Nanoplasmid™ Vectors: The Power of Small
In cell and gene therapies, delivery is a critical factor. Regardless of your modality, optimizing payload, impact, and duration are common challenges. Countless programs, globally, are working on the delivery mechanism. However, many have overlooked a key component in improving therapeutic or manufacturing performance: the plasmid.
In his webinar, Advanced Plasmid Technology – Improving Safety and Performance, Dr. Venkata Indurthi, Aldevron’s chief scientific officer, highlighted several reasons why it makes sense to adopt a modern alternative to the traditional plasmid technology, which was designed initially for basic research more than 40 years ago.
By contrast, Nanoplasmid™ vectors have recently proven to be a transformative replacement in a wide variety of applications, offering greater safety and performance benefits. When it comes to safety, Dr. Indurthi pointed out three key benefits of the Nanoplasmid platform.
First, Nanoplasmid vectors use a patented non-antibiotic selection process. Removing the antibiotic resistance marker addresses a growing safety concern expressed by several agencies. As these therapies are designed for human consumption, removing the possibility of antibiotic resistance transfer is an important benefit.
Second, Nanoplasmids are replication-incompatible with negative organisms, essentially eliminating the possibility of unwanted propagation. And third, the small (<500 bp) backbone eliminates a lot of unnecessary DNA found in traditional backbones, which are typically >2000 bps. That unnecessary DNA from the traditional plasmids may later appear as a contaminant in validation experiments.
Dr. Indurthi spent much of the webinar sharing third-party published data highlighting several performance and manufacturing benefits offered by the Nanoplasmid platform. The small backbone is engineered to reduce plasmid-associated innate immune activation, which translates to much lower observed toxicity and vector inactivation during primary cell transfection.
In practice, the small Nanoplasmid backbone outperforms traditional plasmids and other technologies, though in differing ways by application. In a transposon vector case study, data shared by Poseida Therapeutics concluded that Nanoplasmid technology showed an increase in transposon frequency, decreased manufacturing timelines, robust expansion in the clinic, and several other benefits.
When considering AAV viral vector delivery, a key Nanoplasmid benefit is in manufacturing, where data shows as much as 5x plasmid yields, especially for the ITR containing transfer vector, which tends to be difficult to replicate.
Information for other applications was covered in the webinar as well, relying heavily on third-party published data. If you are working in the field of cell and gene therapy and are trying to improve safety, performance, or find cost-saving, Nanoplasmid may be right for you. You can learn more by viewing Dr. Indurthi’s webinar using the form below and downloading this list of Nanoplasmid publications.