With the use of gene therapy, defective genes can be replaced by functional genes, with the aim to restore normal function. Advanced therapeutic platforms like gene and cell therapies offer tremendous potential for treating these unmet medical needs. However, they are extremely challenging to develop successfully.
Svar have developed a technology, based upon the reporter gene assay format, that allows to assess and quantify the presence, activity, and function of a therapeutic AAV vector. This technology can be adapted for many applications throughout the drug development phases including drug manufacturing processes such as the quantification of vector potency and functionality or the detection of neutralizing anti-drug antibodies (NAb).
In this scientific poster, we showcase examples from the development, validation, and production of a proprietary developed reporter gene cell line – the iLite cGMP/GC and subsequent assay using it in an assay-ready format.