Article Preview: Practical Considerations in Using an Equivalence Approach to Establish Lot Release Limits for Vector Dose

By Nancy Sajjadi and Janice Callahan
Volume 22, Open Access (June 2023)

The approval of several gene therapy products and gene-modified cell therapies over the last five years has led to increasing numbers of investigational new drug applications (INDs) using adeno-associated and lentiviral vectors. However, these successes have been tempered by the risks of dose-related toxicities. The therapeutic window for a product is derived from pre-clinical and clinical dose response models, which assume statistically that measurements of dose are exact. Whether vector is administered directly or used as a critical reagent to prepare a gene-modified cellular product, the assignment of a label concentration to a vector batch is critical for establishing consistency of product used in preclinical and clinical development.

Sajjadi N, Callahan J. Article preview: practical considerations in using an equivalence approach to establish lot release limits for vector dose. BioProcess J, 2023; 22.

Posted online June 19, 2023.