Hereditary diseases are caused by alterations in the genome. Although modern gene-editing technologies can precisely correct such defects, a suitable system has so far been lacking to safely and efficiently transport the large enzymes required for this process into the affected cells. With the newly developed Synthetic Transfer Vehicle (STV) – a delivery vehicle composed of artificially designed proteins – this step can now be achieved for the first time in a cell-specific, efficient, and controlled manner.
“Our STV technology platform enables, for the first time, the targeted and programmable delivery of gene therapeutics into specific cells,” explains Dr. Christoph Gruber, project leader at the Institute of Stem Cell Research.
To advance this approach and prepare for the founding of a startup, Dr. Christoph Gruber, Dr. Florian Giesert, and Prof. Wolfgang Wurst have secured €3.1 million in funding from the BMFTR as part of the GO-Bio Next initiative – “Preclinical testing of a universal transfer vehicle for gene therapeutics (EDGE).” The project marks an important step toward translating the STV technology into clinical application and is part of a broader program aimed at developing a platform-based solution for targeted genetic corrections in hereditary diseases.
The technology enables significantly higher delivery efficiency compared to established methods such as lipid nanoparticles (LNPs) or virus-like particles (VLPs). In addition, the specificity of the vehicles can be precisely tailored to the target tissue, making STVs a highly efficient and versatile tool for gene therapy.
“With STVs, we are introducing an entirely new approach to the targeted delivery of therapeutics. They combine high efficiency with precise controllability and could thus form the basis for the next generation of gene therapies,” says Dr. Florian Giesert. The researchers are now preparing for preclinical testing, with the goal of transferring the approach to clinical development in the medium term.
“Our goal is to advance these technological innovations so that they directly benefit patients – enabling therapies that not only alleviate symptoms but can actually cure,” adds Prof. Wolfgang Wurst.
The technology platform aims to provide an end-to-end solution for the treatment of genetic disorders – from the targeted delivery of therapeutic enzymes to precise genetic correction in the target tissue. This could make it possible, for the first time, to develop broadly applicable, curative gene therapies for a wide range of hereditary diseases.
About the research group at the Institute of Stem Cell Research
The group led by Dr. Florian Giesert, Dr. Christoph Gruber, and Prof. Wolfgang Wurst uses methods from protein design and synthetic biology to develop innovative technologies in the field of gene and cell therapy. Their goal is to understand the molecular mechanisms of diseases and create new therapeutic approaches.
To the press release by the BMFTR (in German): EDGE bringt Gentherapien sicher dorthin, wo sie wirken - BMFTR GO-Bio
