Lentiviral vectors are one of the foundational technologies of cell and gene therapy. Along with adenoviral vectors, they are one of the most widely used means of introducing genetic information into cells. They are used in the lab, the manufacturing facility, and the clinic, particularly for the production of therapies for rare disease.
For example, the approved gene therapies and are made using lentiviral vectors as is Rocket Pharmaceutical’s leukocyte adhesion deficiency-1 candidate, . Yet, despite their importance to the industry, current vector production processes are far from optimal, says Adriano Leuzzi, head of process development at Italy-based manufacturing and development services firm, ReiThera. “The manufacturing challenges are mainly related to scalability, productivity—use of the right cell line, media, and transfection reagents that can effectively deliver the plasmids inside the cell at the right efficiency—and process yield,” he tells .
Lentiviral vectors are also highly sensitive to temperature and pH changes—as well as to damage caused by shear forces in culture. And this can be a major problem during process development, Leuzzi says. “All of these aspects need to be considered from the early stage of the development process up to the late stage of cGMP clinical production.
The goal is to build a robust and easily scalable manufacturing platform to target the highest performance process and meet the regulatory quality requirements needed to .
