Viruses make excellent vectors because they can infect hard-to-reach cells and predictably modify gene expression.Credit: Tumeggy/ Science Photo Library Getty Images
Cell and gene therapies are changing lives. They engineer patients’ cells or DNA to create personalized therapies, unlocking long-lasting or even curative treatments for diseases with high unmet needs, from cancer to rare genetic disorders.
Currently, these treatments are complex and costly to produce. This translates to high prices, and limits access for patients. “Scientists in academia and industry are working diligently to reduce the cost of these treatments and make access more equitable,” says Amitabha Deb, senior vice president of process and analytical development at viral vector manufacturer iVexSol, which uses its lentiviral vector technology to reduce the time and expense involved in cell and gene therapy development.
At the heart of gene therapy is the process of reprogramming cells, using genetic instructions delivered by vehicles called viral vectors. Viruses, such as adeno-associated virus and lentivirus, are very efficient vectors because they can infect hard-to-reach cells and predictably modify gene expression. Producing viral vectors more efficiently is central to accelerating the manufacturing process, making the final product more affordable to patients and health systems.
Cost-effective and reliable vectors
With any new therapy, winning approval is only part of the battle. Once a treatment starts scaling up for clinical use, any gap in vector production could bring costly delays, both for developers expecting a return on their investment, and for patients waiting to benefit from an effective new treatment. Investing in early process development is crucial.
A potential bottleneck is transfection, where genetic material is introduced into the host cells, leading to the production of viruses. This stage is enhanced by using the right transfection reagent to optimize the introduction of genetic material, along with other production parameters, to ensure process scalability, reliability and cost-effectiveness. This improves the odds of achieving high physical and functional titres, and increases the percentage of viral capsids that are filled with therapeutic cargo. “In producing any viral vector, the transfection reagent needs to guarantee both high titre and infectivity, as well as quality, to satisfy the needs of a particular disease indication,” says Deb. “It must also be suitable for future GMP production.”
Working with expert partners helps reduce process development time and accelerate the journey towards the clinic. Reagent kits can make the transfection process reliable, scalable, cost-effective and standardized — key considerations for viral vector manufacturing. “Without a partner with a deep understanding of the process and product, efforts to reduce costs will be in vain,” says Deb.
iVexSol has a strong relationship with US-based Mirus Bio, which has more than 20 years of experience developing technologies for cell and gene therapy delivery. Mirus offers solutions to increase process efficiency for viral vector production, and a unique reagent formulation that aims to boost virus quality and functional titres compared to traditional polymer-based options. “We have tested many transfection reagents and Mirus gives us the best results, yielding more infectious viral particles,” says Deb.
Finding the optimal approach to viral vector production can make the difference between success and failure for a new therapy. Efficient production will be reflected in the quality and reach of the final product. “The most important thing is to reduce costs so ultimately your medicines will cost less,” says Deb. “Yes, these medicines have unprecedented clinical outcomes, but they need to be accessible to the broadest patient population.”