Innovations to improve personalized medicine
They are conducting research in personalized medicine: Michael Fichtner, Ines Lahmann and Clara Vázquez García (from left to right).
Three projects by researchers at the Max Delbrück Center in the Helmholtz Association have won 2025 GO-Bio Initial funding from the German Federal Ministry of Education and Research (BMBF). From synthetic DNA vectors to human-like models of neuromuscular disease, to better assessing the risk of cancer and immune deficiencies, the funded projects aim to make personalized therapies more affordable. The funding specifically supports early life science research that has innovation potential.
The grants cover a one-year conceptual phase during which research teams can hone ideas and carry out market analyses, which include investigating the commercial potential of their ideas. The funding supports individual projects at universities or non-university research institutions with up to €100,000 for 12 months. Other expenses, such as costs of patenting, material and consumables are also eligible for funding. Successful conceptual projects have the opportunity to advance to a two-year feasibility phase in a second selection process.
Sleeping Beauty modifies immune cells
Chimeric antigen receptor (CAR) and T-cell receptor (TCR) therapies have been widely successful in treating many forms of cancer. They involve extracting immune cells from patients, introducing therapeutic genes into the cells and injecting these modified cells back into patients. The genetic modification equips patients’ immune systems to recognize and fight their cancers, affording many a cure. The therapy is also being investigated to treat incurable autoimmune diseases.
Currently, researchers use modified viruses to introduce genes into immune cells. However, producing these viral delivery systems is complex, expensive and time-consuming, limiting access to personalized therapies.
Michael Fichtner
Instead of using viruses, Dr. Michael Fichtner, a postdoc in the Mobile DNA lab of Dr. Zsusanna Izsvak, will explore the feasibility of producing and using synthetic transposons to transfer therapeutic DNA into human cells.
Fichtner will be applying “Sleeping Beauty” transposase technology to modify cells, which was developed at the Max Delbrück Center in the Izsvak lab. It takes advantage of a DNA transposon – a DNA sequence that has the capacity to “jump” into different spots in the genome. The technology consists of the Sleeping Beauty transposon and transposase, an enzyme that cuts the transposon out of a stretch of DNA that contains it and then integrates it into the target genome. With his €94,000 grant, Fichtner hopes to identify and develop novel ways to produce synthetic transposons to introduce patient specific therapeutic genes into immune cells. This could significantly reduce production costs and enable broad access. It may also lead to novel personalized therapies.
“The technology has the potential to make a huge impact,” says Fichtner. “I hope that it can help to cure diseases for which no cure is available, or make prohibitively expensive therapies more affordable.”
Drug screening for neuromuscular disorders
Ines Lahmann
Dr. Mina Gouti’s Stem Cell Modeling of Development and Disease lab has developed advanced platforms that resemble the human neuromuscular junction, including a 2D high-throughput screening system and 3D neuromuscular organoids. These models are derived from patient-specific induced pluripotent stem cells, potentially enabling researchers to understand disease mechanisms and response to therapies in individual patients.
Dr. Ines Lahmann, a postdoc in Gouti’s lab has been awarded €100,000 to explore whether these new models can be used to develop novel personalized therapies to treat neuromuscular diseases.
In tissues, the neuromuscular junction is where nerves and muscle fibers meet. It is an essential synapse that enables the nervous system and muscles to communicate. Improper function of these junctions can cause neuromuscular disorders, such as Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS), conditions for which effective treatments remain elusive.
A human self-organizing 2D neuromuscular junction model. Immunofluorescence analysis of the whole dish shows muscle cells (magenta) organized in bundles surrounded by spinal cord neurons (cyan).
Cells in both the 2D and 3D models self-organize into functional neuromuscular junctions, explains Lahmann, and thus mimic neuromuscular development in the living organism. “With the BMBF support, we can move to the next step of applying advanced human neuromuscular in-vitro models to clinical settings. Our ultimate goal is to improve patients' lives by accelerating drug development and identifying effective treatments.”
A tool to assess immune fitness and cancer risk
Primary immunodeficiencies (PIDs) are congenital diseases of the immune system. They are difficult to diagnose because only a fraction are caused by mutations in a single gene. Likewise, only 10% of inherited cancers can be linked to a single gene mutation.
Clara Vázquez García
Dr. Clara Vázquez García, a post doc in the laboratory of Professor Kathrin de la Rosa, has been awarded €95,000 to explore whether a blood-based biomarker can help diagnose PIDs and cancer susceptibility.
She and her project team have developed a new test that analyzes a small portion of genetic material from B cells circulating in blood. Specifically, the test analyzes more than 70 genetic features related to a person’s DNA repair machinery and immune system to assess whether they are functioning well.
The results can, for example, help predict whether a person is at high risk of developing cancer and diagnose and stratify people with immunodeficiencies. Currently, the only alternative test to identify mutations scattered across a patient’s genome is to sequence the entire genome of patients, which is prohibitively expensive, says Vázquez García. “We aim to develop an affordable test that can give deep genomic insight and provide people with better access to personalized treatments.”
Kathrin de la Rosa heads a Max Delbrück Center guest group, and a research group at the Centre for Individualised Infection Medicine, a joint venture between the Helmholtz-Centre for Infection Research and the Hannover Medical School. Vázquez García’s project arose from a fruitful collaboration among researchers at both the Max Delbrück Center and researchers from the Berlin Institute of Health at Charité, which was funded by the Stiftung Charité..
Text: Gunjan Sinha
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Contact
Gunjan Sinha
Editor, Communications
Max Delbrück Center
+49 30 9406-2118
Gunjan.Sinha@mdc-berlin.de or presse@mdc-berlin.de
- Max Delbrück Center
The Max Delbrück Center for Molecular Medicine in the Helmholtz Association (Max Delbrück Center) is one of the world’s leading biomedical research institutions. Max Delbrück, a Berlin native, was a Nobel laureate and one of the founders of molecular biology. At the locations in Berlin-Buch and Mitte, researchers from some 70 countries study human biology – investigating the foundations of life from its most elementary building blocks to systems-wide mechanisms. By understanding what regulates or disrupts the dynamic equilibrium of a cell, an organ, or the entire body, we can prevent diseases, diagnose them earlier, and stop their progression with tailored therapies. Patients should be able to benefit as soon as possible from basic research discoveries. This is why the Max Delbrück Center supports spin-off creation and participates in collaborative networks. It works in close partnership with Charité – Universitätsmedizin Berlin in the jointly-run Experimental and Clinical Research Center (ECRC), the Berlin Institute of Health (BIH) at Charité, and the German Center for Cardiovascular Research (DZHK). Founded in 1992, the Max Delbrück Center today employs 1,800 people and is 90 percent funded by the German federal government and 10 percent by the State of Berlin.
