Gene therapy has been a promising technology for a long time. However, early setbacks and unclear regulations meant that many pharma companies looked elsewhere for new and promising drugs.

In recent years, however, gene therapy has made a comeback and has finally begun to deliver on its potential with the release of several FDA-approved gene therapy treatments for a variety of disorders.

However, as with all new and experimental treatments there are pitfalls that must be avoided and due to the challenging past there are issues to address. With Svar's Gene Therapy targeted product and Services we aim to aid in addressing and solving these issues and help our customers develop these new and exciting treatment options.

Neutralizing antibodies against viral vector

A threat to treatment efficacy and a potentially safety concern

Since most gene therapy projects use a recombinant Adeno-Associated Virus (AAV) vector, where some of the viral genome has been replaced by a therapeutic cargo, immunogenicity testing is especially important as patients that have been infected by AAV viruses previously may carry neutralizing antibodies that prevent the gene therapy vector from transfecting the host cell and delivering its cargo.

This would then eliminate any therapeutic effects and potentially lead to other more severe cases of unwanted immunogenetic effects.