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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 various CAR T cell platforms.
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. Zamtocabtagene autoleucel is currently being investigated in pivotal stage clinical trials in the EU and USA. 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.
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
Target: CD19, CD20
Technology
Tandem CAR TTandem CAR T
Tandem CAR T (chimeric antigen receptor) are genetically engineered autologous T cells expressing a chimeric antigen receptor recognizing two antigens (i.e. CD19 and CD20) on cells expressing them.
Zamtocabtagene autoleucel is currently evaluated in different types of B-NHL (non-Hodgkins lymphoma).
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. Its annual incidence is 7-8 cases per 100,000 people.
3rd line is the treatment that is given when both initial treatment (first-line therapy) and subsequent treatment (second-line therapy) don’t work, or stop working.
NCT04792489
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.
Please visit ClinicalTrials.gov (NTC04792489).
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. Its annual incidence is 7-8 cases per 100,000 people.
2nd line is the treatment that is given when initial treatment (first-line therapy) doesn’t work, or stops working.
NCT04844866
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.
Please visit ClinicalTrials.gov (NCT04844866).
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. Its annual incidence is 7-8 cases per 100,000 people.
1st line The first treatment given for a disease. It is often part of a standard set of treatments, such as immuno-chemotherapy and radiation.
Mature B cell neoplasm
B cell neoplasms comprise a highly heterogeneous group of diseases originating from B cells. The most common pediatric mature B cell neoplasm belongs to the non-Hodgkin lymphomas (NHL). NHL is one of the most common malignancies in children accounting for about 7% of all cancers in patients under 20 years of age with a median age at initial diagnosis of 10.8 years. Epidemiology data is limited due to the low incidence rates.
MCL
Mantle cell lymphoma (MCL) is a non-Hodgkin Lymphoma where malignant B cells usually accumulate in lymph nodes. MCL represents 5-7% of malignant lymphoma with an annual incidence of 1 case per 200000 people.
MB-CART 19.1
Target: CD19
Technology
Mono CAR TMono CAR T
Second generation CAR T. Genetically engineered autologous T cells expressing chimeric antigen receptors recognizing CD19.
SLE
Systemic lupus erythematosus (SLE) is a devastating autoimmune multisystem disease that poses several clinical challenges. It can result in substantial morbidity, mortality, and associate with poor quality of life. The annual incidence is approximately 5 cases per 100,000 people.
MB-CART 2219.1
Target: CD19, CD22
Technology
Tandem CAR TTandem CAR T
Tandem CAR T are genetically engineered autexpressing themologous T cells expressing a chimeric antigen receptor recognizing two antigens (i.e. CD19 and CD22) on cells.
B ALL pediatric
B-cell acute lymphoblastic leukemia (B-ALL) is a rare disease and is a common childhood cancer that is the leading cause of pediatric cancer death. The disease harbors from a mutation in B cells that may cause the cancerous B cells to survive and grow better than normal cells.
MB-dNPM1-TCR.1
Target: dNPM1
Technology
TCR TTCR T
Like CAR T cells, T cell receptor-engineered T cells (TCR T) are modified in a laboratory. However, compared to CAR T cells they can detect targets from inside the cancer cells (intracellular targets). This allows them to capitalize on the natural mechanisms of T cells. TCR-based solutions may be more suited to treating solid tumors and may benefit cancer patients with unmet medical needs.
AML
Acute myeloid leukemia (AML) is an aggressive form of leukemia with an annual incidence of 3.7 cases per 100,000 people in the western world. It affects myelopoiesis with a heterogeneous biology. It is a rapidly progressing disease and patients not responding to or relapsing after conventional therapy are in need of innovative treatments.
MB-CART FolR1
Target: FOLR1
Technology
Mono CAR TMono CAR T
Second generation CAR T (chimeric antigen receptor). Genetically engineered autologous T cells expressing a CAR recognizing one single disease-associated antigen on cells expressing them.
Ovarian cancer
Ovarian cancer is considered one of the gynecologic deadliest cancers globally. It has a high morbidity and mortality rate due to the lack of effective treatments addressing it. Diagnosis is usually made at an advanced stage of the disease which also affects the high mortality rate. The annual incidence is 6.6 cases per 100,000 people globally.
MB-CART GD2-IL18
Target: GD2
Technology
Mono CAR TMono CAR T
Second generation CAR T (chimeric antigen receptor). Genetically engineered autologous T cells expressing a CAR recognizing one single disease-associated antigen on cells expressing them.
GBM
Glioblastoma is one of the brain‘s most aggressive cancers. It has a global yearly incidence of 3 cases per 100,000 people. The treatment is challenging due to the nature and location of the tumor. The prognosis is often poor, and patients' unmet medical need is high.
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.
