Gene therapy, which is the controlled replacement or repair of defective gene variants that pose a health threat, is one of the highest profile future technologies in global medical research. A high transduction rate is crucial for its success. This means the greatest possible efficiency when addressing cells in the body and genetically changing them in the desired way. The bottleneck here is usually the access to the cells: In the blood and primary cells that are important for the treatment, the success rates with traditional methods usually fall below 30 percent.
Experts at the Institute of Radiation Biology (ISB) at the Helmholtz Zentrum München and SIRION Biotech GmbH from Martinsried have therefore developed an efficient method to overcome this critical hurdle. By using so-called lentiviruses and new substances called non-toxic poloxamers, they were able to increase the success rate to over 80 percent. This method, called LentiBOOSTTM, significantly moves gene therapy forward on its way to market approval, according to the start-up company in a recent press release.
In dealings with various blood cells, academic institutions have already confirmed the efficiency of the technology, which was developed with substantial contributions from ISB scientists Dr. Nataša Anastasov, Dr. Ines Höfig and Prof. Michael J. Atkinson. The LentiBOOSTTM strategy is also already generating a great deal of interest in the industry. USA supervisory authorities recently gave the go-ahead for use of this so-called “Munich solution” in a broad-based phase III clinical study. SIRION Biotech accordingly received the first substantial, seven-digit milestone payment, which will also profit the Helmholtz Zentrum München thanks to a utilization agreement negotiated through Ascenion.
The use of lentiviruses in gene therapy is relevant for numerous approaches for the future, such as with the use of stem or CAR-T cells*. “We are really very happy that the technology has developed so well and that we can contribute to new therapeutic approaches,” says Nataša Anastasov, project leader on the part of the Helmholtz Zentrum München.