic toxicities. Our technology confines the benefits of immune checkpoint (IC) inhibition to tumor cells, thus providing cancer patients a long-term possibility for progression-free survival and low risk of side effects. To this end, we develop antibody-based therapeutics that specifically bind tumor cells and simultaneously inhibit innate or adaptive ICs. Our lead candidate focuses on the immune checkpoint CD47 in Acute Myeloid Leukemia (AML), an aggressive disease with limited treatment options and a high need for innovative therapies.

Sanofi’s model for R&D cooperation in Translational Medicine Projects

Scientific collaboration between pharmaceutical industry and academia and/or biotech will become more and more important in the future. Patients will require no longer only drugs but individual solutions for their specific problems. Normally, the complete solution cannot be covered by one supplier alone with the consequence that co-operations will be mandatory. Those collaborations have to be built significantly different from the past to create win/win-situations for all stakeholders.

Translational concepts in heart failure research and treatment

The Comprehensive Heart Failure Center (CHFC) in Würzburg integrates basic research on the mechanisms of disease with advanced cardiac imaging and a clinical research unti under one roof with the goal to improve the prevention and treatment of heart failure.

Antigen-specific therapy of hormone receptor-targeting autoimmune disease

Autoimmune antibodies have been recognized to cause heart failure in many patients. The antibodies are directed against beta1-adrenergic receptors and mimic the effect of natural ligands, such as epinephrine (adrenaline). This mechanism leads to a chronic over-stimulation of the receptor and consequently, to heart failure. Valerie and Roland Jahns and Martin Lohse conceived cyclic peptides to treat this auto-immune mechanism at the Rudolf-Virchow Centre at Würzburg. In disease models, the cyclic peptides successfully reversed auto-antibody-mediated propagation heart failure.

The cyclic peptides have already been tested in humans in a placebo-controlled trial, and were shown to be well tolerated - a Phase II study was carried out in patients with chronic heart failure (NCT01391507). This trial was terminated early and converted to a pilot study by the sponsor Janssen (J+J) for strategic, but not for medical reasons. An additional phase IIb study on COR-1 is currently being organized at the German Comprehensive Heart Failure Centre, Univ. Würzburg, which still holds the proprietary rights on the patents (EudraCT 2015-002010-68).

Auto-antibodies may also be directed against thyroid stimulating hormone (TSH) receptors in the thyroid gland, which they activate like the naturally occurring hormone TSH. This mechanism leads to a chronic excess stimulation of these important regulatory receptors and consecutive hyperthyroidism (Graves´ disease). Novel cyclic peptides effectively treat the auto-antibody-mediated propagation of hyperthyroidism. They reduce stimulatory antibodies by immune interference. In disease models, the cyclic peptides successfully improved goiters (thyroid enlargements) and hyperthyroidism and also cured eye symptoms (“auto-immune orbitopathy”) which frequently occur in this condition and are especially hard to treat.

Detection, characterization and monitoring of early and advanced systemic cancer

For decades, primary tumors have been used to guide treatment decision for systemic cancer. Recent data have shown that (i) cancer cells disseminate long before diagnosis; (ii) evolve independently at distant sites and (iii) continue to change under therapy. Therefore, new concepts and technologies are needed that are specifically designed to the needs of systemic cancer.

The Good and the Bad of NER or the Double Edged Sword of DNA Repair

Our understanding of DNA repair mechanisms and the knowledge how to specifically interrupt  these pathways may lead the way to develop new anti-cancer compounds. Nucleotide excision repair (NER) is the most versatile DNA repair mechanism and crucial for the repair of DNA damages induced by UV light.

Several of the proteins involved in this pathway also play a crucial role in transcription. Our analysis of the NER pathway aims to dissect these functions with the goal to specifically interfere with the repair process without affecting transcription.

Rescuing the lost in translation: SPARK Norway and the global SPARK Network

A small percentage of the life science discoveries made in academia are effectively translated into new treatments, diagnostics or improved clinical practice. To address this translational gap the SPARK Program was created at Stanford in 2006.

It helps scientists to turn good ideas into great projects and solutions through education, mentoring and financing. The talk introduces the SPARK approach, and how it is being implemented in Norway as part of a growing international network.