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.
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 MoreRead more about the science behind the NAbs challenge, and opportunities to overcome it.
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
Read nowImmune responses to AAV vectors: overcoming barriers to successful gene therapy
Read nowRelationship Between Neutralizing Antibodies Against Adeno-Associated Virus in the Vitreous and Serum
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