The new class of Advanced Therapy Medicinal Products (ATMPs) is being developed from living cells, tissue compositions or gene vectors at the Berlin Center for Advanced Therapies (BeCAT).
The development starts with basic and technological research.
The goal is the application in a clinical trial.
The focus is on the development of ATMP of all classes in four clinical subject areas for diseases with a high medical need. Participating special fields of the Charité are: Oncology, Immunology, Molecular Biology, Genetics, Transplantation Medicine, Nephrology, Cardiology/Cardiac Surgery and Traumatology.
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Based on the extensive scientific and clinical preliminary work, medical needs and international trends, BeCAT concentrates on four research areas:
(A) Cell-based products for the promotion of endogenous regeneration
(B) Tissue engineering and combination products
(C) Development of cell-based gene therapeutics for gene repair
(D) Development of targeted tumor therapy with immune cells
The four thematic fields are linked via three so-called “technological” research platforms:
(E) ATMP manufacturing process – Development and control
(F) Biomarker – Development and application
(G) Clinical development and regulation
The Working Groups (WGs)
The working groups, consisting of scientists, clinicians, engineers and other experts, are assigned to the various research subject areas and platforms but are linked together in a project-oriented manner.
Thus, technological development, basic science, translation expertise and clinical research are brought together on not only a spatial but also an intellectual level.
- WG Prof. Abou El-Enein
- WG Prof. Blankenstein
- WG Prof. Duda
- WG Prof. Eggert
- WG Prof. Falk
- WG Prof. Kornak
- WG Prof. Kurtz
- WG Dr. Künkele
- WG Dr. Lüders-Theuerkauf
- WG Prof. Mundlos
- WG Prof. Na
- WG Prof. Perka
- WG Prof. Pieske
- WG Prof. Pratschke
- WG Prof. Priller
- WG Prof. Reinke
- WG Prof. Romagnani
- WG Dr. Sabat
- WG Prof. Sauer
- WG Prof. Sawitzki
- WG Prof. Sittinger
- WG Prof. Schmitt
- WG Prof. Spranger
- WG Prof. Stamm
- WG Prof. Tschöpe
- WG Prof. Uckert
- WG Prof. Volk
- WG Prof. Willimsky
- WG PD Winkler
WG Prof. Abou El-Enein
Charité - BCRT (Junior Research Group, Physician)
The WG will coordinate the research Platform G "Clinical Development and Regulation" in cooperation with the clinical PIs. It has already established a large national and international network with academic and official institutions (PEI, EMA) for the further development of ATMPs' regulatory and health economic guidelines. On the one hand, effective regulatory implementation strategies will be developed, especially for the first application of new and increasingly complex ATMPs in patients. On the other hand, it is also important to consider the manufacturing processes from a health economic point of view as early as possible and to set the course for this in good time.
Contact Professor Abou El-Enin
Tel. +49 (0)30 450 539 594
WG Prof. Blankenstein
Charité - Immunology (Institute Director, Natural Scientist)
The WG will coordinate the research focus of "T-cell engineering" in the research field D "Targeted tumor therapy with immune cells" primarily from a preclinical and methodological point of view. The focus will be on the T-cell receptor engineering of tumor-specific T cells, which will be generated with the help of the humanized mouse platform established by the WG for the first time worldwide.
An important aspect is the research into the synergy of approaches directed precisely against tumor cells (lysis of tumor cells) with those that address the tumor microenvironment (tumor stroma) that protects the tumor.
Contact Professor Blankenstein
WG Prof. Duda
Charité - Julius Wolff Institute/BCRT (Institute Director, Engineer)
The WG has a strong research focus on the biomechanical aspects of the restoration of tissue homeostasis especially after trauma or other events. The attraction, proliferation and differentiation of stem/progenitor cells and, thus, the tissue composition can be manipulated through targeted biomechanical manipulation of natural or synthetic matrices.
A further research focus is on understanding and influencing early healing processes after trauma of the musculoskeletal system with a focus on angiogenesis and inflammation. Large animal models that represent the biomechanics of the muscle/bone system are well established.
Within BeCAT, the WG will focus on the further development of regenerative cell therapies and matrix/tissue interactions (subject areas A+B) and coordinate the implementation of tissue/matrix 3D printing (subject area B).
Contact Professor Duda
WG Prof. Eggert
Charité - Pediatric Hematooncology (Clinical Director, Medical Doctor)
The clinical and scientific focus of the WG is on childhood tumors, with the prognostically particularly unfavorable neuroblastoma at the center of research. National and international networks for therapy optimization of neuroblastoma have been established and numerous clinical studies initiated.
An increasing focus is on the development of immune cell-based approaches for the therapy of neuroblastomas and also other pediatric tumors, such as lymphomas (subject area D).
In addition, approaches for the gene therapy of congenital rare diseases of hematopoiesis will be developed and implemented in the active clinical hematopoietic stem cell transplantation program (subject area C).
The extensive clinical study expertise flows into Platform F (Clinical Development).
Contact Professor Eggert
WG Prof. Falk
Charité/DHZB - Cardiac Surgery (Clinic Director, Physician)
The clinic is one of the leading international cardiosurgical clinics.
The development of new next-generation technologies for risk-minimized and sustainable functional reconstitution in cardiac surgery is the central concern of the research work of the WG. Hybrid ORs allow near-to-patient OPs on large animals. The development of composite products for valve replacement and heart function stabilization/improvement as a conservative approach or in connection with apparatus-based heart function support ("artificial heart") or cardiovascular operations will be the focus of interest within the framework of BeCAT.
The extensive clinical study expertise flows into Platform F (Clinical Development).
Contact Professor Falk
WG Prof. Kornak
Charité - Genetics/BCRT (Junior Research Group, Physician)
The research focus is on genetic diseases of the musculoskeletal system and includes genetic diagnostics and consultation of patients as well as the development of gene therapeutic approaches, e.g. for osteopetrosis.
There is extensive experience in gene typing, ontology, preclinical malfunction models and non-viral gene transfer into adult stem cells to develop cell-based gene therapies (Subject area C).
Contact Professor Kornak
WG Prof. Kurtz
Charité - BCRT (Natural Scientist)
The WG deals with the standardized collection and administration of extensive data on the origin, characterization and functionality of pluripotent stem cells and their derivatives (Cellfinder database), which are implemented and coordinated on an international level (Data Registry of the European Bank of iPSC; IMI project). In addition, the WG's own research focuses on the generation of human kidney organoids from iPSC, which has progressed well in recent years.
Both expertises will be incorporated into the BeCAT concept (Platform F and subject area B).
Contact Professor Kurtz
Tel. +49 (0)30 450 539 424
Tel. +49 (0)30 450 539 428
WG Dr. Künkele
Charité - Pediatric Hemaoncology (Junior Research Group, Physician)
The research focus of the WG is the development of T-cell therapy, especially for solid tumors in childhood, with an initial focus on CAR-T cells for targeted neuroblastoma therapy (Subject area D).
New concepts could be developed from the experiences of the first clinical pilot studies and are now to be implemented in the sense of an iterative improved approach.
Biomarker studies should help to stratify responders/non-responders and to understand the reasons for the different response rates (Platform F).
Contact Dr. Künkele
WG Dr. Lüders-Theuerkauf
DHZB - Tissue Engineering Lab (Junior Research Group, Natural Scientist)
The focus of research interest is the establishment of new tissue engineering approaches for the replacement of heart valves. In close cooperation with the TU Berlin, the first hopeful experiences with the use of matrix 3D printing and subsequent colonization with cells for a new class of functional heart valves have been achieved - this will also be the focus of research within BeCAT (Subject area B).
Contact Dr. Lüders-Theuerkauf
WG Prof. Mundlos
Charité - Genetics/BCRT (Institute Director, Physician)
The WG is internationally recognized for the application of state-of-the-art molecular biological and bioinformatic techniques for the clarification of rare diseases, especially in musculoskeletal development. The latest results for the identification of interactions between chromate structures, also known as topologically associated domains (TADs), using chromosome conformation capture methods (capture Hi-C and 4C-seq) are groundbreaking. TADs regulate the contact of enhancer regions with their target genes. The formation of mutant neo-TADs can be the molecular cause of altered gene regulation and malformation phenotype. This leads to new approaches in gene therapy (Subject area C).
However, the various NGS technologies are also an essential basis for product characterization (Platform E) and biomarker analysis (Platform F).
Kontakt Professor Mundlos
WG Prof. Na
Charité - Hematooncology (Junior Research Group, Physician)
The research focuses of the WG are new preclinical models for the optimization of tumor-specific T-cell therapy (Subject area D) and the analysis of mechanisms and approaches for the promotion of immune reconstitution after hematopoietic stem cell transplantation, particularly the restoration of bone marrow niches for various immune cells through combined cell therapies (Subject area A).
Contact Professor Na
WG Prof. Perka
Charité - Orthopedics (Clinic Director, Physician)
The regeneration of muscular damage, particularly with/without accompanying bone fractures, is the focus of the research interest.
Cell therapeutic approaches with mesenchymal or other stromal cells are pursued which promote angiogenesis and muscle regeneration directly via their secretome or indirectly via immunomodulatory, anti-inflammatory effects.
Another focus is the development of personalized matrix products using 3D printing (Subject areas A+B).
Contact Professor Perka
WG Prof. Pieske
Charité - Cardiology (Clinic Director, Physician)
An internationally recognized research focus of the clinic is the cardiac insufficiency of ischemic and non-ischemic genesis. To this end, new diagnostic methods are being developed, especially for the stratification of patient groups and new therapeutic approaches.
The BeCAT concept focuses particularly on the development of novel stents with cellularization (endothelial cells) on biocompatible materials with targeted cell adhesion-promoting coatings, as the classic drug-eluting stents have many limitations, e.g. inhibition of protective endothelialization (Subject area B).
Contact Professor Pieske
WG Prof. Pratschke
Charité - Surgery (Clinic Director, Physician)
A clinical and internationally visible focus of the clinic is liver surgery, from partial resection (tumor, etc.) to organ replacement by transplantation. Hepatocyte function, regeneration and replacement are, therefore, also the focus of research.
Cell-based therapies for the promotion of liver regeneration after partial resection (Subject area A) and by direct liver cell replacement in malfunction (Subject area C) are to be developed and immune cell-based liver tumor therapies established (Subject area D) within the framework of the BeCAT concept.
Contact Professor Pratschke
WG Prof. Priller
Charité - Neurology/Psychiatry (Senior Physician, Physician)
The interaction between the regeneration of neuronal cells and brain-specific stroma reactions (microglia, immune cells) is increasingly becoming the focus of interest, not only in neurological autoimmune diseases, such as multiple sclerosis, but also in other chronic neurological and neurodegenerative diseases.
One focus of the WG's research is the generation of patient-specific iPSC lines for ex vivo disease models to gain a better understanding of the pathogenesis of defined neurological and psychiatric diseases.
Cell therapeutic approaches with myeloid progenitor cells for application in neurodegenerative diseases (Subject area A) are another research focus.
Contact Professor Priller
WG Prof. Reinke
Charité - Nephrology/BCRT (Founding Director BeCAT, Medical Doctor)
The focus of interest is the best possible treatment of the onset of, respectively, manifest chronic renal insufficiency.
The scientific spectrum ranges from the further improvement of the results of the internationally highly regarded kidney transplantation program through biomarker-based personalized immunosuppression (platform F), improved management of infectious and/or virus-associated malignant complications with antiviral T cells (Subject area D) and new tolerance-promoting cell therapy approaches to indirect (stromal cell therapy) or direct kidney regeneration (with iPSC-generated kidney organoids). The aim of these scientific concepts is to delay or even prevent the progression of progressive renal insufficiency of the most varied genesis (Subject areas A+B).
The WG has extensive experience in investigator-initiated ATMP developments of bench-to-bedside (four cell products in clinical testing so far).
Prof. Reinke heads the GMP unit of the BCRT and is the "qualified person" for the manufacturing processes approved there. The extensive clinical study expertise flows into Platform F (Clinical Development).
Contact Professor Reinke
WG Prof. Romagnani
Charité - Gastroenterology (Junior Research Group, Natural Scientist)
The WG has generated important new data on Innate Lymphoid Cells (ILCs) in the human system and their regeneration after hematopoietic stem cell transplantation.
Depending on the environment, ILCs can become active both as (un)desired effector cells and regeneration-promoting cells (especially ILC 2+3). A targeted manipulation for the application desired can become possible with the increasing understanding of the molecular mechanisms of regulation of this diversity.
On the one hand, the aim is to generate ILC-based cell products for anti-inflammatory/proregenerative effects, e.g. in inflammatory diseases (Subject area A) and, on the other hand, to increase their effector effect in tumor therapy (Subject area D).
Contact Professor Romagnani
WG Dr. Sabat
Charité - Dermatology/Immunology (Physician)
The interdisciplinary WG represents a bridge between dermatology and immunology and stands internationally for the pathogenesis research of inflammatory skin diseases.
Different scientific aspects are dealt with, such as inflammatory, regenerative (e.g. IL-22) and local bactericides and antivirals.
BeCAT aims to develop and clinically test cell therapeutic approaches (regulatory T cells; genetically modified cells that promote regeneration) for severe inflammatory dermatoses of different origins (Subject area A). These studies will be accompanied by extensive biomarker analyses, particularly in skin biopsies, for which the group is also highly visible internationally (Platform E).
Contact Dr. Sabat
WG Prof. Sauer
Charité - Surgery (Physician)
The main focus of research is the therapy of liver failure. To this end, bioreactor systems for long-term liver cell cultivation have been established, the technique of liver decellularization for matrix generation has been optimized, genetically modified hepatocytes have been generated, and preclinical small and large animal models have been developed for testing new therapeutic approaches.
New cell-based approaches with pro-regenerative and anti-inflammatory capacity for improved regeneration after partial liver resection (Subject area A) are to be developed within the framework of BeCAT.
In addition, approaches are being developed to improve the effectiveness (engraftment) of liver cell transplantation by combining products with matrix (Subject area B).
Contact Professor Sauer
WG Prof. Sawitzki
Charité - Medical Immunology (Natural Scientist)
The focus of the WG is, on the one hand, on molecular mechanisms of immunotolerance of T cells and, on the other hand, on the development and validation of gene expression and flow cytometric biomarkers especially for cell-based therapies.
The group has extensive experience in multicenter biomarker analyses in international networks. This expertise will be applied to Platform E within the BeCAT framework.
Contact Professor Sawitzki
WG Prof. Sittinger
Charité - BCRT/Rheumatology (Natural Scientist)
The WG has many years of experience in tissue engineering and has led several products in cartilage and bone regeneration indications to market maturity in spin-off companies. A current research focus is the development of a therapy for heart failure with a new, IP-protected, heart-specific stromal cell type (CardAP). The entire preclinical phase of the autologous therapy has already been completed and is about to enter Phase I. At the same time, a more user-friendly allogeneic off-the-shelf therapy will be developed (Subject area A).
Contact Professor Sittinger
WG Prof. Schmitt
Charité - Hematooncology (Senior Physician, Physician)
A central research focus of the WG is the analysis of the senescence of tumor cells, especially under therapy. Senescent tumor cells that have survived conventional antitumor therapies practically no longer proliferate but have a high energy requirement. They represent a basis for possible tumor recurrences and are, therefore, an interesting new therapeutic goal (Subject area D). Their analysis in the monitoring of antitumor cell therapies could serve as valuable biomarkers (Platform F).
In addition, the WG deals with virus-associated lymphomas and the development of targeted immune cell therapies (Subject area D).
Contact Professor Schmitt
WG Prof. Spranger
Charité - Endocrinology (Clinic Director, Physician)
The clinical and scientific focus of the WG is on obesity and type 2 diabetes.
The recently formed interdisciplinary team "Immunometabolism" (Endocrinology/Medical Immunology) focuses on the interactions between metabolic and immunological changes, particularly the influence of the microbiome and the related adaptive immune system on type 2 diabetes. It has been shown in numerous dietary therapeutic approaches that, despite obvious metabolic improvement, a large number of disorders cannot be corrected.
In addition to cell-based approaches to immune regulation and the treatment of late complications of type 2 diabetes, the targeted modification of the microbiome with living bacterial cells is the focus of the BeCAT projects (Subject area A).
Contact Professor Spranger
WG Prof. Stamm
Charité/DHZB - Cardiac Surgery/BCRT (Senior Physician, Physician)
Cell-based therapies to promote the regeneration of cardiac muscle performance, particularly after ischemic attacks, have been the focus of research at the WG for many years. Internationally acclaimed, controlled studies on the application of hematopoietic precursor cells for heart muscle regeneration have been initiated, which, for the first time, have also shown the limitations of these methods.
The focus is currently on transepicardial regeneration, which is to be induced by the implantation of iPSC-generated cardiomyocyte precursor cells on polymer-based matrices (composite product) (Subject area B).
Contact Professor Stamm
WG Prof. Tschöpe
Charité - Cardiology/BCRT (Senior Physician, Physician)
The main interest of the clinical and scientific work of the WG lies in regenerative therapy approaches for diagnostically - using new biomarkers - grouped subtypes of chronic heart failure. A particular focus is on the undesired inflammatory aspects of different geneses (DCMi).
Projects on biomarker development, selection and monitoring of ATMP in patients with chronic heart failure (Platform F) and on cell-based therapy approaches (Subject area A) are being worked on within the framework of BeCAT.
Contact Professor Tschöpe
WG Prof. Uckert
MDC/HUB - Molecular Biology (Natural Scientist)
The generation of viral and increasingly also non-viral vectors for the gene modification of antigen-specific T lymphocytes is the focus of the WG's work.
The correct combination of the desired tumor-specific T-cell receptor chains in transduced T cells and extensive safety tests for the selection of undesired (autoreactive) specificities have been solved very well technically.
In cooperation with other working groups, BeCAT will focus on the design of non-viral vectors with selected T-cell receptors or chimeric receptors (CAR) for adoptive T-cell therapies for various purposes, such as regulation, tumor killing, virus control (Subject areas A+D) and their exact characterization (Platform E).
Contact Professor Uckert
WG Prof. Volk
Charité - Medical Immunology/BCRT (Institute Director, Physician)
The WG has extensive, internationally recognized experience in the development, validation and analysis of immunological biomarkers for the monitoring of new immune and cell therapeutics.
Biomarker analyses have been performed in the accredited FDA/EMA-compatible study laboratory in more than 50 Phase I/II studies for new therapeutics in recent years. Numerous tests were transferred to the routine diagnostics of the Charité in the Berlin laboratory or commercialized (JPT, Miltenyi Biotech, Beckmann-Coulter, Becton-Dickinson, Siemens, AID, Milenia Biotec).
The focus in the field of cell therapy is on MSC and T-cell therapies to support endogenous regeneration (Subject area A), combined with intensive cell product analysis (Platform D) and immune monitoring of treated patients (Platform E).
Contact Professor Volk
WG Prof. Willimsky
Charité - ECRC/Tumor Immunology (Junior Research Group, Natural Scientist)
The WG focuses on experimental and translational tumor immunotherapies.
The main focus is on the identification of tumor-specific (neo)antigens and the genetic generation of specific T cells directed against them. New preclinical in vitro and in vivo models for the testing and validation of tumor neoantigen-specific T cells have also been established.
The plan is to extend these models using CRISPR/Cas technology for efficacy and safety testing of genetically modified tumor-specific T cells and their further development for therapeutic application (Subject area D).
Contact Professor Willimsky
WG PD Winkler
Charité - Orthopedics/BCRT (Junior Research Group, Physician)
The WG has extensive experience in the use of mesenchymal cell therapies for muscle regeneration in preclinical trauma models and initial clinical trials.
Scientific immunological and molecular-biological analyses accompanying the studies provided information on the correct cell product selection, dosage and mode-of-action. This is the basis for further therapy development (Subject area A). The effectiveness is to be further increased by combining natural/synthetic matrix with cells (Subject area B).
Contact PD Winkler