What seemed a few years ago to be science fiction is now reality: cell and gene therapy play an important role in the treatment of diseases such as cancer. However, firstgeneration gene therapy approaches are based on viral gene transfer systems, which can lead to critical side effects in some cases. They are also complicated and expensive to produce, which limits their suitability for routine use in large groups of patients.
The ‘Sleeping-Beauty’ transposon system developed at the Max Delbrück Center for Molecular Medicine (MDC) by Dr Zsuzsanna Izsvák and Prof. Dr Zoltán Ivics (now at the Paul-Ehrlich-Institut) offers an attractive alternative. Transposons are ‘jumping genes’: DNA elements that can change their position in the genome. These non-viral gene vectors are cheaper to produce and simpler to use than viral gene transfer systems, and have been shown to be considerably more effective in many studies conducted in various model systems. The first clinical trial of the most recent – and most effective – member of the Sleeping Beauty generation (SB100X) begins in 2021.
Ascenion has been working closely with the inventors for many years to secure IP and commercialize the system. Demand is rising rapidly. The team has supported numerous licencing agreements for various applications of SB100X:
– Cell and gene therapy, in particular adoptive (CAR) T cell therapies
– Production of recombinant proteins, such as antibodies
– R&D tool for simple and rapid production of stable genetically modified cells
(Annual Review 2020)
