Overcoming Bottlenecks in AAV Manufacturing for Gene Therapy

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EXPERT INSIGHT

Jacob Smith, Josh Grieger & R Jude Samulski

Latest advances in BIOPROCESSING: overcoming bottlenecks

Recombinant adeno-associated viral vectors (rAAV) have emerged as one of the most powerful tools for gene delivery to treat human disease. Significant advancements in vector development and manufacturing methods, along with promising results in the clinic, has initiated an extraordinary interest in drug development for gene therapies and commercialization. It is an exciting time for AAV-mediated gene therapy; in 2012, the field witnessed the ap-proved use of the gene therapy product, Glybera, for the treatment of lipoprotein lipase deficiency by the European Medicines Agency, and in 2017, the approval of Luxturna, for the treatment of Leber’s Congenital Amaurosis by the US FDA, thus arriving at the threshold of gene therapies reaching patients. Still, with an increasing number of gene therapy programs and a growing de-mand for both pre-clinical and clinical grade vector, the industry must be prepared to implement practical solutions to address the manufacturing bottlenecks and rigorous product characterization needed to meet higher regulatory requirements earlier in clinical development in order to efficiently advance and market these products. Below is an overview of the current status of rAAV vec-tor production from academic to contract manufacturing organization (CMO).

Submitted for peer review: 23 Aug 2018 Published: 6 Nov 2018
DOI: 10.18609/cgti.2018.083
Citation: Cell Gene Therapy Insights2018; 4(8), 815-827.
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