Gene therapy

Researchers led by Michael Kaminski report in “Molecular Therapy” that they have used base editing to repair mutations that cause ADPKD. The approach reduced liver cysts in mice.

Sarah Hedtrich heads a guest group at the Max Delbrück Center. The Johanna Quandt Professor at the Berlin Institute of Health at Charité (BIH) is working on organ-on-a-chip models to develop new treatment options for rare hereditary and inflammatory skin and lung diseases.

While CAR-T cells have proven successful in treating certain types of leukemia and lymphoma, these immunotherapies can become even more effective. A clinical trial run by Charité and the Max Delbrück Center has been awarded a grant of several million euros to test a new approach.

Michael Gotthardt’s research at the MDC looks at how the cardiovascular system and skeletal muscles function, with the ultimate goal of improving patients’ lives. To this end he is working to develop drugs for conditions such as heart failure with preserved ejection fraction, for which there is no effective therapy to date.

Wissensstadt Berlin 2021 kicked off in late June with an extensive program that offers a glimpse into what the city’s scientists are working on – all under the motto “Berlin wants to know.” The MDC is hosting events that bring science to life and give the public an opportunity to discuss the medicine of tomorrow.

Simone Spuler is looking to use stem cells and CRISPR to prevent the muscle tissue of patients with genetic muscle disorders from further deteriorating – or even to repair it. This could lead to the first therapeutic approaches for diseases that have up to now been considered incurable.

A young Berlin-based company called T-knife is a rising star in Germany’s biotech sector. Co-founder Dr. Elisa Kieback, along with colleagues, has catapulted the joint spin-off from the MDC and Charité to the top of the German biotech start-up universe.

Researchers from the MDC and Charité have developed a new gene therapy based around a special T-cell receptor that helps the immune system effectively recognize and fight cancer cells. The safety of the novel therapy is now being tested on patients with bone marrow cancer in a phase I clinical trial.

The Berlin biotech start-up T-knife, a spin-off from the MDC together with Charité, has secured investments totaling €66 million. Four venture capital funds committed to the financing in early August. T-knife develops new cancer treatments using modified immune system T cells.

Harnessing jumping genes to advance gene-therapy cancer treatment: In an effort to optimize the Sleeping Beauty system, the MDCell – Helmholtz Innovation Lab is extending its collaboration with PlasmidFactory.