Inverted Terminal Repeats (ITRs) are DNA sequences flanking the transgene of interest to signal incorporation into adeno-associated virus (AAV) or genome insertion by transposases. Despite sharing the ITR element name, viral vector and transposon ITRs differ in sequence and important properties that need to be considered in handling these vectors.
In my previous discussions on the benefits of NanoplasmidTM vectors, I touched on eliminating protein and antibiotic markers and the advantage of the RNA-OUT marker system, as well as structured DNA repeats and the plasmid replication challenge.
In efforts to bring the next-generation of plasmid technology to the cell and gene therapy space, Aldevron is offering a giveaway to customers developing AAV based therapies. Nanoplasmid benefits in the context of AAV include improved ITR stabilization from its proprietary host strain while increasing plasmid yields compared to conventional plasmid vectors.
For decades, the same plasmid technology has been used in vaccination, cell and gene therapy, and as a raw material in viral vector and RNA production, but that technology often isn’t a good fit for the processes where it’s being used. Now, with the growing availability of Nanoplasmid™ vectors, that is changing.
NanoplasmidTM. We’ve been discussing this topic quite a bit recently, and now our latest whitepaper brings you an in-depth examination of our novel Next-Generation plasmid technology. The etiology of its name, “nano,” originates from its purposefully designed “small” backbone of around 500 base pairs (bp) as compared to traditional plasmids that often exceed 2000 bp. This reduced backbone size is a direct result of removing prokaryotic and antibiotic-resistance genes used to select transformed bacteria and propagate a vector.
As one of the most significant discoveries in gene and cell therapy, CRISPR/Cas9 genome editing systems continue to evolve. In this webinar, “Genome Editing Tools: Beyond Discovery,” Aldevron experts Dr. Jim Williams and Max Sellman detail innovative tools to support gene editing clinical programs, highlighting Nanoplasmids as an HDR template paired with Cas9 nucleases in CAR-T applications.
Can a small plasmid provide big benefits for research and development? That was the question NanoplasmidTM inventor, Jim Williams, Ph.D., summarized in this webinar and panel discussion. Dr. Williams was joined by an experienced Aldevron team to answer questions about the combined power of Nanoplasmid technology and Aldevron manufacturing experience.
For decades, the cell and gene therapy industry has been using the same plasmid technology in vaccination, cell and gene therapy, and as a raw material in viral vector and RNA production. In some cases, that technology isn’t a good fit for the processes where it’s being used. Now, with the growing popularity of Nanoplasmid™ vectors, that is starting to change.
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.
