News concerning cell and gene therapy (CGT) may make you feel like you are on a rollercoaster. The ride moves very quickly with a lot of ups and downs, plus a few unexpected turns. As we move through 2023, excitement for CGT remains high, but there are lingering concerns about manufacturing and the ability of health care systems to keep up with the science.
As the era of genomic medicine gains speed with the innovation of new technologies, we are at the forefront of genetic tools that can prevent, treat, or cure human diseases. It’s an exciting time, as during this early stage in developing new biological drug products, academics and industry are working closely to optimize cell and gene therapies (CGT) for a range of diseases.
Partnerships and transformation are a large part of our evolving biotech landscape. Not surprisingly, they were two common themes at this year’s Meeting on the Med in Barcelona. As an example, attendees discussed how the lack of harmonization across EU member states has led to fragmented access to advanced therapy medicinal products (ATMPs), which varies from country to country. Chris Fox, president of Novartis Gene Therapies, used her company’s product, ZOLGENSMA®, as an example. It’s approved for use in 48 countries but is only accessible in 35.
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.
My last post gave a short introduction to the manufacture of in vitro transcribed (IVT) messenger RNA (mRNA) for vaccines and therapies, outlining the steps from preclinical to clinical stages of production. In this post, I’ll touch on manufacturing considerations, beginning with the upstream process for IVT mRNA.
Witnessing firsthand the growth of cell and gene therapy to progress more programs to the bedside is an exciting time in the biotechnology industry, particularly with new partnership approaches being developed. I was able to get an overview of one particular partnership during a podcast featuring Tom Foti, VP of Protein Business unit at Aldevron, and Kris Saha, Ph.D., Associate Professor of Biomedical Engineering, Medical History and Bioethics at the University of Wisconsin.
Every year, October marks the change in the season, the dropping of temperatures, and the height of conference season for biotech. This year’s ESGCT meeting in Edinburgh, Scotland, was the event’s first in-person occurrence since 2019, and it was exciting to be back – Edinburgh didn’t disappoint! There was rich dialogue amongst industry experts, healthy attendance with over 2,000 delegates, exceptional networking events, and the city of Edinburgh was a joy with several unseasonably sunny days.
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.
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.
