Turning obstacles into opportunities: overcoming efficiency and quality challenges in gene therapy development

The landscape of gene therapy is rapidly evolving, driven by a proactive regulatory environment and significant technological advancements. The U.S. FDA’s record-breaking approval of biologics in 2023 and the anticipated “breakout year” for 2024 underscore this momentum. However, these advancements come with pressures for manufacturers to expedite market entry while managing scale-up efficiencies and emerging challenges.

Scaling up gene therapy production from small to large volumes introduces significant challenges, such as ensuring viral vector quality, maximizing therapeutic cargo, and minimizing product loss. Technological advancements often outpace regulatory approvals, complicating the adoption of new, more efficient processes. Standardization in the industry remains critical, with consistent methods for characterizing viral vectors and assessing potency being essential for quality control and regulatory compliance.

This article, originally published in European Biomanufacturing Review, discusses how collaboration and innovation are key to overcoming these challenges, with a focus on process optimization to enhance production efficiency and quality. Efforts to develop stable plasmid incorporation, outsource manufacturing processes, and advancements in filtration and platform purification are enhancing production efficiency and quality. 

Addressing these opportunities in gene therapy, and finding better characterization solutions using platform technologies are paramount because failure to do so can result in serious adverse events or lead to trial failure of effective therapeutics due to manufacturing shortcomings. 

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