Streamlining development, ensuring quality and safety

When discussing mRNA development, the complexity across a variety of modalities can give pause to how the projects should be approached and developed. With that in mind, Jon Cooper, Ph.D., Senior Director of RNA Sciences & Analytical Development recently presented, How a Sponsored CMC Platform Accelerates mRNA Projects into the Clinic, in this webinar and recently at the mRNA Analytical Development Summit in Boston, to provide an outline of an effective development process.

What do they mean for you?

Capacity: The total amount that can be contained or produced
Capability: The ability to do something
*As defined by the Cambridge Dictionary

One of the issues I’ve heard raised in our industry is about how we define capacity and capability. As you can see from the definitions above, these are two very different items. But what do these two terms mean with respect to the manufacture of mRNA drug substance and/or drug product?

mRNA’s rise amidst the COVID pandemic

The introduction of mRNA vaccines by Pfizer and Moderna for COVID-19 took much of the world by surprise. But for Katalin Karikó, Pieter Cullis, and Drew Weissman, among others, it was merely the culmination of decades of research and development. Implementing a novel modality during such a public health crisis took confidence, determination, and the hard work of millions of people. It didn’t happen overnight.

Downstream challenges in mRNA production

In my previous post, I brought up important questions that should be asked when beginning to design a scalable manufacturing process to meet specific use requirements. In this post, some of the difficulties encountered in process development are reviewed.

Production paths to mRNA

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.

From potential to solution

For more than two decades, in vitro transcribed (IVT) messenger RNA (mRNA) has been considered for its potential to address unmet medical needs and to transform the development and manufacture of medicines. This potential was suddenly realized by the unprecedented response to Covid 19, ushering in a new era in the application of mRNA technologies to solve challenging health problems.

Defining paths and building relationships

While research into RNA, including mRNA and related derivatives, has been ongoing for almost 50 years, it’s taken the COVID-19 pandemic to accelerate the development of vaccines using this technology. mRNA provides an incredible opportunity to develop new treatments for viral based, genetically driven, or cancerous diseases.

A plasmid replication challenge

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.

Capacity is only part of the solution

During conferences and seminars this year, there has been one particular topic that has come to the fore regarding mRNA production – capacity. Most of the concern comes from the impression that there isn’t enough capacity to meet market demand.

An outlook for mRNA, saRNA and Circular RNA

When mRNA vaccines began to roll out in late 2020 to fight the COVID pandemic, it was the first large-scale use of the molecule after years of research. But the success of those vaccines was only the beginning of new opportunities for the RNA field.