The vision of introducing genes into human cells to heal life-threatening diseases is more than 50 years old. Today, the first gene therapies are approved. Transduction – the successful transfer of genes into the target cells via viral shuttle-systems – remains, however, a critical bottle-neck. Frequently only a small proportion of the desired cells are reached, particularly in the case of immune or stem cells, which do not divide and are therefore particularly difficult to transfect. But it is exactly this type of cell that is of the greatest therapeutic importance. LentiBOOST an adjuvant jointly developed by the Helmholtz Zentrum München and SIRION Biotech, addresses exactly this problem. It can increase the efficiency with which lentiviral vectors introduce gene material into human cells from the approx. 30% currently achieved to up to 90% – even in resting cells. A US licensing partner is currently investigating the adjuvant in a gene therapy study. With the approval of the study by the regulatory authority FDA, an initial milestone payment to SIRION Biotech became due, from which the Helmholtz Zentrum München also profits. Ascenion has been working with the SIRION–Helmholtz team for some time already, and has structured appropriate cooperation and licensing agreements between the partners and the licensees.
(Annual Review 2017)
