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Personalized cellular immunotherapies for serious diseases
Miltenyi Biomedicine is dedicated to developing innovative cancer and regenerative treatments for patients suffering from serious diseases with unmet medical needs
Personalized cellular immunotherapies for serious diseases
Miltenyi Biomedicine is dedicated to developing innovative cancer and regenerative treatments for patients suffering from serious diseases with unmet medical needs
Researching immunotherapies in hemato-oncology
At Miltenyi Biomedicine, we believe in the power that personalized cell and gene therapies have to make a difference for patients who have limited treatment options available today. Developments like Chimeric Antigen Receptor T (CAR T) cells are certainly ground-breaking innovations in treating cancer – and we strongly believe that, promising developments notwithstanding, there is significant room to improve available therapies. Our clinical research program includes: CAR T, CAR NK, our proprietary Adapter CAR™ and induced pluripotent stem cells (iPSC) technologies.
Our goal is to create more powerful and effective treatment options, with a sustainable and guaranteed supply, to treat patients worldwide in a timely manner.
Outstanding examples of Miltenyi’ s expertise in the therapeutic arena are: Immunotherapy through innovative CAR T cell approaches currently under study in international trials for solid tumors and hematological malignancies (for more information please refer to our Pipeline below).
We are currently developing a CAR T therapy to treat adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after at least one line of treatment. Our zamtocabtagene autoleucel uniquely targets the combination of both CD19 and CD20 proteins on B cells. This promising investigational therapy candidate has been selected for the PRIority MEdicines scheme (PRIME) in the European Union. In addition, we have submitted an Investigational New Drug (IND) application for a new CAR T cell therapy to the Food and Drug Administration (FDA) in the United States. Further clinical development of this therapy candidate is planned for Asia Pacific.
If you would like to learn more about our research please contact us!
We believe that novel cell and gene therapies will change medicine forever.
We believe that novel cell and gene therapies will change medicine forever.
Our Research Platforms
CAR T Cells
What: Disease-specific autologous immune cells, created from the patient’s blood, that attack and destroy cancer cells. The retargeting of immune cells can be achieved by inserting new genetic information into the patient’s T cells via gene engineering.
Our Concept: Second-generation CAR T therapy optimized to overcome the limitations of current options. In particular, our dual and triple targeting is aimed at reducing disease escape mechanisms, thus providing new treatment options to patients who have not been helped by conventional therapies.
Method: T cells are collected from the patient and genetically engineered with a CAR construct. Our proprietary technology ensures optimized CAR T cell expansion in a timely manner.
Lead Candidates/Indications: We are developing several products for single, double and triple targeting. Our first disease target is DLBCL. Please check our pipeline for more information about other indications we are actively researching.
Adapter CAR™
What: Adapter CAR cells are an improvement of the conventional CAR T technology. They are designed to allow targeting of multiple cancer antigens and control of their activity, thus increasing the safety and efficacy of CAR cells.
Our Concept: Instead of directly targeting a disease-associated antigen on a cancer cell, the CAR technology is split into two components – the CAR T cell and the adapter. The adapter CAR will only be active when both components are available.
Method: The adapter CAR cells are engineered not to recognize a target cell but rather a docking structure present in the adapter. The adapter itself consists of the docking structure and a cancer-specific binding domain. When the adapter is bound to a cancer cell, the CAR will bind to the adapter and initiate killing the cancer cell. The use of different adapters enables targeting different types of cancer cells with only one CAR, simplifying the treatment process and expanding treatment options. Moreover, this technology allows the activity of the CAR to be controlled by applying the adapter at different dose levels. The option to switch off the CAR’s activity is not addressed by current treatments, but it is believed to be important for solid tumors.
Lead Candidates/Indications: We are currently running a proof of concept study as part of our clinical research program in Relapse/Refractory NHL; and we are actively working on Relapse/Refractory AML.
CAR NK Cells
What: Natural Killer (NK) cells belong to the innate immune system. They can be derived from healthy donors and genetically engineered, similarly to CAR T cells, to recognize a specific disease target.
Our Concept: CAR NK cells can be produced from a healthy donor. Compared to currently available CAR T cells, which are produced from the patient’s own T cells, this technology allows ‘off the shelf’ CAR cells to be manufactured, thus providing a convenient and rapid treatment option. Although the technology for delivering the genetic information into the NK cell is very similar to the technology used to engineer T cells, the NK cell has different characteristics compared to T cells. NK cells have an intrinsic anti-tumor activity without needing to be activated. Together with the anti-tumor activity delivered by the CAR, this synergy may enhance the anti-tumor activity of CAR NK cells and prevent antigen escape – thus providing a very efficient therapeutic option. In addition, the short persistence of CAR NK cells could offer a safer means of therapy in various diseases.
Method: Contrary to autologous immune cells, which are created from the patient’s own blood, allogenic CAR NK cells are generated from a healthy donor. The NK cells collected are genetically modified to express a specific CAR aimed at the target of interest.
Lead Candidates/Indications: The main indications for the future CAR NK cell product are hematologic malignancies in general, with special attention to developing a cell therapy candidate for AML.
iPS Cells
What: Induced pluripotent stem (iPS) cells raise the possibility of producing custom-tailored cells for studying and treating numerous diseases and for use in regenerative medicine approaches.
Our Concept: Somatic cells from a donor or a patient are collected and genetically reprogrammed to become iPS cells, enabling products for regenerative approaches to be developed. iPS cells can differentiate into target cells (such as oligodendrocytes) and, subsequently, may regenerate normal tissue functions. We are optimizing a fully closed system to generate iPS cells to be used as a pristine source of differentiated cells.
Method: Once iPS cells are created, they can be expanded, stored and, if needed, expanded over and over again. They can then be differentiated into a spectrum of different cell types (to replace tissues affected by diseases, for example). This technology opens up new possibilities for personalized and/or off-the-shelf therapeutic products that would benefit a greater number of patients who currently have unmet therapeutic needs.
Lead Candidates/Indications: Our clinical research program is currently focusing on Parkinson’s disease, with several approaches under development.
Our pipeline
Miltenyi has a diverse portfolio of proprietary product candidates for hematologic cancers, with several clinical trials currently underway or planned for the near future. The graphic shows examples of product candidates in late pre-clinical and clinical development stages. Additionally we are actively expanding our pipeline by applying our platforms to degenerative disorders, and solid tumors such as pancreatic cancer, ovarian cancer and glioblastoma.
Zamtocabtagene autoleucel1
NCT03870945
A prospective, multi-center, open phase I/II trial to evaluate feasibility, dosage, safety and toxicity of ex vivo expanded autologous T cells genetically modified to express anti-CD20 and CD19 immunoreceptor.
Target:CD19CD20
Technology
Tandem CARTTandem CART
Tandem CAR T. MB-CART2019.1 are genetically engineered autologous T cells expressing chimeric antigen receptors recognizing both CD19 and CD20 epitopes on malignant B cells. It is hypothesized that targeting both epitopes simultanously may prevent CD19 negative relapse. MB-CART2019.1 is currently evaluated in different types of B-NHL.
DLBCL
Diffuse Large B Cell Lymphoma (DLBCL) is an aggressive cancer due to the fast growth rate of B cells. The incidence of DLBCL generally increases with age, and most patients diagnosed with it are over the age of 60. It’s annual incidence is 7-8/100000.
DLBCL
Diffuse Large B Cell Lymphoma (DLBCL) is an aggressive cancer due to the fast growth rate of B cells. The incidence of DLBCL generally increases with age, and most patients diagnosed with it are over the age of 60. It’s annual incidence is 7-8/100000.
MCL
Mantle Cell Lymphoma is a Non-Hodgkin Lymphoma where malignant B cells usually accumulate in lymph nodes. MCL represents 5-7% of malignant lymphoma in western Europe and the annual incidence is 1-2/100000.
MCL
Mantle Cell Lymphoma is a Non-Hodgkin Lymphoma where malignant B cells usually accumulate in lymph nodes. MCL represents 5-7% of malignant lymphoma in western Europe and the annual incidence is 1-2/100000.
FL
Follicular Lymphoma is the most common type of indolent Non-Hodgkin Lymphoma with an annual incidence of 3-4/100000. It usually has a slow disease course but 20% of the patients present with an aggressive disease requiring more intensive treatment.
FL
Follicular Lymphoma is the most common type of indolent Non-Hodgkin Lymphoma with an annual incidence of 3-4/100000. It usually has a slow disease course but 20% of the patients present with an aggressive disease requiring more intensive treatment.
CLL
Chronic Lymphocytic Leukemia is the most common type of leukemia in the western world with more than 20000 new cases per year in the US. It affects mainly older adults above the age of 70. CLL usually shows a slow progression rate but some patients require more intensive treatment.
CLL
Chronic Lymphocytic Leukemia is the most common type of leukemia in the western world with more than 20000 new cases per year in the US. It affects mainly older adults above the age of 70. CLL usually shows a slow progression rate but some patients require more intensive treatment.
MB-CART 2219.1
Target:CD19CD22
Technology
Tandem CARTTandem CART
Tandem CAR T. MB-CART2219.1 are genetically engineered autologous T cells expressing chimeric antigen receptors recognizing both CD19 and CD22 epitopes on malignant cells. It is hypothesized that targeting both epitopes simultanously may prevent CD19 negative relapse. MB-CART2219.1 is currently in late stages of preclinical development.
ALL
Acute Lymphoblastic Leukemia is an aggressive form of leukemia with an overal annual incidence of 1,1/100000 . It is the most common leukemia in childhood - incidence up to 5/100000 - but can affect also adult patients. Patients with relapsed or refractory disease are in need of innovative treatment options.
ALL
Acute Lymphoblastic Leukemia is an aggressive form of leukemia with an overal annual incidence of 1,1/100000 . It is the most common leukemia in childhood - incidence up to 5/100000 - but can affect also adult patients. Patients with relapsed or refractory disease are in need of innovative treatment options.
MB-CART 19.1
NCT03853616
Phase l/ll multi-centric, single arm, prospective, open, dose-escalation study in patients with relapsed or refractory CD19-positive B cell malignancies.
Target:CD19
Technology
Mono CARTMono CART
Second generation CAR T. Genetically engineered autologous T cells expressing chimeric antigen receptors recognizing CD19 epitopes.
NHL
Non-Hodgkin Lymphoma is a group of heterogeneous diseases caused by malignant B cells. These cells commonly share the CD19 epitope thus making innovative therapies directed against CD19 a promising treatment approach especially for those patients who do not respond to conventional therapies.
NHL
Non-Hodgkin Lymphoma is a group of heterogeneous diseases caused by malignant B cells. These cells commonly share the CD19 epitope thus making innovative therapies directed against CD19 a promising treatment approach especially for those patients who do not respond to conventional therapies.
ALL
Acute Lymphoblastic Leukemia is an aggressive form of leukemia with an overal annual incidence of 1,1/100000 . It is the most common leukemia in childhood - incidence up to 5/100000 - but can affect also adult patients. Patients with relapsed or refractory disease are in need of innovative treatment options.
ALL
Acute Lymphoblastic Leukemia is an aggressive form of leukemia with an overal annual incidence of 1,1/100000 . It is the most common leukemia in childhood - incidence up to 5/100000 - but can affect also adult patients. Patients with relapsed or refractory disease are in need of innovative treatment options.
MB-CART 20.1
NCT03664635
A phase I/II trial to assess safety, dose finding and feasibility of ex vivo generated MB-CART20.1 cells in patients with relapsed or refractory CD20 positive B-NHL.
Target:CD20
Technology
Mono CARTMono CART
Second generation CAR T. Genetically engineered autologous T cells expressing chimeric antigen receptors recognizing CD20 epitopes.
NHL
Non-Hodgkin Lymphoma is a group of heterogeneous diseases caused by malignant B cells. These cells commonly share the CD20 epitope thus making innovative therapies directed against CD20 a promising treatment approach especially for those patients who do not respond to conventional therapies.
NHL
Non-Hodgkin Lymphoma is a group of heterogeneous diseases caused by malignant B cells. These cells commonly share the CD20 epitope thus making innovative therapies directed against CD20 a promising treatment approach especially for those patients who do not respond to conventional therapies.
MB-CART 20.1
NCT03893019
A phase l multi-centric, single arm, dose-escalation study in patients with unresectable stage III or IV melanoma.
Target:CD20
Technology
Mono CARTMono CART
Second generation CAR T. Genetically engineered autologous T cells expressing chimeric antigen receptors recognizing CD20 epitopes.
Melanoma
Melanoma is a malignant type of skin cancer developing from pigment producing cells called melanocytes. The annual incidence of new cases in the US is 22.7/100000. Based on the finding that melanoma cancer sustaining cells express CD20 and preclinical models, there is a rationale for CD20 targeting CAR T cells.
Melanoma
Melanoma is a malignant type of skin cancer developing from pigment producing cells called melanocytes. The annual incidence of new cases in the US is 22.7/100000. Based on the finding that melanoma cancer sustaining cells express CD20 and preclinical models, there is a rationale for CD20 targeting CAR T cells.
huBCMA
Target:BCMA
Technology
Mono CARTMono CART
Second generation CAR T. Genetically engineered autologous T cells expressing chimeric antigen receptors recognizing BCMA epitopes.
Myeloma
Multiple Myeloma is a cancer of plasma cells which are normally producing antibodies. It‘s annual incidence in the US is 7/100000. it is still considered an incurable disease. Therefore there is a high need for new treatment options.
Myeloma
Multiple Myeloma is a cancer of plasma cells which are normally producing antibodies. It‘s annual incidence in the US is 7/100000. it is still considered an incurable disease. Therefore there is a high need for new treatment options.
MB-dNPM1-TCR.1
Target:dNPM1
Technology
TCRTCR
T cell with a genetically engineered T cell receptor specific for dNPM1. Compared to CAR T cells engineered TCR cells can detect also cancer targets inside the cancer cell. This may help to overcome certain relapse mechanisms common in cancer.
AML
Acute Myeloid Leukemia is an aggressive form of leukemia with an annual incidence of 3.7/100000 in the western world. It affects the myelopoiesis with a heterogeneous biology. It is a rapidly progressing disease and patients not responding to or relapsing after conventional therapy are in need for innovative treatments.
AML
Acute Myeloid Leukemia is an aggressive form of leukemia with an annual incidence of 3.7/100000 in the western world. It affects the myelopoiesis with a heterogeneous biology. It is a rapidly progressing disease and patients not responding to or relapsing after conventional therapy are in need for innovative treatments.
1 Zamtocabtagene autoleucel is a proposed International Nonproprietary Name (INN).
Our Chimeric Antigen Receptor T (CAR T) cell technology
Chimeric Antigen Receptor T (CAR T) cell technology has already shown significant success in fighting hematological cancers. These genetically engineered T cells – produced from the patient’s own immune cells – have demonstrated their potential to treat hematologic cancers in several clinical trials and approved indications.
However compared to other therapies, the prolonged manufacturing time can mean longer waiting time until patients can be treated. Therefore, we have developed a fast, robust manufacturing process to treat patients sooner and expand the number of patients that can be treated with these life-changing products. Furthermore, our innovative CAR T design simultaneously targets CD19 and CD20 proteins on B cells. It is hypothesized that such a dual-targeting approach can potentially reduce the risk of patient relapse. These innovations are aimed at providing patients with improved treatment options.
There is evidence that CAR T cells have the potential to persist in the patient, thus making them a personalized, living drug. Miltenyi has already developed several type of CAR T cells to optimize their efficacy in treating patients. For example a common tumor escape mechanism employed by cancer cells is to stop expressing the specific antigen recognized by the CAR T cells and thus become unrecognizable to the patient’s immune system. In this case, CAR T cells can be prepared to recognize two different antigens – which is called ‘dual targeting’ – which may help overcome tumor escape mechanisms. Both persistence and dual targeting have the potential to provide a durable response against cancer in patients who have failed one or more lines of therapy.
