Technologies to evade and overcome pre-existing immunity to AAV vectors are needed to treat more patients and allow future re-administration of gene therapies.
Developing techniques to evade AAV neutralization is critical for expanding the efficacy and re-administration potential of gene therapies for monogenic disorders.
Adeno-associated virus (AAV)-derived viral vectors are a promising platform for the delivery of curative, life-changing therapies to a huge number of patients with monogenic disorders. There are currently over 250 clinical trials ongoing worldwide. However, for these therapies to benefit as many patients as possible, techniques must be developed to treat those with pre-existing immunity and to potentially allow re-administration of a dose in the future, should efficacy wane over time. This review discusses the current state and prospects of technologies to evade and overcome these immune responses and allow successful treatment of the greatest number of patients possible.
Earley et al. (Fri,) conducted a review in Monogenic disorders. Adeno-associated virus (AAV)-derived viral vectors was evaluated. Technologies to evade and overcome pre-existing immunity to AAV vectors are needed to treat more patients and allow future re-administration of gene therapies.