Summary:
Gene therapy promises to treat and potentially cure a disease by correcting its underlying genetic cause when current treatment options are limited or ineffective. While this proposition has exciting potential — and has shown encouraging results with a few gene therapy treatments approved by the FDA and/or the EMA that utilize AAV vectors — it still needs to overcome challenges with the safety and efficacy of the treatment. Particularly for AAV-based gene therapy, the key issues exist in the setup of the analytical characterization strategy of the viral vectors, characterization, and quality control parameters. This webinar will provide an overview of analytical challenges, delve into various tools to overcome those challenges, and compare different techniques used for AAV-based gene therapies.

Agenda:
Moderator Opening Remarks
Patricia Piatti, PhD, Senior Manager, Product Marketing at NanoTemper Technologies

Talk 1: Challenges and Considerations in AAV Characterization and Development
Santoshkumar L. Khatwani, PhD, Associate Director, Analytical Development, Sangamo Therapeutics

This talk will cover the following topics:

1. Analytical characterization of AAV
2. Challenges faced by cell and gene therapy community for AAV characterization
3. Two case studies

Talk 2: LC-MS Characterization of AAV Capsid Proteins
Xiaoying Jin, PhD, Senior Principal Scientist, Sanofi

AAV capsid proteins (VPs) are critical for viral infectivity and vector potency. Thus, complete characterization of the constituent viral capsid proteins of AAV vectors, including their sequences and post-translational modifications (PTMs), is highly recommended to ensure AAV product quality and consistency in gene therapy development. The development of advanced analytical methods for characterization of AAV viral capsid proteins (VPs), including LC/MS and LC/MS/MS, allows for the identification and quantification of post-translational modifications to support batch characterization/comparability,  understand structure and function relationship and support process development.

Q&A Discussion

Speakers:

Santoshkumar L. Khatwani, PhD
Associate Director, Analytical Development, Sangamo Therapeutics

Santoshkumar Khatwani received a Master’s degree in Organic chemistry in India. In addition, he obtained a Ph.D. degree in chemistry from the University of Kentucky in 2010. He joined the University of Minnesota for Postdoctoral training in the Department of Chemistry until 2012. Later, he joined BioVision Inc where he built a team focused on designing, manufacturing, and analytics for metabolic enzymes for commercial use. He pivoted to gene therapy when he joined Brammer Bio (now part of Thermofisher Viral Vector Services) as principal scientist and led the team for developing analytical assays for viral vectors. In 2018, he joined Sangamo Therapeutics as a Senior Scientist to help to build an analytical team for developing assays for viral vectors. He left Sangamo to join Asklepios Biopharmaceuticals (North Carolina) as the Director of Analytical Development but after a short duration rejoined Sangamo Therapeutics as an Associate Director where he currently oversees the analytical development for early and late phase viral vectors.

Xiaoying Jin, PhD
Senior Principal Scientist, Sanofi

Xiaoying Jin has been working at Genzyme/Sanofi for 20 years after she obtained her Ph.D. from the University of Michigan. There, she leads a group specialized in protein characterization of recombinant glycoproteins, antibodies, and adeno-associated virus vectors to support development including comparability studies, structure and function relationship studies, reference standard qualification, and forced degradation studies for CMC filings.

Moderator:

Patricia Piatti, PhD
Senior Manager, Product Marketing at Nano Temper Technologies

Patricia Piatti is a Senior Product Marketing Manager at NanoTemper. Before starting her nine-year-long career in Marketing, Patricia obtained a Ph.D. degree from the University of Buenos Aires, Argentina with a thesis she wrote on foot-and-mouth disease virus while she was a fellow at the USDA/ARS Plum Island Animal Disease Center. She then was a postdoc at UCI that focused on mouse polyomavirus and briefly worked at the Vector Center developing adeno-associated viral vectors.

Who Should Attend:

Method Development and Qualification

Analytical Development

Analytical & CMC Operations

Formulation Development

Gene Therapy