Moving Beyond the Beginning

January 3, 2024 by Mark Wetzel

mRNA observations from Berlin

Many people have focused on the potential of what mRNA can accomplish as a therapeutic modality, though the actual breadth of that potential is only now being realized. This was quite evident at one of the premiere events for the field, the International mRNA Health Conference, held in Berlin, Germany, recently, with a great number of questions and observations about the challenges facing the industry.

And who better to provide the backdrop for those questions and challenges than Dr. Katalin Karikó, 2023 Nobel Prize winner in Physiology or Medicine, an mRNA pioneer. In her opening remarks, she detailed the history of mRNA research, challenges along the way and the discoveries that led to the COVID-19 vaccine, which have opened the door for further breakthrough mRNA breakthrough therapeutics.

Conference sessions included a balanced perspective between remarkable scientific achievements, but also remaining challenges that exist for mRNA as a more broadly adopted modality. Several themes emerged:

• An ongoing quest to make an optimal RNA for a given purpose
• While there have been Incredible delivery advances, targeting is still the top challenge
• How can we increase the stability of mRNA at 4°C?
• A need to advance a vaccine platform beyond single antigen

What a difference a year makes
We’ve seen the mRNA field has made substantial advances in just the past year. There have been programs initiated to expand mRNA use in several areas dealing with other human diseases beyond COVID, and to broader areas such as community health and food safety, with some of these programs designed to address acute and chronic human conditions.

One specific area of interest concerned liquid nanoparticle (LNP) advances, such as new formulations, targeting of specific organs and material science. This is an exciting area of the mRNA field where we expect to see further developments as we move forward. A particular area where the excitement is warranted is where several groups are beginning to target solid tumors via administration of modified mRNAs encapsulated singly or through multi-mRNA encapsulation approaches.

This of course requires strong manufacturing expertise, which Aldevron Chief Scientific Officer Venkata Indurthi, Ph.D., addressed in his talk, Streamlined mRNA Manufacturing: Innovative Solutions to Reduce Timeline to Clinic. Scott Ripley, Ph.D., General Manager, Nucleic Acid Therapeutics at Cytiva, presented a talk on Addressing GMP Manufacturing Challenges of mRNA-LNP Drug Products, where he further detailed the complexities of producing and encapsulating mRNA into LNPs.

Building the future
As mRNA advances, it’s critical to build an advocacy network. That is why, outside of the science, the key highlight of the event was the launch of the new Alliance for mRNA Medicines (AMM), with Danaher, Aldevron’s parent corporation, being a charter member of the organization. AMM is set to become a leading global organization dedicated to advancing and advocating for mRNA and next-generation encoding RNA therapeutics and vaccines for the benefit of patients, public health, and society.

We look forward to advancements in the field and the advocacy of AMM. As part of that, I’ll share more about the scientific discussions from the event, discussing more on what the future of mRNA may hold.

• Learn more about Aldevron’s mRNA platform and services
• Have questions on this topic? Contact Mark Wetzel
• Visit the AMM website
• Have an idea for a topic? Let us know!

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