A Treatment Challenge
Today, gene therapies are dosed using a type of programmable virus known as AAV. While effective in delivering treatments, the use of AAV (adeno-associated virus) comes with a systemic challenge: Up to a third of patients have pre-existing, naturally acquired antibodies to these viruses, called neutralizing antibodies (NAbs).
This natural defense mechanism limits the use of AAVs to deliver therapeutic doses of gene editing or replacement treatments, since the body will form an immune response. In addition, the formation of NAbs following AAV-delivered therapeutics currently limits these approaches to a one-time use.
What Are We Doing About It
Our Promising Solution
We’re working to solve this systemic challenge by enrolling patients in our NAbs research study, empowering them with the information, and utilizing those data to employ new technologies to eliminate the NAbs barrier.
Learn from the Literature
Start a NAbs Study
Learn how to join CRD’s NAb research study to obtain a screening kit for a patient. Participating in this study will allow researchers to better understand NAbs and develop tools to address high NAbs so all patients can potentially receive AAV-based gene therapies.