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.
Citation:
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.
https://doi.org/10.12665/J22OA.Sajj-Call
Posted online June 19, 2023.