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.
In the past, the TIDES USA conference event has been focused on oligonucleotide and peptide therapeutics, providing information on development trends and technologies for those industry segments. That is changing now, with an additional focus on mRNA therapeutics coming into the mix.
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.
Successful – for both Aldevron and our clients. That is how I would describe Aldevron’s first Shingijutsu Kaizen event and the first Danaher CEO Kaizen event of 2023, held the week of April 10. Not only did the efforts surpass all expectations but it surpassed deliverable goals--success!
After years of service providers becoming more specialized, we’re now seeing a reversal in that trend. Enterprise offerings and solutions are on the rise, from consumer offerings to huge corporate deals. We can attribute this growth to an increased demand for simplicity of transactions, clear accountability, and building a win-win situation for both parties.
Recombinant proteins are an essential component of many vaccines. However, producing these crucial proteins can be challenging. From selecting the appropriate expression system to optimizing fermentation and purification processes, there are many factors to consider when designing a process to make high-quality proteins that can be used for vaccine development.
Imagine trying to hold water in a shape without it being in a container. That essentially summarizes mRNA without encapsulation. mRNA is, by nature, very fragile, so you need to protect it so that it can reach its targeted location when it goes into a body. Without that protection, mRNA will be quickly denatured or hydrolyzed. That’s why encapsulation is so important when developing mRNA-based therapeutics.
Overnight success is often the result of years of effort and setbacks, which could be the life story of an mRNA pioneer, Dr. Katalin Karikó, now with the Perelman School of Medicine at the University of Pennsylvania. Karikó spent decades focusing her research on how mRNA could be used for therapies, which ultimately helped lead to the development of COVID-19 vaccines.
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.
