Advancement and Evolution

November 20, 2024 by Aldevron

Looking ahead to the journey of cell and gene therapy

As someone who lives and breathes cell and gene therapy, learning about new technologies is not only exciting, but a reminder of just how fast our industry is evolving and how much potential we have to change the world. Seeing that potential during Biotech Week Boston in September left us feeling truly energized and inspired by the amazing people, cutting-edge technologies, and groundbreaking innovations we engaged with, showing where personal journies could lead.

A New Era: Cell-free DNA Platforms
One of the most exhilarating advancements we learned about is the rise of cell-free DNA technologies. Generation Bio’s cell-free platform stood out, offering a non-viral, scalable approach to gene therapy using closed-ended DNA (ceDNA).

This platform bypasses the limitations of viral vectors by utilizing a proprietary rapid enzymatic synthesis (RES) method, which allows for efficient, large-scale production of DNA. It has the potential to deliver durable, redosable therapies to a broader range of patients, revolutionizing how we approach genetic treatments. It’s hard not to be excited when you see what these technologies can do.

AAV Manufacturing: The Push for Efficiency and Scale
Another huge takeaway was the relentless drive to improve adeno-associated virus (AAV) manufacturing. From optimizing viral titers to improving full-to-empty capsid ratios, the work being done to scale up AAV-based therapies is nothing short of remarkable.

As more companies move toward developing off-the-shelf gene therapies, the demand for efficient, reliable production methods is growing exponentially. This provides companies such as Aldevron, with its Nanoplasmid^TM technology, an opportunity to help developers improve their viral vector production and make it easier to scale up AAV-based treatments.

People, Conversations, Ideas
Beyond the incredible technology, meaningful conversations with industry thought leaders sparked new excitement for the future of cell and gene therapy. One conversation that resonated focused on integrating process development thinking into the drug discovery phase.

By prioritizing consistency, productivity, and scalability from the start, we can avoid complications in later development stages. It requires upfront effort from research teams, but the long-term benefits in efficiency are undeniable. You can learn more about those benefits in our newest whitepaper, Set Your Cell and Gene Therapy Program Up for Success from Day One.

What’s Next?
As we look ahead, we feel a great sense of excitement about what’s to come. Whether it’s advancing non-viral delivery systems, fine-tuning AAV manufacturing, or embracing the revolutionary potential of cell-free DNA platforms, the future of bioprocessing is bright.

Cell-free technologies are pushing the boundaries of what’s possible, with these platforms offering the potential for faster, scalable, and cleaner approaches to gene therapy. As they continue to evolve, we see them becoming a cornerstone of how we develop and deliver transformative therapies.

As said at the beginning, the potential for the future seen at Biotech Week Boston gave additional excitement to take what was learned and apply it to the work being done at Aldevron. Together, we’re helping shape the future of cell and gene therapy, making strides toward a world where transformative therapies are within reach for all.

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