Overcoming Development And Manufacturing Challenges With Lentiviral Vectors

By Pratima Cherukuri, Chief Scientific Officer, Genezen

Up until the last few decades, human diseases were predominantly treated with small molecule drug therapies that were produced using standardized technologies. The effects of these drugs were also commonly short-lived within the body. With the dawn of the “biologics revolution” and significant advances in biotechnology in recent years, there has been a significant increase in the use of more targeted and personalized therapies to address complex diseases in the form of cell and gene therapies.

One type of cell and gene therapy, gene-modified cell therapy, uses methods where cells (e.g., T-cells or other blood cells) are extracted from the patient and genetically modified before being transfused back into the body. Currently, major applications of gene-modified cells aim to repair or replace defective cell populations; target cancer cells, pathogens, or infected cells; and modulate immune responses. This results in a personalized therapy and often relies on the use of viral vector technologies to facilitate gene editing. Unlike traditional small molecule drug therapies, cell and gene therapies have the potential to be longer-lasting and offer potentially curative effects on medical conditions that small molecule therapies have been previously unable to treat.

This executive summary covers a closer look at cell and gene therapy pipelines, choosing a viral vector, including adeno-associated virus, gamma retrovirus, or lentivirus, and current lentiviral trends.