Top 5 mRNA/RNA Advancements of 2024 (Part 2)
By Anna Rose Welch, Editorial & Community Director, Advancing RNA
How hard should it be to summarize a year of the encoding RNA space in a single column?
Well, considering I set out to do that very task and am now on part 2 of that “single” article should suggest it’s not quite as easy or as concise a task as I had anticipated. This probably shouldn’t be surprising to me (or to any of you who are regular readers of my often-multi-part articles.) After all, I’ve spent a year talking about the numerous ways in which working with a novel modality and building a sector from the ground up is not an easy task.
Though 2024 may have felt a bit like stumbling around in the dark (as most “beginnings” do), as I took stock of the year behind us, there were five overarching ways in which I saw our industry taking steps forward and finding itself on sturdier scientific and clinical footing. You can read my first two takeaways here in Part 1.
Here, in part 2, I continue down this list with my last three takeaways, starting with…
mRNA Gets Some Much- Needed Representation
In October of 2023, the Alliance for mRNA Medicines launched during the mRNA Healthcare Conference in Berlin. Over the past year, I’ve continued to track the organization’s growth, both in terms of membership (now totaling 70+ companies), as well as engagement/learning initiatives. In the past year, the organization aired two webinars — one on the highly anticipated Platform Designation Guidance, as well as one on advancements in RNA modifications. Advancing RNA was also happy to be home to two pieces of contributed editorial on the current and future state of the industry penned by AMM members Khaled Yamout and Deborah Day Barbara (“mRNA: The Fourth Pillar of Pharmaceutical Innovation & Intervention,” and “Innovation Advances In mRNA Medicine.”) I was also thrilled to be able to participate in two of the organization’s in-person meetings, including a summit on harmonizing the mRNA regulatory pathway, as well as the inaugural Ascent meeting.
It goes without saying that having an organization within which we can unite and continue to learn from is a necessity for any industry — especially for a new sector that is facing rapid (but also nuanced) growth in the upcoming years. To ensure those products grow the way we/our companies and our patients need them to, it’s critical that there are are not only supportive reimbursement and regulatory policies in place for their continued R&D and commercialization, but there also must be an understanding and trust of mRNA technology. Fortunately, throughout the year, AMM has also been holding forums with global regulators, collaborating with legislators on the Hill, as well as hosting events on how to confront the misinformation that’s swirling around mRNA vaccines.
mRNA/RNA is no different than us, really (actually, she is us/inside us); she wants and deserves to be seen. Though AMM is still in its infancy/in growth mode, it’s comforting to know we now have an organization that will advocate for our companies’ and products’ best interests and ensure our mRNA/RNA therapies get the attention and respect they deserve.
Partnerships & Acquisitions Reveal We Can’t Do It All On Our Own
In the earlier days of the cell and gene therapy space, there was a big debate on whether to bring manufacturing in-house or to outsource it (build vs. buy). I see a somewhat similar discussion emerging in the mRNA space; however, it’s not a discussion about building or buying. Rather, the bigger question I see emerging is whether we (RNA biotechs) can or should do it all. Should we integrate mRNA/RNA and LNP expertise under one roof, or should we instead collaborate with a partner that is specialized in nucleic acid delivery?
Now, those of you who have worked in/observed the evolutions in the cell and gene therapy space in previous years will know that the answer to the build vs. buy question was dictated predominantly by funding and a company’s strategy (and in some cases pandemic-era constriction in the outsourcing sector). Similarly, the answer to the integrate vs. partner question in the RNA-LNP space will depend on a company’s size, resources, talent, and strategy. However, throughout 2024, our ongoing work in the therapeutic realm has continued to illuminate the complexities of developing LNPs. As one executive nicely explained in part one of my 2025 RNA outlook articles, the delivery challenge has continued to emphasize that LNP development for all our different cargos, organs, and therapeutic approaches is an art that does not permit a one-size-fits all strategy. Likewise, this recent Advancing RNA panel on LNP R&D/CMC provides even more color to the “art” or LNP development.
Though RNA delivery has always been considered one of the most formidable barriers to overcome, in the past year, we started to see more companies making high profile decisions to address the integrate vs. partner question. In particular, there were a few big acquisitions/mergers and partnership announcements in 2024, including the recent merger of Chroma & Nvelop Therapeutics (now nChroma), and Orna’s acquisition of Renegade. Likewise, we saw LNP platform company NanoVation Therapeutics team up with Novo Nordisk for RNA delivery and RNA immunotherapy company Epitopea join forces with delivery company Genevant.
Academia Positions Itself To Help Our Science “Catch Up”
To be clear, academia has always been in the mRNA game; after all, Kariko and Weismann’s Nobel Prize-winning discovery happened at the University of Pennsylvania. However, in the past year or so, I’d argue there’s been a resurgence or perhaps a new-found appreciation for the work that academia can do to help push forward our understanding of RNA biology, function, and development. And it couldn’t come at a better time; as one speaker emphasized during the AMM Ascent conference back in November, “We need to allow the science to catch up.”
It's impossible these days to scroll through LinkedIn and not find research updates from leading academic researchers. Over the past year in particular, I’ve noted a number of updates from the University of Pennsylvania, in particular, a number of which have been nicely catalogued here in this blog post (“A year after the Nobel Prize, Penn’s mRNA research is revving up”).
However, it’s also worth noting and celebrating the number of new academic RNA centers that have been announced or come “online” in the past year, including the Johns Hopkins RNA Innovation Center; the University of Virgina’s Center for RNA Science & Medicine; and the University of Rochester’s and University of Albany’s partnership forming the Center of Excellence in RNA Research. Outside of the U.S., Australia continues to bolster its identity as an RNA ecosystem. Earlier in 2024, Macquarie University announced the start of construction on an RNA research and pilot manufacturing center, and the University of Western Australia received government investment to launch a production facility to develop RNA oncology therapies.
On the manufacturing front, Advancing RNA contributors Zoltan Kis and Adithya Nair with the University of Sheffeld have published several columns over the past year outlining different technological advancements in the mRNA space, including this article on Quality by Digital Design (QbDD) and continuous mRNA purification using Oligo-DT. And, of course, we can’t forget the news out of MIT about its (FDA-supported!) three-year research program to establish a pilot-scale mRNA continuous manufacturing platform in partnership with ReciBioPharm.
These observations and the examples I’ve provided throughout this wrap-up are hardly all-inclusive. If there are any trends you have been taking stock of over the past year, or if you have any additional thoughts/details to add to any of my points above, please don’t hesitate to be in touch via my email or LinkedIn.
Wishing you a happy — and productive — 2025 and thank you (times a million) for your readership/support of Advancing RNA throughout 2024 and in the future.
Miss part 1? Check it out here.