KU Leuven (Leuven, Belgium) is a top university in Europe with a dedicated focus on research and innovation. Reuters ranks KU Leuven as Europe’s most innovative university for the 4th year in a row in the ranking of Europe’s top 100 innovative universities.

Prof. Dr. Karen Vanhoorelbeke is one of the principal investigators of the Laboratory for Thrombosis Research at KU Leuven Campus Kulak, in Kortrijk, Belgium. She obtained her PhD at KU Leuven in 1996 and did a post-doc in Prof Hans Deckmyn’s lab at KU Leuven Campus Kulak Kortrijk. In 2007 she started her own lab at the same university where she is now full professor. In 2010 she did a 10-month sabbatical in the lab of Prof Dr Tim Springer, Harvard Medical School, Boston, US. She was coordinator of the MSCA project PROFILE (Grant Agreement no. 675746, 2015-2019) and is the coordinator of the current MSCA project TOLERATE (Grant Agreement no. 101072729, 2022-2026). In 2022 she received the Esteemed Career Award of the International Society on Thrombosis and Haemostasis (ISTH) (2022).

Her team has expertise in fundamental TTP-related research, in (auto)antibody generation and preclinical TTP animal models. They were the first to generate a reliable animal model of acquired TTP in baboons. This model was a major breakthrough in understanding TTP pathophysiology and was furthermore used to prove the efficacy of an innovative treatment strategy (inhibiting microthrombi formation) for TTP. They perform fundamental research on the mode-of-action of ADAMTS13 in vitro using pioneering tools like in house developed murine monoclonal anti-human and anti-murine ADAMTS13 antibodies and ADAMTS13 mutants. She is/was (co)promoter of 30 PhD students. Her current lab consists of 2 post-docs, 2 PhD students and 2 technicians. DC2 will be hosted in her lab.

Prof. Dr. Karen Vanhoorelbeke
Head of Laboratory for Thrombosis Research, Ku Leuven
Coordinator of TOLERATE Project

Sanquin (Amsterdam, Netherlands) is a knowledge-driven not-for-profit organisation that supplies life-saving products, focusing on the needs of the care sector. Through scientific research, Sanquin looks for and finds new solutions for medical problems in the field of transfusion medicine, hematology and immunology. New fields of research include stem cells and cellular immunotherapy. These studies are conducted in close collaboration with academic centres such as the Amsterdam University Medical Centers and the Leiden University Medical Centre in the Netherlands and abroad.

Sanquin Research is very well-equipped for Biomedical Research in the area of Life Sciences. Current equipment includes a nano-LC LTQ Orbitrap XL ETD and a Orbitrap Fusion Lumos Tribid mass spectrometer, an Airyscan confocal microscope, FACSCanto and FACSCalibur (BD Biosciences), an Imagestream (Amnis, Seattle, USA), a two-photon microscope (Leica SP8 MP) as well as other dedicated lab facilities for hematology research.

Prof. Dr. Jan Voorberg, a leading expert on immune TTP. The Voorberg-lab has pioneered novel and innovative peptide presentation approaches that have allowed for the identification of ADAMTS13 derived peptides that are presented on MHC class II. They have successfully characterized CD4+ T cell responses in patients with immune TTP. The TOLERATE project will allow the team to work closely with both clinical and translational experts on novel approaches for inducing tolerance in patients with immune TTP.

Within TOLERATE, DC5 will work within the Cellular Hemostasis group at Sanquin Research on the development of novel tolerogenic approaches for immune TTP.

Prof. Dr. Jan Voorberg
Group Leader Molecular Hematology, Sanquin

Semmelweis University (Budapest, Hungary) was established in 1752, when Hungary’s legendary Empress Maria Theresa herself founded the university Medical Faculty. SU is the oldest medical school in Hungary, and has more than 250 years of experience in training doctors and medical professionals for their careers.

Each year, over 1,500 students -including English and German speaking students- earn their diplomas from one of the Semmelweis University’s six faculties, namely the Faculty of Dentistry, the Faculty of Health and Public Administration, the Faculty of Health Sciences, the Faculty of Medicine, the Faculty of Pharmaceutical Sciences, and András Pető College, with its Conductive Pedagogic Centre.

Prof. Dr. Zoltán Prohászka is head of the Research Laboratory and professor of immunology at Department of Medicine and Hematology at Semmelwies University.

He received his MD from the Semmelweis University in 1995. After training in experimental and clinical immunology, he worked in the Research Laboratory providing complement diagnostic measurements before completing a fellowship in laboratory medicine. He was appointed head of the Research Laboratory and George Füst Complement Diagnostic Laboratory in 2007 and serves as consultant in complement diagnostics including complement genetics and thrombotic microangiopathies.

The Research Lab with its 6 mentors and 8 themes, where 20 researchers finished their studies and got the PhD degree in the past 10 years, is affiliated to the 'Theoretical and Translational' Doctoral School of Semmelweis University and will host DC8.

Prof dr Zoltán Prohászka
Head Research Laboratory,
Department of Medicine and Hematology Semmelweis University

AP-HP is the largest university medical center in France and in Europe. AP-HP includes 38 hospitals located within Paris and its suburb (population 12 million people), 7 faculties of medicine, 2 faculties of pharmacy, 92.000 professionals including 12.000 doctors and 1500 searchers. AP-HP realizes 40% of French hospital publications and treats 7 million patients per year, with an annual budget of 7 billion euros. The hematology department of Saint-Antoine hospital APHP belongs to the Medical and Academic Department ORPHé (AP-HP.6 – Sorbonne University) and includes the clinical unit for thrombotic microangiopathies (Prof. P. COPPO). The hematology biology department of Hospital Lariboisière APHP belongs to the medical Academic Department BIOGEM (APHP. Nord – Paris Cité University) and includes the ADAMTS13 biologic platform (Prof. A. VEYRADIER).

The French reference center for thrombotic microangiopathies (CNR-MAT - www.cnr-mat.fr) is one among more than 100 reference centers for rare diseases of the country. It was created ex-nihilo in 2006 (qualification by the National Plan for Rare Diseases from the French Health and Science Ministry) with the following organization: Paul Coppo, head coordinator; Laurent Mesnard, associated coordinator; 7 main clinical departments with a very specialized expertise for TMA; 24 satellite clinical departments spread all over the country; 2 central laboratories included in the TMA center: one dedicated to ADAMTS13 (led by A. Veyradier) and one dedicated to the complement system (led by V. Frémeaux-Bacchi and L. Mesnard).

At the national level, the CNR-MAT is involved in the management of more than 2000 patients with various forms of TMA, representing > 11.000 consultations, almost 5000 hospitalizations, resulting in a scientific production of an average of 10 manuscripts per year on journals with an impact factor > 5. The medical activity of Saint-Antoine coordinating center in 2022 consisted in a local cohort of 254 TMA patients, about 300 consultations and roughly 350 recourse advices. The TMA center also coordinates a therapeutic educational program and the official national guidelines. Since 2000, the Lariboisière ADAMTS13 platform has performed roughly 40.000 ADAMTS13 phenotypic assays and 150 genotypic analyses. The French cohort of TTP (French TMA registry) includes ~1700 patients: ~1000 auto-immune TTP (iTTP), ~100 inherited TTP (cTTP) and ~600 acquired TTP of unidentified pathophysiology (uTTP). The TTP biobank includes about 15.000 samples from TTP patients both in acute phase and during their follow-up in remission. The CNR-MAT is also involved in multiple academic (as PI) or industrial (as national PI or co-PI) trials, and belongs to EuroBloodNet, one of the 24 European Reference Networks identified for the representation of specific groups of rare diseases. In France, the CNR-MAT is part of the MARIH consortium (filière Maladies Rares Immuno-Hématologiques), that includes the reference centers involved in other immune-hematologic diseases.

Prof. Dr. Paul Coppo obtained his MD in clinical hematology in 2003, his PhD in Hematology and Hematopoiesis in 2006 and his qualification for research direction in 2008 (Medical school of Sorbonne University). For both his PhD and his post-doc (2004-2006), he was trained in the research laboratory of Prof. W. Vainchenker at Institut Gustave Roussy, Villejuif, France. He has been a professor of hematology since 2010 (Sorbonne University). He shares his time between Saint-Antoine Hospital for clinics and the direction of the CNR-MAT, and the Research institute “Les Cordeliers” (INSERM UMRS 1138, Sorbonne University).

Prof. Dr. Laurent Mesnard obtained his MD in clinical nephrology in 2005, his PhD in 2009 and his qualification for research direction in 2017. He has been a professor of nephrology since 2018 (Sorbonne University). For his post-doc he was trained in the lab of Dr F. Campagne (Cornell University, New York, Institute of Computational Biomedicine. Bioinformatics). Since 2021 he heads the Nephrological Intensive Care Unit, Dept. of nephrology, Tenon Hospital, APHP, and teaches at Sorbonne University. He is associated coordinator of the CNR-MAT, with an emphasis for atypical hemolytic uremic syndrome and genetic explorations. The other members of the team are Raïda Bouzid (team leader of the research team), Anne-Sophie Bretaud, Farida Boukenna, Mohamed Hichour, Jean-François Trebaut (clinical research technicians), and Stella Meimoun (medical secretary).

Prof. Dr Agnès Veyradier obtained her MD in biology-hematology in 1998, her PhD in hemostasis-biochemistry in 2000 and her qualification for research direction in 2004 (Medical school of Paris-Saclay University). For her post-doc (2002-2004), she was trained in the HHMI research laboratory of Prof. JE Sadler in Washington University of Saint-Louis, Missouri, USA. She has been a professor of hematology expert in hemostasis disorders since 2005 (Paris-Saclay and Paris-Cité University). She shares her time between Lariboisière hospital for clinics and management of the ADAMTS13/VWF units, Saint-Louis Research Institute (URP 3518) for research and Medical school of Paris-Cité University for teaching.

Dr. Bérangère JOLY (Pharm.D, PhD) is an Associate Professor of Hematology specialized in hemostasis; she is Prof Veyradier’s right-hand assistant for all projects devoted to ADAMTS13 and TTP. Dr. Nathalie Itzhar-Baikian (MD, PhD) is a Physician in Hematology-Oncology specialized in VWD and other bleeding disorders. The other human resources of the team include Mrs Elisabeth Barbosa (technical coordinator), Mrs Adeline Delton, Hélène Deniau and Chloé Doinel (laboratory technicians), Mrs Maria Mahieu and Sylvie Lavarde (secretaries).

AP-HP’s role in TOLERATE is to identify risk factors associated with morbidity and shortened life expectancy after an acute episode of immune-mediated TTP and identify original strategies to improve life-expectancy in these patients. DC1 will acquire skill on how to manage patients suffering from iTTP, will learn how to make the clinical diagnosis and how to treat and organize an optimal follow-up of these patients. DC1 will gain experience in the management of clinical databases and statistical analyses, and design and execution of works based on registry data.

From left to Right: Mohamed Hichour, Stella Meimoun, Anne-Sophie Bretaud, Jean-François Trebaut, Paul Coppo, Raïda Bouzid, Farida Boukenna

From left to right: Bérangère Joly, Hélène Deniau, Agnès Veyradier, Chloé Doinel, Nathalie Itzhar (Front), Sylvie Lavarde (Behind), Maria Mahieu, Elisabeth Barbosa, Adeline Delton

RCSI University of Medicine and Health Sciences (Ireland) is an independent, not-for-profit institution based in Dublin, Ireland. It aims to improve human health by promoting innovative research that leads to better diagnostics, therapeutics and devices, tackles important healthcare delivery issues, informs policy and clinical practice and enhances the quality of education of healthcare professionals.

RCSI is Ireland’s largest medical school (4,000 students representing >60 nationalities). Globally, it is positioned at #201-250 in the Overall Rankings and 45th in the International Outlook category in the 2022 Times Higher Education (THE) World University Rankings which reflects its global focus and collaboration. It is also ranked joint No.2 in the world by THE for social & economic impact under United Nations’ Sustainable Development Goal (SDG) 3 ‘Good Health and Wellbeing’.

Vascular Biology is one of RCSI’s six strategic research themes. RCSI’s ‘HR Excellence in Research’ award (renewed in 2018) is a recognition by the European Commission of RCSI’s commitment to delivering fair recruitment practices and a supportive and stimulating working environment for researchers. It also holds an Athena SWAN Bronze award in recognition of its positive gender practices.

Prof Dr James O’Donnell received his medical degree from Trinity College Dublin. He completed haematology training in the Hammersmith and Royal Free Hospitals in London. He is a Fellow of both the Royal College of Physicians of Ireland, and the Royal College of Pathologists (UK). Prof O’Donnell is currently Professor of Vascular Biology in the Royal College of Surgeons in Ireland; Director of the Irish Centre for Vascular Biology; and a Consultant Haematologist in the National Coagulation Centre in Dublin.

The Haemostasis Research laboratory led by Prof. O'Donnell has focussed on biochemistry relating to clinical bleeding and thrombosis. It will host DC7.

Prof. James S. O’Donnell MB, PhD, FRCPI, FRCPath
Consultant Haematologist, National Coagulation Centre, St. James' Hospital
Professor of Vascular Biology and Director of Irish Centre for Vascular Biology,
Royal College of Surgeons in Ireland

The team at the Irish Centre for Vascular Biology

The Helmholtz-Zentrum Dresden-Rossendorf (Dresden, Germany) is a member of the Helmholtz Association of German Research Centres. The HZDR pursues interdisciplinary research in the fields of Energy, Health and Matter in Dresden, Freiberg, Görlitz, Leipzig, Schenefeld near Hamburg (Germany) and Grenoble (France).

Prof. Dr. Michael Bachmann is one of two directors of the Institute of Radiopharmaceutical Cancer Research at the HZDR focusing on research and development towards radiolabelled compounds for the functional characterization, therapy, imaging and diagnosis of tumors. The institute also develops immunotheranostics, including bispecific antibody derivatives and cellular immunotherapies, for the therapy and diagnosis of tumors but also autoimmune and infectious diseases. In parallel, Prof. Dr. Michael Bachmann is a professor at the Medical Faculty of the Technische Universität Dresden (TUD) and one of the directors of the National Center for Tumor Diseases (NCT/UCC). He is an expert in the development of bispecific antibody derivatives and chimeric antigen receptor (CAR) technologies for the redirection of immune cells towards tumor cells. He is the inventor of the modular, switchable and flexible CAR platforms known as UniCAR and RevCAR system.

As part of the TOLERATE PhD network, Prof. Dr. Michael Bachmann will act as the main supervisor of the DC3 project entitled “RevCAR-T cell therapy to treat autoimmune disease: iTTP as the model system”. The DC3 is hosted by the Department Radioimmunology of the Institute of Radiopharmaceutical Cancer Research at the HZDR.

The Radioimmunology group has expertise in the development of novel strategies and technologies of antibody-based immunotherapies and cellular immunotherapies as well as diagnostics from bench to bedside. In addition to Prof. Bachmann, the team of supervisors consists of Dr. Anja Feldmann (Head of the Department Radioimmunology) and Dr. Claudia Arndt (Group Leader, Department Radioimmunology).

Prof. Dr. Michael Bachmann
Director of the Institute of Radiopharmaceutical Cancer Research

anicells (Niel, Belgium) accelerates and facilitates the early phase go-to-patient process of cell and gene therapy products. ANI is a spin-off company of the University of Antwerp and the Antwerp University Hospital in Belgium and built on the expertise of people specialized in clinical development and manufacturing of genes, cells and tissue engineering, classified as ATMP in Europe. ANI has a proven track record in the development of technical product dossiers and in the delivery of early-stage cell and gene therapy to the patient.

ANI works closely with cell therapy developers guiding their go-to-patient strategy by offering regulatory support, translating and developing your research into a GM-qualified process, and GMP-compliant manufacturing and QP batch release of Advanced Therapy Medicinal Products (ATMP).

ANI’s facility holds the necessary licenses and GMP accreditation for the manufacture of cell and gene therapy products classified as ATMPs within Europe, including CAR T-cells.

Prof. Dr. Nathalie Cools (Supervisor DC4; 20% commitment) is founder of anicells and has profound experience in early phase development of cell therapy products. Her research has resulted in more than 70 scientific publications in peer-reviewed international journals (WoS citations: 2.079, WoS H-index: 25), and 6 supervised PhDs at UA and 7 ongoing PhD programs. Nathalie Cools has a strong background in cell therapy and immunology, including cellular biology and flow cytometry. Nathalie Cools will supervise DC4.

Prof. Dr. Nathalie Cools
Managing Director, anicells

Ahead Therapeutics (Cerdanyola del Valles, Spain) is a biotechnology company that will put a breakthrough nano-tech platform on the market for treating and curing autoimmune diseases.

In an autoimmune disease, the immune system attacks by mistake our self-tissues. This type of error causes more than one hundred autoimmune diseases that are very well described. Ahead Therapeutics technology fixes this error, re-programming the immune system. It restores the original tolerance (self-tissue recognition). So, the result is that the autoimmune attack stops. There are currently only palliative treatments for autoimmune diseases based on anti-inflammatories and immunosuppressants, all of them producing many side effects. Ahead Therapeutics will go further, offering a curative therapy.

Ahead Therapeutics' approach corresponds to a new knowledge field known as "antigen-specific immune tolerance generation". This is achieved by mimicking a physiological process. The product is a "Liposome" that encapsulates the "specific autoantigen" (a piece of protein) responsible for activating autoimmunity (the disease). The product generates tolerance against the autoantigen, and consequently, it cures the disease. It is a technological platform: only by changing the auto-antigen encapsulated, we can address different diseases. That is, we do not have a single product, but several. We are in the final preclinical phase of several products (“assets”) for different diseases: Rheumatoid Arthritis; Myasthenia Gravis; Type 1 Diabetes; Multiple Sclerosis; Celiac Disease; Myositis and Neuromyelitis Optica.

Marta Vives-Pi, PhD, got her PhD Thesis in Medicine from the Universitat Autonoma de Barcelona. Later on, she was post-doctoral researcher at Germas Trias I Pujol Hospital (Barcelona) funded by the Juvenile Diabetes Research Foundation (New York), and she specialized in the autoimmune reaction in type 1 diabetes. In 2000 she became group leader at IGTP, and Associate Professor of Immunology at the Universitat Autònoma de Barcelona (UAB). Since 2018, she is Scientific Director of AHT. Her current research interests are focused on the understanding of immunology of type 1 diabetes, the loss of peripheral tolerance and the development of immunotherapies for autoimmune diseases. She combines research activities in Autoimmunity (Academia and Industry) with teaching on Immunology. She will be the supervisor of DC6.

Bruna Barneda Zahonero, PhD, MBA, is a professional with more than 4 years in the intellectual property sector, a master's degree in business administration (MBA) and > 12 years of experience in R&D projects in the field of Biomedicine. She holds an Msc and a Ph.D. in Molecular Biology and Biochemistry (UAB), and an Msc in business administration (UPF-La Salle BES). During her scientific career, Bruna carried out biomedical research at different competitive laboratories placed in the UAB, the Bellvitge Biomedical Research Institute (IDIBELL) and Vall d'Hebron Research Institute (VHIR). There, she was engaged in the coordination and management of R&D projects focused on neuroscience, cancer and epigenetics fields, and actively involved in teaching tasks at the University. Before joining AHT, she worked in ZBM Patents & Trademarks as a Business development manager and Patent advisor where she was involved in the dynamization of R&D projects from the initial idea definition and the Intellectual property strategy, to product planning and market uptake. She works as Preclinical & Clinical development director in AHT and will co-supervise DC6.

From left to right: Martí Dalmases, Míriam Salvadó, Bruna Barneda, Marta Vives-Pi, Daniel Maspoch, Sílvia Rodríguez, Antonia Maria Cano