However, developing gene therapy dugs is complicated and very expensive. Consequently, drug prices will be very high, typically in the region of hundreds of thousands of US Dollars per patient. Since the price is so high per patient, it becomes essential to administer the drug to only those patients that have a high probability of benefiting from the drug.
One of the most commonly used viral vectors in gene therapy is adeno-associated viruses (AAV). These vectors offer several benefits as they generate low toxicity and pathogenicity while providing long-term transgene expression. However, a large proportion of the population has been subjected to the AAV virus previously during their lifetime and has developed antibodies against the virus. The presence of such neutralizing antibodies (NAb) can completely abolish the effect of AAV-based gene therapy in those patients. Furthermore, NAbs can also develop upon administration of the drug, making repeated administration ineffective.
Clearly, screening patients for pre-existing NAbs is an important and necessary step to provide gene therapy to the right patients at the right time.
Svar is in a unique position to offer assistance in any gene therapy project. We have robust platforms suited for all phases of drug development, as well as the experience and knowledge from our diagnostic and bioanalytical services. We are currently developing cell-based assays for immunogenicity and potency testing. Furthermore, we can create custom projects tailored to your specific needs to create truly tailor-made assays. Our Bioanalytical Services offer flexible solutions adapted to your needs and requirements.
Together, we can move the promise of gene therapy to reality.