Climbing Towards the mRNA Summit

August 16, 2023 / by Kyle Studey

Celebrating the work ahead

On my flight to Boston for the mRNA-Based Therapeutics Summit, I followed my usual routine as an uneasy flier; put headphones on and tune out until landing. This time, however, the music continued as I found certain lyrics of one particular song repeating in my mind throughout the two-day gathering of industry leaders:

And just as these words reflect on a transition period, my time at this meeting felt like one of celebration of work left to do, a common theme amongst the community as we enter our second year since full FDA approvals of Spikevax and Comirnaty. A greater focus on supply chain, logistics, and the regulatory landscape constantly underpinned development updates and clinical data, as the industry faces the next wave of mRNA therapy launches in a post-pandemic world.

The Ways that We’ve Grown

The main event kicked off with a panel discussion, instilling the theme of the conference early on. Panelists commented on the work their companies are doing, and where we can take mRNA technology in the future. Even as we think about all the possibilities, we were reminded that as mRNA is a highly adaptable, potent format, we also need to focus on unmet needs where mRNA therapies might offer a glimpse of hope.

It was an excellent segue into the first presentation of clinical data, that of Moderna’s mRNA-1345 vaccine for Respiratory Syncytial Virus (RSV), which follows in the steps of GSK and Pfizer, where there is finally a safe and effective vaccine after 60 years of effort. BioNTech followed with their own update on Autogene Cevumeran, a cancer therapy under their iNeST platform. The clinical data in one track was accompanied by analytics in another, as companies showed new techniques, along with advancements in older techniques, to further improve the characterization of mRNA drug products.

In yet another track, CMC was the focus, highlighting key lessons learned as companies move from bench scale to the clinic, as well as developing and optimizing platform processes that enable shorter lead times. The air was filled with such revelry over these successes that not even a Boston thunderstorm could dampen it.

The Places We’ll Go

It was Peter Marks, Director of CBER, who offered a note of caution to the community on Day 2 about complacency, however, as the future of platform technology was probed. He was joined by Marco Cavaleri of the EMA and others in a panel discussion where the 2022 FDA Reform Act was highlighted.

While I wonder if Marks’ message that, “we will know (what a platform technology looks like) when we see it,” satisfied the audience, it was clear that the regulatory bodies recognize the desire to streamline the data generation and review process to bring therapies to patients as safely and efficiently as possible.

Finally, the need for a high quality, reliable, simplified supply chain was made clear. And whether drug sponsors choose to manufacture their products in house with acquired raw materials, or outsource to a true end-to-end manufacturing partner, ensuring programs can support the rigors of the approval process requires a lot more questions than “can I have a quote?”. Being prepared to answer those questions will only build on the excitement of the places we’ll go.

• Want to learn more about this topic? Contact Kyle Studey
• Read more about Respiratory Syncytial Virus (RSV) vaccines in Patrick Paez's Perspectives post
• Watch Jon Cooper's webinar on CMC platform optimization
• Visit our mRNA Platform page
• Have a topic to suggest? Let us know!