Addressing the challenges of purification and quality control in gene therapy

Akash Bhattacharya, Audrey Chang, Leisha Kopp, Klaus Richter, Shawn Sternisha

Recombinant adeno-associated virus (rAAV) vectors are a highly promising mechanism for therapeutic gene delivery. However, most industrial cell lines exhibit inefficient gene packaging, which results in a heterogeneous population of vectors composed of empty, partially-loaded, and fully-loaded capsids. Purifying, identifying and quantifying these different species is vital from a production and quality control standpoint. Improperly loaded viral capsids do not produce the desired therapeutic effect, but may still elicit an unintended immune response. This article and expert panel discussion will focus on a variety of pertinent topics in rAAV process development, with a focus on the benefits of analytical ultracentrifugation for vector purification and characterization.

Cell & Gene Therapy Insights Aug. 2021

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