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MEDICINE

RESEARCH

TECHNOLOGY

REGENERATIVE THERAPIES

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REVIEW & OPPORTUNITIES

Berlin-Brandenburg Center for

REGENERATIVE THERAPIES

2 3Welcome

Demographic transition and lifestyle changes have led to the increas-ing prevalence of chronic diseases amongst all age groups as well as physical traumas that particularly af-fect mobile, elderly patients with dete-riorated regeneration capacity. These increased medical needs pose a seri-ous challenge to our healthcare system and its financial resources. In order to adequately address these needs, Re-generative Medicine aims to transform the current focus on “treatment of disease” into one that concentrates on “regeneration of health”. As such, we hope to support an approach that will sustainably improve patients’ quality of life and decrease the economic bur-den placed on the healthcare system.Regenerative Medicine is a relatively new and rapidly expanding field. Its goal is to restore the function of im-paired cells, tissues and organs by stimulating endogenous regeneration and repair mechanisms or by provid-ing laboratory-grown biological re-placements. The pursuit of these two concepts – “biological replacement” and “stimulation of endogenous re-generation” – has given rise to exten-sive basic science research, resulting in encouraging first clinical applications.A major challenge that Regenerative Medicine currently faces is the fast and efficient translation of basic re-search results and technological devel-

opments into clinical practice. This is confounded by the fact that academic and clinical investigations are largely separated from industry-driven re-search and development. As a result, numerous promising projects fail to receive the cost intensive support that could lead to further clinical advance-ment or they remain unnoticed due to inadequate selection criteria, which in turn leads to high failure rates in the costly product development process.Translational centres can help to tack-le these shortcomings, and thus, play a crucial role in bridging the develop-ment gap. Their goal-oriented struc-tures and processes are designed to substantially de-risk research projects, thereby ensuring that clinical settings have swift access to improved treat-ments.Based on these premises, the Berlin-Brandenburg Center for Regenera-tive Therapies (BCRT) was founded in 2006 as a transdisciplinary translational clinical centre. It is jointly operated by the Charité – Universitätsmedizin Berlin and the Helmholtz-Zentrum Geesthacht. Over 250 employees – among them physicians, scientists, en-gineers, experts in translational medi-cine, graduate students and technical staff – are employed at the BCRT´s two sites: the Campus Virchow-Klini-kum of the Charité, with its close links to the BCRT partner clinics and the

Institute of Biomaterial Science based at the Teltow Campus of the Helm-holtz Zentrum Geesthacht.

This impressive partnership has flour-ished for over 10 years and looking back, we can proudly acknowledge that the founding vision, namely to create a transdisciplinary translational centre was indeed a ground-breaking venture. A decade later, the profound fruits of this enterprise are clearly vis-ible and the BCRT can now boast an excellent international reputation. In-deed, together with its international partners, the BCRT is a leading player in the field of Regenerative Medi-cine. As a result, BCRT-led research has contributed substantially to the development of novel therapies and products, which in turn have shaped the healthcare landscape as we know it. This brochure highlights several quintessential examples of the BCRT’s vision in action.

To summarize, the BCRT is a strate-gic, innovative joint venture that suc-cessfully integrates and utilises the strengths of its founding members, thus facilitating swift translation of research results into real benefits for patients and healthcare players alike. We sincerely hope that you will enjoy reading this brochure.

Prof. Dr. Karl Max Einhäupl, MD Chief Executive Officer Charité – Universitätsmedizin Berlin

Prof. Dr. Axel Radlach Pries, MD Dean Charité – Universitätsmedizin Berlin

Prof. Dr. Wolfgang Kaysser Scientific Director Helmholtz-Zentrum Geesthacht GmbH

contents

Welcome

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mission statement

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RegeneRative medicine

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ReseaRch aReas

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milestones

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Refined tRanslation

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success stoRies

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netWoRking and impact

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facts and figuRes

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„Fully realising the potential of regenerative medicine requires patience and the courage to forge a new path.“

translation – these three “T” are the key principles. Over the last decade, we have developed novel concepts and initiated multiple clinical trials. Products derived from our research have broken into the market and are benefitting patients.

The success of the BCRT rests upon close partnership with a range of clin-ics and academic institutions, a na-tional and international network, and collaboration with industry. We are grateful to all of them.

Special thanks go to our staff, whose commitment and courage fill the con-cept of the BCRT with life and ener-gy, and to the managements of both the Charite – Universitätsmedizin Berlin and the Helmholtz-Zentrum Geesthacht, as well as our external funding bodies.

Prof. Dr. Hans-Dieter Volk, MDDirector and Spokesman BCRT

Prof. Dr. Georg N. DudaDeputy Director BCRT

Prof. Dr. sc. nat. Andreas LendleinDeputy Director BCRT

collaborative research, regenerative cures

The Berlin-Brandenburg Center for Regenerative Therapies (BCRT) is the place where medical science, basic re-search and technology meet. Our cen-tral aim is to investigate endogenous regeneration mechanisms of the hu-man body and to translate these find-ings from the laboratory bench into clinical practice. We ensure that the innovative approaches of Regenera-tive Medicine reach patients speedily.

The starting point of our work is the investigation of endogenous regenera-tion mechanisms of the human body with the help of patient samples and novel, clinically relevant model sys-tems. We thereby strive to answer a set of seemingly straightforward ques-tions: How does the body heal itself after an injury or during a disease ? What sets “good” and “bad” healers apart ? It is these processes that we are interested in, with the ultimate goal of exploiting and influencing them. Im-portantly, Regenerative Medicine ex-tends beyond stem cell therapies.

The stimulation of endogenous re-generation is the cornerstone of our work. Such stimulation can be achieved through biomaterials, cells or biologi-cally active factors as well as combi-nations of all three. We have already made progress in regulating inflamma-tion and, particularly after injuries, the inhibition of scarring and the re-for-mation of an important support tissue called the extracellular matrix. Simi-larly successful is the development of

a range of biomarkers, which puts per-sonalized medicine within reach. Much of our work relies on the development of targetable drug-release systems, canny combinations of biomaterials and cells as well as finding ways to stimulate and regulate vascularization, which forms the basis of a successful healing process.

A major effort is the efficient and fast translation of research findings from the bench to the bedside, which means our ultimate yardstick for successful translation is reaching a first-in-man trial. To accomplish this challenging task, we have chosen a deeply collab-orative and transdisciplinary approach. At the BCRT, specialists from medical and natural sciences and from engi-neering collaborate not only beyond the classical boundaries of their dis-cipline, but also beyond the tradition-ally narrow definitions of diseases. To make it work, we have created open labs, facilitated interaction between disciplines and incentivised staff to undertake collaborative projects. Our adapted infrastructure supports these projects through a number of core units. They ensure the standardisation of a range of processes and, in unique fashion, allow us to manufacture therapeutics and medicinal products in-house under good manufacturing practice for first-in-man clinical trials.

All clinical trials are monitored by as-sociated scientists and accompanied with intense scientific research to

elucidate the underlying mechanisms of individual responses. This effort is crucial to optimise our diagnostic markers and the results are fed back into an iterative cycle of continuous improvement. We have termed this shuttling between bench and bedside “refined translation”. This concept is at the core of our ambition to de-risk the development of better diagnoses, therapies and medicinal products.

Our pioneering approach is at the leading-edge of translational research and constitutes a paradigm shift. It does not only require innovative think-ing and forward-looking behaviour of our researchers, but also of fund-ing bodies. Required are fresh ideas about how to benchmark and value the collaborative development of di-agnostic tools, advanced therapies and medicinal products – from inception to implementation in the clinic. We have been at the forefront of debat-ing these issues, particularly in an in-ternational context at the “TRANS-LATE!” forums, and jointly published positon papers with experts from vari-ous countries that offer guidance on the way forward. We stay abreast of those changes and shape the training of our junior scientists at the Berlin-Brandenburg School of Regenerative Therapies (BSRT) according to our transdisciplinary approach.

We are delighted to look back on 10 successful years for the BCRT. Transdisciplinarity, technology and

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What kind of advanced medicines does Regenerative Medicine develop ?

Medicinal products help to heal

SUBCELLULAR LEVEL Biologically active factors are employed to initiate, stimulate and regulate regenerative pro-cesses or cellular interactions. The analysis of genetic materi-al and soluble factors opens up new lines of inquiry that often lead to new therapies.

CELLSCells are the cornerstone of many approaches in regen-erative medicine. They are versatile and reliable tools to stimulate and support the res-toration of tissues, control re-generative processes and also to directly replace functionally impaired cells.

TISSUESTissue structures can be built with the help of natural or syn-thetic biomaterials and cells. These tissues can support the regeneration of organ func-tions, for example heart valves, cartilage and vessels.

Scientific progress over the last years has led to fundamentally new classes of drugs: Tissue - engineered products, somatic cell and gene therapies as well as combinations of these classes. They are summarised under the umbrella term Advanced Medicinal Therapy Products (ATMP) and subject to specific

regulatory requirements, which take the complexity and novelty of these novel products into account. On the EU-Level, regulatory requirements and procedures for market authorisation have been har-monised, which ensures that the latest medicinal products reach patients faster.

Regenerative processes often start slowly. Bio-material-based prod-ucts can accelerate the healing process, for example as implants that controllably release drugs or as scaffolds that cells can populate.

What is a biomarker?

A biomarker is a measurable indicator of a specific cel-lular, molecular or genetic state of a patient, and thus, a powerful diagnostic tool. In Regenerative Medicine, biomarkers are important to predict therapeutic suc-cess and are therefore fundamental in making precision medicine a reality. Biomarkers help to determine the efficacy and the safety of novel therapies and, because of their functional link with the underlying condition, can reveal new therapeutic targets.

Precision medicine

Precision medicine describes an approach to health-care, in which the selected treatment concept takes the individual characteristics of each patient into account. This approach is also labelled individual medicine, be-cause it makes it possible to combine patient-specific effectiveness and tolerance of a therapy. Similarly, the progression of the healing process can be tracked indi-vidually and precisely, which allows for prompt adap-tions in case they become necessary. For all the above, precision medicine is a particular focus of Regenerative Medicine.

ORGANS Organ function is restored through regeneration or bio-logical replacement. We also work on lab-grown organs to safely test new drugs in a more realistic environment or to pos-sibly use them as organ replace-ments in the future.

MEDICAL NEEDWhen acute or chronic diseases, wear or trauma affect body functions, traditional medicine treats symptoms and, if possible, the un-derlying causes. A full recovery and a complete regeneration of the function is rarely achieved. Continuous or repeated treatment is often necessary, burdening the patient with a range of side effects and the healthcare system with high cost. Regenerative Medicine responds to that challenge by developing curative therapies that regenrate health. This novel approach aims at providing new thera-pies for unmet medical needs and at driving down the cost of treatments, but foremost at sustainably improving patients’ quality of life.

What is regenerative medicine?The goal of Regenerative Medicine is to develop therapies that regenerate injured or functionally impaired cells, tissues and organs. This is achieved through the stimulation of endogenous regenerative processes or through biologically compatible replacements.

Key strategies: Endogenous regeneration or biological replacement

The stimulation of endogenous, i.e. the body’s natural, regeneration is a crucial strategy of Regenerative Medi-cine and the primary research focus of the BCRT. We work with a set of “regenerative tools”: certain cell types, biologically active factors, biomaterials or combi-nations thereof. These agents can be employed to regu-late inflammation, to mobilise stem cells or to stimulate vascularisation. The most recent approaches of “living” medicaments are not limited to human cells, but explore the use of microorganisms. An alternative approach of

regenerative medicine aims – in case of most serious damage or lack of regenerative capacity – at replacing tissues and even whole organs with lab-grown material. While artificial skin (particularly after burns), cartilage and heart valves are routinely used today, replacing increas-ingly complex structures like the bladder or oesophagus remains a challenge. But considerable progress has been made and those efforts have spawned the establishment of miniaturised organ models, which are useful in drug testing.

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Prof. Dr. Michael Sittinger In situ tissue engineering

Our field integrates knowledge from cell biology and material science to develop “living” combination products. These prod-

ucts include biocompatible transplants together with cells or bio-logically active agents. They can be easily integrated into diseased tissue to stimulate regeneration. Major areas are the regeneration of bone and cartilage, including the spine.

Prof. Dr. Stefan Mundlos, MD Molecular analysis and cell engineering

Understanding the molecular basis of functional and structural features of cells in health and disease is a fundamentally important task. We therefore

drive the development of powerful analytical tools that identify such underlying molecular characteristics. These insights enable researchers to pursue new approaches in regenerative medicine, employing, e.g. the latest technologies in molecular biology to precisely target genetic modifications of cells, repair defective genes and generate pluripotent stem cells.

Prof. Dr. Andreas Lendlein Polymer-based biomaterials

Drawing on our growing knowledge about the structure of biological materials, we design and investigate new polymeric

materials and their functions. We strive to stimulate endogenous regeneration and to temporarily replace tissues or organ functions with a new generation of biomaterials. For this purpose, structured polymers and novel drug release systems can be tailored to the specific needs of the application.

COLLABORATIVE RESEARCH, REGENERATIVE CURESRegenerative Medicine pursues innovative, curative approaches, which require the collaboration of experts from the fields of medicine, science and engineering. It is by working together that we overcome the traditionally narrow view of diseases. To facilitate such transdisciplinary thinking in our daily lives, we have established open lab structures and set incentives for cross-field projects. This sense of collaboration has been infused into the BCRT on all levels and helps to bring the pioneering approaches of Regenerative Medicine faster from the bench to the bedside.

Cardiovascular system Prof. Dr. Carsten Tschöpe, MD

Novel diagnostic tools, e.g. biomarkers, help us to finely differentiate dis-eases of the heart muscle and, as a consequence, allow the application of precisely targeted regenerative therapies. As the regenerative capacity of the heart muscle is, unfortunately, very low, we also work on growing heart tissue in the lab to reduce the need for organ transplants in the future. Moreover, we develop cell therapies to treat peripheral artery occlusive disease (PAOD) that can stimulate the regeneration of muscle tissue and promote vascularization.

Musculoskeletal system Prof. Dr. Georg N. Duda

Most bone fractures are a prime example of endogenous regenera-tion as they heal without difficulty. 15% of patients, however, experi-ence delayed or incomplete healing. To identify these patients at the time of first treatment, we develop diagnostic tools. We then can im-mediately apply our regenerative therapies, which are based on cells, bioma-terials and biologically active factors. More complex is the regeneration of muscle and tendon injuries, but early results from cell therapy studies indicate that even the delicate skeletal muscle can be regenerated. As mechanobiological aspects are pivotal in the musculoskeletal system, we will integrate regenerative therapies with medical technology products, such as implants.

Immune system Prof. Dr. Petra Reinke, MD

The immune system maintains the body’s integrity by fighting exogenous (pathogens, germs) and endogenous (tumours, tissue homeostasis) challenges. We discovered through biomarker studies that the “immunological age” of a patient does not necessar-ily correspond to the real age and that it has profound influence how well endogenous regeneration works. Undesirable immune reactions may not only lead to autoimmunity or preclude organ transplants, but they can also interfere with endogenous regeneration. Based on these findings, we have adapted our animal models to reproduce disease patterns of immune aged patients more truthfully. We have developed cell therapies, in which we enrich and expand desirable T lymphocyte populations. These biomarker-driven immunomodulation therapies ad-dress a range of medical needs.

in Which research Fields are We active?Research at the BCRT spans three clinical research fields: diseases of the immune system, the musculoskeletal system and the cardiovascular system. They are closely connected with our three technology-driven research fields: molecular analysis and cell engineering, polymer-based biomaterials and in situ tissue engineering.

REGENE RATIVE

THER APIES

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The Berlin-Brandenburg Center for Regenerative Therapies celebrates its 10th anniversary.

In collaboration with the Journal “Science Translational Medicine”, the international symposium “Translate!” is held for the first time to discuss challenges and opportunities in translational medicine.

The World Technology Evaluation Center evaluates the BCRT and describes it as a flag-ship project in translational research.

Following refurbishment, the former dental clinic on the Campus Virchow becomes the “Institutsgebäude Süd” and on the Campus Teltow construction work on the “Biomedi-zintechnikum II” is completed. With these new buildings, the BCRT further expands its range of modern, highly specialised labs for translational research.

The international conference “Advanced Functional Polymers for Medicine” is held for the first time. Since then, the AFPM provides an annual platform for junior scientists from medicine, chemistry, material science and biology.

The BCRT spins out the company “Cellogic GmbH”, which provides health economic analyses to prepare the market introduction of medicinal projects.

The first clinical trial of a therapy developed at the BCRT is conducted successfully: The cell therapy that was developed in collaboration with “Pluristem Therapeutics Inc.” exploits the regenerative potential of placenta cells in other tissues.

The BCRT embarks on its first strategic, long-term collaboration with industry. The fruitful collaboration with the Israeli company “Pluristem Therapeutics Inc.” leads to several pre-clinical and clinical studies. Among these is a treatment of peripheral artery disease whose phase III clinical trial was successfully funded in 2016.

The first students enrol in the graduate school “Berlin-Brandenburg School of Regen-erative Therapies”, which has been funded by the Excellence Initiative of the German Research Foundation.

The BCRT spins out the company “CellServe GmbH”, which aims to further develop an innovative cell therapy to strengthen the heart muscle.

The Berlin-Brandenburg Center for Regenerative Therapies is established.

2006

2006

2007

2007

2009

2010

2010

2011

2012

2014

2016

milestones

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Flow cytometryMulti-parameter cell analysis and sorting

Good manufac-turing practiceClean-room facility to produce cell, tissue and gene therapy products

Cell harvestingProvides human tissue samples, standardised and characterised

Clinical development platformAdvising on EU and FDA regulation and authorisation

Science managementOrganises and opti-mises the research environment

IMPROVEMENT CYCLE

Implemen-tation in clinical practicePartners in pharma and biotech industry, hospitals and health insurers

Scientific monitoring of clinical trials

Comprehensive scientific monitoring of clinical trials is ensured through our

scientists and core units

Our ambition is that therapies developed at the BCRT reach the first-in-man clinical trial. We have adapted both our infrastructure and processes to achieve that efficiently. Projects receive early and continuous sup-port in the areas of regulatory affairs, intellectual prop-erty protection and market potential through internal advisors. A crucial part of our infrastructure are the core units: These central labs offer all users access to stand-ardised leading edge technologies, they provide the unique capability and expertise to produce therapeutics and medicinal products in-house under good manufac-

turing practice for first-in-man trials and they enable us to analyse these clinical trials scientifically with biomarker studies to elucidate the mechanisms that determine suc-cess and failure of a therapeutic approach. It is precisely this information that enables us to optimise diagnostic markers and that is fed back into a continuous improve-ment cycle. We have called this shuttling between bench and bedside “refined translation”. It generates new therapeutic approaches and substantially de-risks the de-velopment before cost-intensive pivotal clinical trials are initiated.

DevelopmentClinically relevant model systems in vitro

(e.g. patient tissue) and in vivo (e.g. animal models that reflect a disease pattern ad-equately) ensure translational relevance,

robustness and reproducibilityClinical trials

Planning and leading phase I/IIa clinical trials with associated

scientific analyses

Analyses of responder and non-responder patients

Reveals safety, pharmacokinetics and -dynamics of new therapies

First-in-man trials to ensure safety and to test efficacy in phase I/IIa clinical trials

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Innovation platformIndustry contacts, intellectual property and market analysis

BiomarkersAnalysis of patient samples and validation of new tests Next

Generation SequencingLeading-edge technologies to sequence DNA and RNA

Controlled drug delivery systemsProduction and characterisation of drug delivery systems

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DEVELOPMENT CYCLE

Basic research

Market aspectsEarly assessment of commercial potential and intellectual property protection, finding partners in industry and academia for co-development or licensingFeasibility

Proof-of-concept studies to validate an approach

Project criteriaAddressing an unmet medical need, excellent basic research and availability of new technologies. Fresh ideas are also from our proximity to the clinic.

Optimised and new approachesImproved or new therapeutic approaches are based on the careful analysis of biomarker panels and of therapeutic responses

reFined translation – betWeen bench and bedside

Biomarker discovery to predict therapeutic responseAllows stratification of patient groups to personalise therapies (precision medicine)

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Restoring cardiac function

Fact 1.8 million people in Germany suffer from cardiac insufficiency (myo-carditis) and over 50,000 die from it every year

Medical need Strengthen the heart muscle sustainably, as transplants and artificial hearts are a viable option for few patients only

BCRT approach Differential diagnostics and cell therapy to stimulate heart regeneration

Our heart is a highly effective organ – daily it pumps over 7,000 litres of blood through the body. After a heart infarction or an infection, however, it hardly recovers. To precisely characterise the resulting cardiac insufficiency, we have identified a biomarker. For patients with severe chronic myo-carditis we have developed a cell therapy, which relies on isolating cells from an individual’s heart tissue, expanding the cells in the lab and then re-inject them. As these cells are the patient’s own ones and fully differentiated there is no risk of rejection: The cells know, contrary to pluripotent stem cells, what to do and where to go and do not leave any scar tissue. This patented therapy is being developed further in the spin-off company “CellServe GmbH”, which plans first clinical trials in 2017.

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CORE UNIT Regeneration

Fact Critical-sized bone defects do not heal without help

Medical need Material with good availability and no infectivity

BCRT approach Hydrogel with defined properties

The treatment of critical bone fractures still relies on implanting bone grafts. To replace bone grafts, a biocompatible material needs to possess well-defined material properties and internal structure. Based on gelatin, we have developed a dimensionally stable and elastic hydrogel. Its pores allow the infiltration of bone-forming cells and ensures permeability to nutrients. In pre-clinical tests we could demonstrate that the implantation of the sub-structured, soft material alone stimulates bone forma-tion – neither growth factors nor additional cells have to be added. The hydrogel itself is completely dissolved after eight weeks. We are currently establishing the production of samples under good-manufacturing practice, to prepare for clinical trials in the near future.

by soft biomaterialsCORE UNIT

Functional genomics

Fact Analysing the genome alone does not reveal the causes of rare genetic diseases

Medical need Link disease pattern (phenotype) to genetic cause (genotype)

BCRT approach Standardised description of disease patterns and software to filter and annotate genome data (ontology)

The aim of functional genomics is to establish a relationship between an individual’s physiological and morphological traits, the phenotype, and the variants and mutations of the genes. Sequencing the genome is only the beginning. To achieve an accurate and standardised description of disease patterns, we have designed and established an online database, the “Human Phenotype Ontology”. Our software tool “Exomiser” then links the description with the gene sequence and other databases to identify potentially disease-causing genetic variants and mutations. Particularly for rare, inheritable diseases and atypical disease pattern, our “Exomiser” analysis has contributed to improved diagnosis and has provided the basis of new therapeutic approaches.

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with cell therapies

REGENE RATIVE THER APIES

At the BCRT, we work towards the development of better diagnostic markers, therapies and medicinal products. On this page, we present five case studies that illustrate groups of projects that have flourished because of the collaboration of different research fields and that have openend up new treatment options.

success stories

CORE UNITPersonalised stimulation

Fact 15 % of bone fractures heal insufficiently

Medical need Identify patients at risk, stimulate regeneration

BCRT approach Biomarker discovery and targeted therapies to modulate the immune response and to stimulate regeneration

Bone fractures are common and usually heal well – a prime example of endogenous regeneration. Unfortunately, slowly or insufficiently healing fractures are often only diagnosed late – limiting reha-bilitation and driving up cost. As some features of a bone fracture resemble a wound with a highly engaged immune system, we were able to identify an immunological biomarker panel to stratify patients into groups: The patient group at risk is characterised by the presence of specific immune cells that indicate an aged immune system. Our patented test has been licenced out to an industry partner for further development. To provide a therapy option for those patients at risk, we develop regenerative therapies: One therapy uses a cell type that, when injected into the fracture, stimulates bone formation. Another therapy inhibits the activity of the “old” immune cells by releasing biologi-cally active factors from a biomaterial.

of bone fracture healingCORE UNIT

CORE UNITPersonalised

Fact Treatment of undesired immune reactions is non-sustainable, often requiring permanent therapy and causing severe side effects

Medical need Tailored and sustainable modulation of the immune system

BCRT approach Biomarkers and cell therapies with T lymphocytes

To effectively control undesired immune reactions, e.g. in tissue transplantation or autoimmunity, the immune system has to be suppressed with strong drugs. We have identified a biomarker that for the first time allows us to set a truly patient-specific dose for immunosuppressive drugs, mitigating their severe side effects. We are leading a Europe-wide clinical trial that has started in 2016 at 10 sites and that seeks to confirm promising results from initial trials.We are also developing cell therapies that employ specific subsets of T lymphocytes. The first one increases the tolerance of the immune system towards graft tissue by isolating, expanding and then injecting a population of so-called regulatory T cells. The second therapy employs a different kind of T cell, so called effector T cells, to help the body fight severe viral infections. These tools will en-able us to modulate and support the immune system based on the identified need. The encouraging results of the currently running clinical trials have already led to 2nd and 3rd generations of these cell therapies, which will further expand the patient groups that will benefit from treatment.

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immunomodulation

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Joined-up thinking, acting and learning

Collaboration with industryTo successfully translate regenerative therapies from the lab to the patient, early and purposeful collaboration with partners in industry is vital. We are committed to strategic partnerships and engaged in co-development projects. We are also encouraged by our achievements in the classical fields of technology and knowledge transfer, e.g. patents, licences, consulting and spin-offs. The expertise of our core units is particularly in demand as contract research. Supported by state, federal and EU grants, we are also active in a range of collaborations with small and medium enterprises.

Local roots – global networkBeing located in the capital region Berlin-Brandenburg we have access to an excellent range of academic, clinical and industry partners. We benefit from the proximity to regulators and health insurers. We build on these local connections to drive our patient-oriented research for-ward.

On the international level, BCRT scientists are active in several EU projects and lead, for instance, the consortia “BIO-DrIM”, “The ONE Study” and “PACE”. We collaborate closely with the Wyss Institute (Harvard, US), the Baylor College of Medicine (Houston, US) and Tianjin University (Tianjin, China). Professor Duda is associate faculty at the Wyss Institute, Professor Mooney from the Wyss won an Ein-stein-Fellowship to do research at the BCRT. We have a long-standing collaboration with the Baylor College and a successful exchange pro-gramme. At Tianjin University, a joint research lab investigates bioma-terials. By working at all these sites, our PhD students advance their projects and boost international collaboration. Software tools spread across the globe quickest: Our scientists devel-op tools like the “Exomiser” to analyse genome data. They initiate and run databases to collect, analyse and make available data on cell types (“CellFinder”) and loads on joint implants (“Orthoload”).Regenerative Medicine is a global effort, that is, why we are engaged at home and abroad.

International conferences and white papersTo promote debate in and about the young field of Regenerative Medicine, we have es-tablished two international conferences. The annual seminar „Advanced Functional Poly-mers for Medicine” fosters interdisciplinary exchange between junior researchers and experts from chemistry and material science, biology and medicine. At the “Translate!” forum, experts from academia and clinical researchers are joined by industry representa-tives to discuss publicly innovative strategies and new models to make translational re-search a resounding success.

We aim to productively join-up the central players and stakeholders in Regenerative Medicine to forge a new way of thinking and acting – putting the concept of “restoring health” centre stage. To achieve that, we collaborate within the BCRT and with external partners on a national and international level, we train a new generation of specialists and promote a qualified, public discourse.

Open labsCollaboration at the BCRT starts with the small things in our daily life: work spaces, equipment, reagents and staff rooms. We have established spacious, modern and open laboratories – that are never too far from a tea kitchen. The exchange of ideas thrives. Projects that have the potential to generate fundamentally new approaches by bringing different research streams together are encouraged through a range of internal support schemes.

Training the next generation of specialistsOur graduate school, the Berlin-Brandenburg School of Regenerative Therapies, provides training and support to junior scientists: A mix of core and elective courses provides PhD students, postdocs and “clini-cian scientists” with a deep understanding of methods and technolo-gies that reach beyond their own specialisation. At the BSRT, they look into the challenges of translational science but also familiarise with con-cepts and strategies to successfully translate research to the clinic. To foster debate and to learn more about the practical side of translation, Translational Research Club provides an excellent forum. A particularly successful event is the annual three day "PhD Symposium", which is or-ganised by the students themselves and features international speakers.

Continuous training of cliniciansThe success of Regenerative Medicine is only possible if we keep the door between the laboratory and the clinic open: To enable clinicians to spend time in translational re-search and to build a career in clinical science, the BSRT established the “Clinical Scientist” programme, which has been adopted by the Charité. We also promote the exchange of scientists with the research and development departments of companies, of which Pluristem is the prime example.

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Facts & Figures

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2,700 PATIENTS involved in clinical trials

24

12 already commer-cialised

ACTIVEPATENTFAMILIES

1,700 PUBLICATIONS

ADDITIONAL THIRD-PARTY

330

4

250 STAFF

5,000citations for our top 25 papers

20papers in the most cited 1% in a year and field *

* Source: Thompson Reuters Web of Science

DFGFederalEUIndustryOther

Medical doctors, scientists, engineers, trans-lation experts, PhD students and technical staff from 30 countries

ca. € 30 million

42%

25%

18%

9%

6%

* data for Charité only

since 2006

FUNDS*

Phase I-III associated contract research

interventional studies and companion

diagnostics

biomarker15 6

CLINICAL TRIALS

BIOMARKER STUDIES

SPIN-OFFS

INDUSTRY PARTNERSHIPS

PRODUCTS in the market

updated 2016

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imprint

Published byBerlin-Brandenburg Center for Regenerative Therapies (BCRT)Charité – Universitätsmedizin Berlin Campus Virchow-KlinikumAugustenburger Platz 1, 13353 Berlinwww.b-crt.de

V.i.S.d.P.Prof. Dr. Hans-Dieter Volk, MD

Concept, editing and translation Dr. Christoph Feest Alexandra Scherer

CopyeditorTraudl Kupfer, www.traudl-kupfer.de

DesignCorinna Kallich, www.corinja.de

PhotosMeike Kenn, www.meikekenn.comPeer Schroeder, www.hardcopy-press.de Birgit Formann, Charité

PrintDruckerei Rüss, www.druckerei-ruess.de

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Sponsored by:

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