Bioanalysis for Gene-edited Proteins in Target Cells and Tissues

The general principal of gene therapy is to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein. To be able to evaluate the gene therapy platform technology or the delivery and expression of a particular gene therapy product, we must be able to quantify and characterize the gene-edited proteins in targeted cells or tissues. The accurate bioanalysis results can help the researchers to monitor the gene-edited proteins over time after dosing and then guide decisions on lead selection and lead optimization.

Unlike other therapies, gene therapy often introduces a protein that is very similar to the endogenous abnormal protein, sometimes at low concentration. When ELISA reagents are not available, mass spectrometry comes in handy to differentiate the gene-edited protein versus the endogenous protein by monitoring specific peptides containing the mutated amino acids. At NovaBioAssays we have built a suite of analytical tools (as discussed in Non-regulated bioanalysis) to characterize and accurately quantify various level of the protein in different matrix. We can provide assays for both characterization and quantification of the gene-edited proteins. The characterization results are based on high resolution mass spectrometry and can inform us the mutations, truncations and PTMs of the protein.