uniQure Developing Method for Re-administration of Gene Therapy

One of the issues with gene therapy using AAV vectors is that patients develop neutralizing antibodies against the vector, which may keep the patient from receiving a second dose of the gene therapy treatment, if needed.  Other patients may have anti-AAV antibodies from naturally acquired infections that could keep them from receiving a first dose.  uniQure has shown that in non-human primates, an immuno-adsorption technique can reduce the level of anti-AAV antibodies enough that a factor IX AAV vector can be successfully re-administered.  This has the potential to extend the applicability of gene therapy to nearly all patients regardless of their antibody status.