Prof. Dr. Ulrich Siler,

• Lecturer in Gene and Cell Therapeutic Systems

Research

In vivo Gene Delivery

Gene therapy represents a treatment option which allows to correct/compensate for the primary cause of a genetic disease. First clinical success in gene therapy was published in the year 2000. Since then, several gene therapy products such as Imlygic, Strimvelis, Kymriah, Luxturna and Zolgensma have received marketing approval, and others are about to be launched.

In ex vivo gene therapy, target cells are isolated from patient samples, genetically modified under GMP conditions, and then re-transplanted into the patient. In in vivo gene therapy, which targets the eye, brain or muscles for example, infectious particles are injected directly into the patient.

In ex vivo gene therapy, lentiviral vectors enveloped by vesicular stomatitis virus glycoprotein (VSVG) are state-of-the art and are applied ex vivo to target cells with proliferative potential (hematopoietic stem cells, T cells). While ex vivo gene therapy has established proof of principle, it faces an economic challenge: In addition to large-scale GMP-compliant vector production, personalized GMP-compliant ex vivo processing of patient cell increases the overall costs of treatment.

In vivo delivery would significantly reduce the total cost of treatment per patient.

Ulrich Siler is lecturer for Gene and Cell Therapeutic Systems at the University of Applied Arts and Sciences Northwestern Switzerland (FHNW) since 2022. With more than 18 years of hands-on experience in the development of retroviral vectors, in pre-clinal GLP-conform animal studies and in clinical gene therapy, his research now focuses now on in vivo gene delivery.

Prior to joining the Institute for Pharmaceutical Technology at FHNW, Ulrich Siler was heading a research lab first at University Children`s Hospital Zürich from 2004 to 2020 and subsequently at University of Zürich, focusing on gene therapy. There he was involved in the first temporarily successful clinical gene therapy trial to cure patients suffering from X-linked Chronic Granulomatous Disease (X-CGD). In parallel, he developed a lentiviral gene therapy vector for the treatment of patients suffering from p47phox-deficient form of CGD, which is nearing clinical application. In 2017, he completed the habilitation process at Medical Faculty of the University of Zürich with his inaugural lecture, which was awarded by the Venia Legendi for Pediatric Immunology, Experimental Immunology and Gene Therapy.

Ulrich Siler graduated in Biochemistry from the University of Tübingen (Germany) in 1997, where he also received his PhD in 2001 for his research on the extracellular matrix of human bone marrow in the working group of Prof. Gerd Klein. After his PhD, Dr. Siler spent three years as postdoctoral researcher at Roche Vitamins Ltd which became DSM Nutritional Products during this time. Thereafter, he left industry and returned to academia in 2004.

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• Master: Gene and Cell Therapeutic Systems

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