Author Archives: Alengo Nyamay'antu

Working Toward an AAV Platform: Challenges, Threats, and Opportunities

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Recent successes in gene therapies are driving continued expansion of the pipeline. The number of clinical trials using adenoassociated virus (AAV) and lentivirus (LV) vectors is growing each year. Currently underway are 669 AAV and 266 LV trials in the United States, 1,250 AAV and 94 LV trials in Europe, and 223 AAV and 140 LV trials in the Asia–Pacific region (1). And as target indications broaden, many candidates treat conditions that affect much larger patient populations than before, calling for…

October 20, 2023

Optimizing the AAV Transfection Process in Suspension Cells

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Given the potentially curative nature of gene and gene-modified cell therapies and the successful launches of several such products over the past five years, markets and investments are growing significantly in the sector. In the United States alone, the US Food and Drug Administration has granted approval for several genetic therapies, including Strimvelis (autologous CD34+, developed by GlaxoSmithKline and later sold to Orchard Therapeutics) in 2016 Luxturna (voretigene neparvovec, Spark Therapeutics), Yescarta (axicabtagene ciloleucel, Kite Pharma/Gilead), and Kymriah (tisagenlecleucel, Novartis)…

October 29, 2021

Transfection Best Practices for AAV Gene Therapy Programs

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As viral vectors continue to push gene therapy innovations closer to market, many researchers are setting their sights on optimizing transfection, the process of delivering corrective genetic material into cells. It’s not just a question of how to transfect them, but also how to do so efficiently and at high volumes. Approaches that work for one cell line might not perform well for others, and transfection protocols can have different implications for scalability and cost during production for clinical trials.…

October 28, 2021