The field of Cell and Gene Therapy has witnessed expanding numbers of regulatory approvals in recent years. Of the approved products, a significant number are viral vector-based, either employing the viral vector for the delivery of a therapeutic transgene to cells ex vivo during the manufacturing process (e.g., LVV for modified cells), or via direct administration to the patient (e.g., AAV-based gene therapy product) to achieve a therapeutic effect. Given the rate of recent approvals, coupled with technological advances in molecular sciences, and partly driven by evolving regulatory expectations around controls, there is an evolution of product understanding, particularly in the application of analytical methods used to support process development and characterize or monitor product attributes. The aim of this session is to examine these changes in greater detail, including analytical method choices for optimal assessment of product characteristics, attribute criticality and monitoring. This may also include innovative approaches to determination of potency, including the use of surrogate metrics in the context of a potency assay matrix. A greater scientific understanding of viral vector-based Gene Therapy product attributes will improve the overall quality of these important therapeutic products.

Session Speakers:

LYFGENIA's Journey: Lessons Learned in the Development of LVV-based Cell & Gene Therapy Products

Marc d'Anjou, bluebird bio, Inc.

Potency Method Development, Bridging and Control Strategies for AAV Gene Therapy

Ping Carlson, Passage Bio

Gene Therapy Product Analytics: Potency Method Validation and Optimization

Wandong Zhang, BioMarin Pharamceutical Inc.

Additional Panelist:

Jessica Chery, CBER, FDA

Christopher Storbeck

Appearances