Introducing Lonza’s AAV suspension transient transfection platform to de-risk your path to clinic

Adeno-associated viral vector (AAV) is the most common delivery vehicle for potentially curative in vivo gene therapy. Following a couple of landmark approvals, this field has experienced accelerated pipeline growth and investor interest in the past 5 years. Currently, AAV therapies are predominantly targeting rare genetic disorders for which the patient populations are often limited. As a result, drug developers feel immense pressure to be the first to market and commercialize their therapies. In this article, we will focus on a robust, streamlined platform process for rapid production of AAV using Lonza’s suspension-adapted HEK293 cell line and proprietary plasmids to ensure high productivity, and in-process analytics enabling enhanced full-to-empty capsid ratio. The approach for building a reliable, de-risked path to the clinic to avoid unforeseen costs and compliance-related delays will be discussed.

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