The emerging field of cell and gene therapies (CGT) is a transformative new category of medicine seeking to treat diseases by restoring or reconditioning cells or genes. While both approaches aim to repair the underlying cause of genetic or acquired diseases, cell and gene therapies are distinct from one another in their mechanism of action. Although the fields of cell and gene therapies are in their infancy with regards to clinical applications, the research and development of novel cell and gene therapies is rapidly expanding. Today, more and more CGT applications are making it through R&D towards clinical trials and beyond. Research and development in these application areas require large numbers of high-quality cells. Stirred-tank bioreactors facilitate efficient cell expansion when conventional 2D-culture systems reach their limits.
Find more information about Cell and Gene Therapy
Cell therapy techniques aim to treat diseases by harnessing cells to deliver treatment. Alternatively, cell therapy can be used to repair or replace damaged tissue. In cell therapy, cells are cultured and if required modified outside a patient’s body before being infused into the patient. The cells can originate from the patient themselves (autologous cells) or from a healthy donor (allogeneic cells). Stem cells are one of the most widely used cell types harnessed for cell therapy today. These undifferentiated cells bring great value to regenerative medicine for their ability to differentiate into almost any type of cell in the body.
Gene therapy aims towards restoring abnormal or dysfunctional expression patterns of a disease-related gene by introducing genetic material to replace, enhance or silence its function. Gene therapy intends to achieve long-lasting treatment or prevention of diseases including certain types of cancer, inherited disorders, and viral diseases. It has been estimated that approximately 10,000 human diseases are caused by a mutation in a single gene, and gene therapy could represent a therapeutic option for many of these. Ex vivo gene therapy is the removal of specific target cells from a patient, which then undergo genetic modification, before being transplanted back into the patient. This is an important crossover with cell therapy, as applications like CAR-T cell therapy rely upon ex vivo gene editing. In vivo gene therapy involves gene transfer directly into a patient and is ideally suited for monogenetic disorders. It involves the transfer of genetic material into a patient’s cells with the aim of restoring normal function.
Some cell and gene therapies take a hybrid approach to combatting diseases and can be considered both cell and gene therapies. These work by editing genes within specific types of cells and inserting them into the body. Chimeric antigen receptor (CAR) T cell therapy is a novel technique used in the treatment of blood cancers including lymphomas, some types of leukemia, and multiple myeloma. T cells are isolated from a patient’s blood, and then genetically engineered with the addition of a gene for a special receptor (CAR), which is highly personalized for a patient’s specific cancer phenotype. Once administered back to the patient, the newly engineered CAR-T cells are able to recognize and attack cancer cells. Similar engineered immune cell approaches include tumor-infiltrating lymphocytes (TILs) and tumor-specific T cell receptor (TCR)–modified T cells.
From a scientific concept to the development of novel therapies
The Eppendorf Bioprocess Unit builds on decades of experience in upstream bioprocessing; since more than ten years Eppendorf is committed to contribute to the advancement of stem cell cultivation in stirred-tank bioreactors. The scalability, robustness, quality, and cost-effectiveness of cell culture processes are key to success when translating basic research to the development of commercially viable cell and gene therapies. Eppendorf supports you in developing powerful upstream bioprocesses for cell and gene therapy development. Contact us to learn more about how we can support you!
Producing large amounts of cells in conventional 2D-culture systems is very labor- and time-intensive. Stirred-tank bioreactors reduce manual labor when handling large cultures, simplify scale-up, and like this facilitate efficient cell expansion. Eppendorf supports you with transferring your process from flasks or bags to bioreactors and seamlessly scaling-up from small to larger working volumes.
Accelerating the development of bioprocesses is key to shorten the time to market. Parallel bioprocess control systems from Eppendorf facilitate parallel experimentation in small working volumes and, like this, process development in a time- and resource-saving manner.
Reproducing culture success requires reproducing the cells’ growth environment. Stirred-tank bioreactors and bioprocess control software from Eppendorf enable you to precisely monitor and control the cells’ growth environment. Like this, you are always well informed on what is happening in your bioreactor and are able to optimize your bioprocess, compare different runs, and reproduce culture results.
The automation of routine tasks provides scientists more time for valuable research. Bioprocess control software from Eppendorf enables you to automate culture feeding and parameter control to reduce the working time spent on a running process.
Upstream bioprocessing for various cell and gene therapy applications
Optimizing the upstream bioprocess is an essential part of cell and gene therapy development. Stirred-tank bioreactors from Eppendorf provide extensive options for monitoring and control of critical process parameters in real-time. Through scalable bioreactor design, results yielded in small scale can be transferred to larger working volumes.
Organized for the first time in 2017, the Stem Cell Community Day (SCCD) connects researchers from academia and industry across the world, to discuss recent advances and new technologies in the field of stem cell production. With a special focus on the controlled cultivation of stem cells in stirred-tank bioreactors, the SCCD is an optimal place to exchange knowledge and share expertise with scientists around the world.
Our next Stem Cell Community Day will take place on November 21, 2023 in Leiden, The Netherlands, and online. Why not get involved?