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Abstract
Introduction: Recent success in gene therapy of certain monogenic diseases in the clinic has infused enthusiasm into the continued development of recombinant adeno-associated viral (AAV) vectors as next-generation biologics. However, progress in clinical trials has also highlighted the challenges posed by the host humoral immune response to AAV vectors. Specifically, while pre-existing neutralizing antibodies (NAbs) limit the cohort of eligible patients, NAb generation following treatment prevents vector re-dosing.
Areas covered: In this review, we discuss a spectrum of complementary strategies that can help circumvent the host humoral immune response to AAV.
Expert opinion: Specifically, we present a dual perspective, that is, vector versus host, and highlight the clinical attributes, potential caveats and limitations as well as complementarity associated with the various approaches.
Declaration of interest
A Asokan is a consultant to Genzyme and Voyager Therapeutics and an inventor on patents licensed by the University of North Carolina to Asklepios Biopharmaceutical, Inc. Funding was received from the National Institute of Health (NIH). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending or royalties.
Notes
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