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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Spotlight Article

Analytical Technology Used in the Latest Development of Gene Therapy Candidates

Vector Characterization & Validation

Zhenhong Li, Zhuchun Wu, Tomoko Maekawa, Win Den Cheung, Keith Webber, Li Zhi, Kevin O’Brian, Brian Howie, Claire Zhang, Bhargavi Kondragunta, Tristan Marshall, & Franz Gerner

Adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. In the last 3-5 years, significant resources have been invested in the development and evaluation of these therapies in clinical studies. Commercial products are emerging which provide significant benefit to people with debilitating diseases. These great strides forward in gene therapy are only possible with the support of sophisticated analytical technology. Some of the advancements in analysis of well-characterized biologics can be leveraged to further development in the molecular therapy arena. In addition, new analytical applications are needed to facilitate the accelerated development time lines often encountered in gene therapy and the rare disease space. This article captures the latest developments in analytical technology that provides key measurements and characterization support for the development of gene therapy products at REGENXBIO Inc.

DOI: 10.18609/cgti.2019.059
Citation: Cell & Gene Therapy Insights 2019; 5(4), 537-547.
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