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Gene therapies are maturing rapidly with an increasing number of these treatments gaining regulatory approval in recent years. Viral vectors, such as adeno-associated viruses (AAVs), have emerged as one of the most used gene delivery vehicles in many disease contexts. Their unique features make them suitable vectors for in vivo gene therapies for various conditions including ophthalmological, metabolic, hematological, and neurological diseases.

In this GEN webinar, our expert speakers will discuss strategies and tools for designing optimal viral vectors for gene therapy delivery. They will present an integrated platform designed to support the discovery stages of a viral gene therapy program. During the webinar, you’ll learn:

• How WuXi AppTec can help to engineer and optimize viral vectors, including rationalized promoter design and transcription unit optimization.

• Fast, scalable, cost-effective viral vector production methods using industry standard transient transfection, packaging cell lines, and the novel TESSA technology.

• How to utilize a range of in vitro and in vivo assays for efficacy and safety evaluation.

A live Q&A session followed the presentation, offering a chance to pose questions to our expert panelists.

Weiheng Su
Weiheng Su, PhD
Head of AAV
OXGENE, a WuXi Advanced Therapy company
Qiong Zhou
Qiong Zhou, PhD
Executive Director, Discovery Biology
WuXi AppTec