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Challenges in gene therapy discovery and development


Charles River Laboratories

Gene therapy holds tremendous potential for revolutionizing the treatment of genetic disorders and other diseases. Among the numerous delivery systems explored, adeno-associated virus (AAV) has taken the lead in efficiently and safely delivering therapeutic genes to target cells.

However, AAV gene therapy faces developmental challenges, including anticipating and addressing immune responses, as well as the underlying complexity of targeted diseases. The functionality of AAV vectors is impacted not only by their design, but also by their scaling and manufacturing processes. This is why the design and development of new viral vectors should be approached as a continuum of interdependent activities, from understanding and controlling manufacturing procedures to analytical procedures used to determine purity and functionality.

This webcast will explore how best to address challenges at each stage of in vivo AAV gene therapy discovery and development, from plasmid development and manufacture to clinical production and first-in-human studies with defined regulatory requirements around vector production and the testing/release of material.

You will learn:

• Major challenges in gene therapy early-stage discovery

• The connectivity between discovery and clinical manufacturing

• Considerations for leveraging a platform approach to simplify manufacturing and phase-appropriate QC testing

Unable to join the live event? Watch on demand. Register now to ensure that you receive information on how to gain access after the live event.

This webcast has been produced by Charles River Laboratories, who retails sole responsibility for content. About this content.


Christina Fuentes, Senior Consultant, Dark Horse Consulting

Christina Fuentes

Christina Fuentes, Ph.D., is a Bioengineer with experience in gene and cell therapies. Dr. Fuentes' work with DHC clients has ranged from long-term embedded support to vendor management to technical lead from initial product design stages through to first-in-human/IND approval.

James Cody, Associate Director, Technical Sales and Evaluations, Charles River Laboratories

James Cody

James is a member of Charles River’s gene therapy CDMO business development team and is based at the viral vector manufacturing site in Rockville, MD. He has worked in the viral vector CDMO space since 2018, originally for Vigene Biosciences, which was acquired by Charles River in 2021. James obtained a Ph.D. and completed postdoctoral training in cancer gene therapy at the University of Alabama at Birmingham (UAB), studying oncolytic viruses. Afterward, he worked as a research scientist in virology, cell biology, cancer, and parasitology. In his current role as the Associate Director of Technical Sales and Evaluations, James provides technical support to the business development team, helping to guide client discussions and onboard new projects by collaborating with subject matter experts across various functional groups.


Nikki Forrester, Freelance Science Writer and Editor

Moderator Nikki Forrester

Nikki Forrester is a science journalist who covers biology, natural history, climate, and the culture of academic research. She earned a Ph.D. in ecology and evolutionary biology in 2019.


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