Join us for BEBPA’s 14th Annual EUR Bioassay Conference consisting of four days of cutting-edge presentations, invigorating panel discussions, and insightful Q&A sessions. Each day will feature a specific topic. This year’s topics include:
- Developing potency bioassays
- Dealing with “unorthodox” dose-response curves
- Automation and novel statistical approaches
- Product specific case studies
One of our very own scientists, Ulrike Herbrand, will discuss gene therapy products as promising drugs in the treatment of hereditary diseases or tumors.
Scientist, Charles River Laboratories
The New Bioassay Challenge: Gene Therapy Products
Gene therapy products are promising drugs e.g. for the treatment of hereditary diseases or tumors. For the correction of such altered genes or site-specific modifications different methods exist. Transfer of therapeutic nucleic acids into human somatic cells can be achieved for example via transfection by means of small-interfering RNAs (siRNA, which is not a gene therapy product, but requires similar testing) or via transduction by means of recombinant lentiviral (LV) or adeno-associated viral (AAV) vectors as the most commonly used procedure. The latter method takes advantage of the natural high effective ability of viruses to transfer their genetic material into cells. During development and for QC release of such gene therapy products, in addition to the detection of the gene product at the transcriptional level (RNA) and translational level (protein), the functional bioactivity of the protein is of decisive importance. This mechanism of action (MoA) should be reflected in vitro using a functional bioassay, e.g. based on a reporter. In case studies the functionality of the above procedures was demonstrated for each tested gene therapy model using a specific target.