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14 BioPharm International ® Emerging Therapies eBook September 2023 www.biopharminternational.com C ell and gene therapies (CGTs) can potential- ly treat, and sometimes even cure, a wide range of genetic and acquired diseases rang- ing from immunodeficiencies to cancer (1). For this reason, the field has been growing at an astro- nomical rate. Case-in-point: while there were only 27 FDA-approved CGTs at the end of 2022, ClinicalTrials. gov now cites more than 1500 ongoing clinical trials. Having impeccable data about the makeup of a new therapeutic is vital to successful clinical trials that take a new drug candidate one step closer to FDA ap- proval. But the myriad designs of CGTs in development today make that uniquely challenging. In this article, the author will explore some of the specific hurdles associated with lentiviral CGTs. Many approved, and investigational CGTs alter the target cells using a viral vector. Infection delivers a genetic payload that may repair a damaged or defective gene or supercharge the cells to target a tumor within the body. While different viruses can be used, lentiviral vectors are a go-to tool for the stable insertion and ex- pression of genes in CGT therapies designed to treat diseases resulting from a single mutation. They're also a preferred tool for modifying somatic and chimeric antigen T cell (CAR-T) cells (1). This article discusses the importance of accurately quantifying and monitoring the lentiviral load with different technology options, such as quantitative polymerase chain reaction (qPCR) and droplet digital PCR (ddPCR) technology. Technologies such as qPCR and ddPCR can illuminate the lentiviral titer and un- derscore the importance of correctly getting the len- tiviral copy number Lentivirus: advantages and challenges Across many indications, researchers have incorporated lentiviral vectors into their cell and gene therapies be- cause they offer numerous advantages. Lentiviral vec- tors can enable long-term transgene expression, with a packaging capacity of up to nine kilobases. This allows Navigating the Intricacies of Lentiviral CGTs Marwan Alsarraj is biopharma segment manager, Digital Biology Group, Bio-Rad. Scientists are leveraging ddPCR technology to get an accurate read on viral titer and vector copy number. BLUEBAY2014 - STOCK.ADOBE.COM