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With our current lighthouse topic, we are highlighting our expertise and know-how in one of the most innovative research topics of our time: cell and gene therapy. Thanks to these advances, diseases that were previously considered incurable could become treatable in the future. Cancer, neurological diseases and hereditary diseases - innovative therapies could soon be a quantum leap in medicine. This would be unthinkable without LSR technologies.
Cell and gene therapyEnablersSeries on the topic
Many biological and medical technologies are required along the process chain of cell and gene-based therapies - from development to production and application. A large proportion is produced by the LSR and IVD industry. Both industries thus form the basis for academic and industrial research, development and production of therapeutic products as well as for the diagnosis of patients before, during and after therapeutic application. CAR-T cell therapy technology is currently the best-known model in the field of cell and gene therapies (GCT). In this cell-based gene therapy, components of the body's own immune system, known as T cells, are removed from the body and genetically modified so that they can recognize cancer cells. The so-called chimeric antigen receptor T cells (CAR-T), which are multiplied in vitro, are then administered to tumor patients suffering from B-cell neoplasia, for example. Elimination of the cancer cells and thus a cure becomes possible.
Adenovirus-associated gene therapies are also a well-known example of GCT and are used, for example, in the treatment of spinal muscular atrophy, a rare disease. Here, genetically reprogrammed viruses of the AAV9 type are used to apply therapeutic human genetic information to newborns. The patient's dysfunctional gene is replaced by a functional gene. This requires production of the human genetic material, multiplication of the viruses as vectors and comprehensive quality controls in all process steps. Patients are usually selected on the basis of genetic analyses, e.g. in newborn screening. Patients receiving GCT are subject to close monitoring during treatment and comprehensive diagnostic follow-up care. Both are made possible by in-vitro diagnostic technologies.
Decades of genome research mean that cell and gene therapies can now be produced and used as treatments. The LSR industry is an important partner for research.
Hermann, 78 years old, was diagnosed and treated with DLBCL several years ago. DLBCL is a malignant tumor disease of white blood cells, the lymphocytes. He is now showing symptoms again. A more serious form of DLBCL is diagnosed.
Laboratory diagnostics on the lymph node
Laboratory diagnostics
Hermann's cancer cells are genetically examined to check whether CAR-T cell therapy is promising. The genetic tumor analysis gives the green light. Doctor and patient decide in favor of the therapy.
Laboratory diagnostics for genetic tumor analysis
Artificially produced DNA is introduced into the cells so that the T cells recognize the cancer cells.
This encodes the genetic information for the CAR gene and the chimeric antigen receptor (CAR) is expressed on the cell surface.
Expansion of genetically modified CAR-T cells.
Shipping, production and quality control take about 4 weeks.
Treatment and monitoring
Infusion of the genetically modified CAR-T cells. They can now recognize and fight Hermann's cancer cells.
Recovery
Grandfather Hermann has recovered. The genetically modified CAR-T cells have recognized and fought the cancer cells.
Lara is diagnosed with spinal muscular atrophy (SMA) through newborn screening. SMA is a genetically inherited neuromuscular disease caused by the loss or alteration of the SMN-1 gene. Symptoms include muscle weakness and muscle wasting.
Newborn screening for mutation in the SMN1 gene
The laboratory checks whether the therapy is promising. To do this, Lara must not have any antibodies against AAV9 (adeno-associated virus type 9) in her blood, as these would otherwise have a negative effect on the therapy.
Laboratory diagnostics monitor the course of the disease, including possible side effects
AAV9 is used as a vector. This means that the envelope and the function of infecting human cells are used to insert the correct SMN1 gene into Lara's cells.
The treatment is carried out with a very high number of viral vectors. 1.1 × 1014 vectors per kg are required. Lara weighs 3.4 kg - this means she needs a dose of 4.5 × 1014 vectors.
Lara receives the treatment by injection.
Granddaughter Lara learns to move and walk. Thanks to the non-mutated SMN1 gene, the muscle cells can develop correctly. The muscle weakness is alleviated.
The LSR and IVD industries produce many different components and technologies along the process chain and are therefore part of the most relevant stakeholders for GCT - from research to application. We supply academia, hospitals, smaller spin-offs as well as the biotech and pharmaceutical industry including associated Clinical Research Organizations (CROs) and Contract Manufacturing Organizations (CDMOs).
Thanks to decades of genome research, it is now possible to produce GCTs and use them as therapeutics. On the one hand, this has led to the establishment of diagnostic applications such as genome sequencing. On the other hand, building blocks and technologies for production have also been further developed, such as applications in cell culture or for genetic modification. Cell and gene therapies would be unthinkable without our expertise, our know-how and our technologies.
IVD-Produkte und LSR-Technologien werden in nahezu allen Schritten der Zell- und Gentherapie verwendet.
The LSR industry's product portfolio includes consumables, reagents and small and large devices. These products and technologies are used as manufacturing components for the GCT to be administered and include products for the cultivation of cell material, gene editing, virus production and products for virus purification.
Quality control of each individual production step is of utmost importance and is supported by the LSR industry with a variety of technologies to answer e.g. questions of virus-mediated gene therapy such as virus quantity and purity, tropism and antigenicity. Without quality controls, the efficacy of GCT cannot be tested - before it is administered to patients.
A large number of industry products meet important GMP standards that guarantee industrial production (GMP = Good Manufacturing Practice). These include, for example, products for cell isolation and cultivation, which are required for the use of CAR-T cell therapies. But also products for the production of viral envelopes and their purification for virus-mediated gene therapy.
An important factor in the production of GCT is the workflow under cleanroom conditions, as cell products that are applied to patients as GCT cannot be sterilized. In the cleanroom, the sterility of consumables is just as relevant as the control of particles in the air or liquids used, such as water or buffer solutions.
The production of cell and gene-based therapies is currently still characterized by many patient-specific individual solutions and high costs. However, long-term financial viability through the SHI system depends on scalable processes that need to be integrated into the development and production processes at an early stage. The LSR industry provides technologies that are important for upscaling. These include bioreactors for the cultivation of cells and viruses on a larger scale or scalable flow cytometry units for scalable cell isolation.
Many of our technologies are building blocks in the manufacturing process - from equipping cleanrooms to quality control and upscaling.
Our building blocks include products for the cultivation of cell material, gene editing, virus production and products for the purification of GCT. Many of the industry products meet the high GMP standards.
GCT cannot be sterilized. This makes the conditions in the cleanroom all the more important. The sterility of consumables is just as relevant as the control of particles in the air or liquids used such as water or cleaning solutions. We supply products and technologies that meet cleanroom conditions.
The production of GCT is characterized by many patient-specific individual solutions and high costs. Scalable processes are necessary for long-term financial viability. We supply technologies that are important for upscaling. These include bioreactors for the large-scale cultivation of cells and viruses as well as scalable units for cell isolation.
The quality control of each individual process step is extremely important. We support this with a variety of technologies. In this way, we help to check whether GCT is effective, targeted and safe - before it is applied to the patient.
The LSR department has defined cell and gene therapy as a lighthouse topic and will provide an insight into the fascinating world of these highly innovative therapeutic options every month in 2024. After all, cell and gene therapy is THE topic of the future in medicine.
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