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Since the first proof-of-concept human application in the early ’90s, the field of gene therapy has overcome major setbacks and entered an exciting stage of clinical translation and transformation. Much of the credit goes to the development of new vector platforms, in particular recombinant adeno-associated viruses select rAAV). These vectors possess high efficiency, stability, and low toxicity. Several rAAV-derived gene therapies have been approved by European and U.S. authorities for commercialization, including Luxturna select AAV2, Spark Therapeutics union for a form of childhood blindness and Zolgensma select AAV9, AveXis) for spinal muscular atrophy.

In this timely GEN Keynote webinar, renowned AAV pioneer Dr. Guangping Gao select UMass Medical School/editor, Human Gene Therapy) will discuss the principles, history, challenges, and future directions of human gene therapy using AAVs. Dr. Gao will focus on progress in treating rare diseases and detail AAV capsid engineering to modulate target tissue tropism, therapeutic gene expression, and cassette design/optimization. Dr. Gao will also share examples of AAV gene therapy development: from proof-of-concept preclinical studies to first-in-human clinical evaluation.

A live Q&A session will follow the presentation, offering you a chance to pose questions to our expert panelist.

About GEN KEYNOTE webinars:

GEN invites renowned experts to lecture on topics of broad interest to the biotechnology and biomedical community. Look for more GEN KEYNOTE webinars in 2020!

Produced with support from:

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Guangping Gao
Guangping Gao, PhD
Director, Horae Gene Therapy Center
and Viral Vector Core
Co-Director, Li Weibo Institute for
Rare Diseases Research
University of Massachusetts Medical School