What’s holding up gene therapy? Making drugs on a large scale

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As interest in gene therapy grows, manufacturers face physical, biological, and engineering challenges to developing and producing drugs on a larger scale. “For early-stage Phase I trials, little material is required, but for Phase II and Phase III trials, or a licensed product, you need large-scale production equipment,” says Farzin Farzaneh, PhD, professor of molecular medicine at King’s College London.

Farzaneh’s lab participates in extensive academic and industrial collaborations, including projects to accelerate the development and clinical translation of viral vectors. According to him, a major challenge is the limited availability (and expense) of facilities that satisfy current good manufacturing practice (cGMP) requirements. Part of his lab’s collaborative work is developing closed manufacturing systems, which avoid problems with environmental and other contamination.

Related article:  Will 2020 see the debut of promising gene therapy for hemophilia A? It's up to the FDA.

Manufacturing challenges are being addressed not only by academically oriented collaborations, such as those involving the Farzaneh lab, but also by collaborations between companies. For example, Symbiosis Pharmaceutical Services and Cobra Biologics recently completed a project to bring together expertise in viral vector and drug manufacturing.

If such production-enhancing innovations succeed, they will help ensure a bright future for gene therapy.

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